11,940 results
Search Results
2. Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP)
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Bernard J. Smilde, Clemens Stockklausner, Richard Keen, Andrew Whittaker, Alex N. Bullock, Annette von Delft, Natasja M. van Schoor, Paul B. Yu, and E. Marelise W. Eekhoff
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Fibrodysplasia Ossificans Progressiva ,Saracatinib ,AZD0530 ,Clinical trial ,Drug repurposing ,Drug repositioning ,Diseases of the musculoskeletal system ,RC925-935 - Abstract
Abstract Background Fibrodysplasia Ossificans Progressiva (FOP) is a genetic, progressive and devastating disease characterized by severe heterotopic ossification (HO), loss of mobility and early death. There are no FDA approved medications. The STOPFOP team identified AZD0530 (saracatinib) as a potent inhibitor of the ALK2/ACVR1-kinase which is the causative gene for this rare bone disease. AZD0530 was proven to prevent HO formation in FOP mouse models. The STOPFOP trial investigates the repositioning of AZD0530, originally developed for ovarian cancer treatment, to treat patients with FOP. Methods The STOPFOP trial is a phase 2a study. It is designed as a European, multicentre, 6-month double blind randomized controlled trial of AZD0530 versus placebo, followed by a 12-month trial comparing open-label extended AZD0530 treatment with natural history data as a control. Enrollment will include 20 FOP patients, aged 18–65 years, with the classic FOP mutation (ALK2 R206H). The primary endpoint is objective change in heterotopic bone volume measured by low-dose whole-body computer tomography (CT) in the RCT phase. Secondary endpoints include 18F NaF PET activity and patient reported outcome measures. Discussion Clinical trials in rare diseases with limited study populations pose unique challenges. An ideal solution for limiting risks in early clinical studies is drug repositioning – using existing clinical molecules for new disease indications. Using existing assets may also allow a more fluid transition into clinical practice. With positive study outcome, AZD0530 may provide a therapy for FOP that can be rapidly progressed due to the availability of existing safety data from 28 registered clinical trials with AZD0530 involving over 600 patients. Trial registration EudraCT, 2019–003324-20. Registered 16 October 2019, https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-003324-20/NL . Clinicaltrials.gov , NCT04307953 . Registered 13 March 2020.
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- 2022
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3. Protocols for multi‐site trials using hyperpolarized 129 Xe MRI for imaging of ventilation, alveolar‐airspace size, and gas exchange: A position paper from the 129 Xe MRI clinical trials consortium
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Jonathan H. Rayment, Bastiaan Driehuys, Chase S. Hall, G. Wilson Miller, Zackary I. Cleveland, Sarah Svenningsen, Rachel L. Eddy, Jim M. Wild, John P. Mugler, Ho-Fung Chan, Peter Niedbalski, Mario Castro, Neil J. Stewart, Sean B. Fain, Giles E. Santyr, Brandon Zanette, Jason C. Woods, Guilhem Collier, Grace Parraga, Robert P. Thomen, Matthew M. Willmering, and Jaime F. Mata
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medicine.medical_specialty ,business.industry ,Image quality ,Multi site ,Clinical trial ,Lung structure ,Breathing ,Medicine ,Image acquisition ,Position paper ,Radiology, Nuclear Medicine and imaging ,Medical physics ,business ,Pulmonary disorders - Abstract
Hyperpolarized (HP) 129 Xe MRI uniquely images pulmonary ventilation, gas exchange, and terminal airway morphology rapidly and safely, providing novel information not possible using conventional imaging modalities or pulmonary function tests. As such, there is mounting interest in expanding the use of biomarkers derived from HP 129 Xe MRI as outcome measures in multi-site clinical trials across a range of pulmonary disorders. Until recently, HP 129 Xe MRI techniques have been developed largely independently at a limited number of academic centers, without harmonizing acquisition strategies. To promote uniformity and adoption of HP 129 Xe MRI more widely in translational research, multi-site trials, and ultimately clinical practice, this position paper from the 129 Xe MRI Clinical Trials Consortium (https://cpir.cchmc.org/XeMRICTC) recommends standard protocols to harmonize methods for image acquisition in HP 129 Xe MRI. Recommendations are described for the most common HP gas MRI techniques-calibration, ventilation, alveolar-airspace size, and gas exchange-across MRI scanner manufacturers most used for this application. Moreover, recommendations are described for 129 Xe dose volumes and breath-hold standardization to further foster consistency of imaging studies. The intention is that sites with HP 129 Xe MRI capabilities can readily implement these methods to obtain consistent high-quality images that provide regional insight into lung structure and function. While this document represents consensus at a snapshot in time, a roadmap for technical developments is provided that will further increase image quality and efficiency. These standardized dosing and imaging protocols will facilitate the wider adoption of HP 129 Xe MRI for multi-site pulmonary research.
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- 2021
4. A white paper on a neurodevelopmental framework for drug discovery in autism and other neurodevelopmental disorders
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Anett Kaale, Tony Charman, Covadonga M. Díaz-Caneja, Claudia Bagni, Matthew W. State, Stefan Leucht, Declan G. Murphy, F. de Andres-Trelles, Spyridon Siafis, Jan K. Buitelaar, Oscar Marín, Emily Simonoff, C. Arango, Daniel Umbricht, J Cusak, Randi J Hagerman, Gahan Pandina, M. Parellada, Sébastien Jacquemont, Eva Loth, P P Wang, and Baltazar Gomez-Mancilla
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Autism ,Psychological intervention ,03 medical and health sciences ,0302 clinical medicine ,White paper ,Clinical trials ,Multidisciplinary approach ,130 000 Cognitive Neurology & Memory ,medicine ,Genetics ,Humans ,Pharmacology (medical) ,Autistic Disorder ,Child ,Biological Psychiatry ,Pharmacology ,Neurodevelopmental disorders Donders Center for Medical Neuroscience [Radboudumc 7] ,Drug discovery ,Neurodevelopmental disorders ,Settore BIO/13 ,medicine.disease ,030227 psychiatry ,ddc ,Clinical trial ,Neuropsychopharmacology ,Psychiatry and Mental health ,Neurology ,Drug development ,Engineering ethics ,Neurology (clinical) ,030217 neurology & neurosurgery - Abstract
Contains fulltext : 235396.pdf (Publisher’s version ) (Open Access) In the last decade there has been a revolution in terms of genetic findings in neurodevelopmental disorders (NDDs), with many discoveries critical for understanding their aetiology and pathophysiology. Clinical trials in single-gene disorders such as fragile X syndrome highlight the challenges of investigating new drug targets in NDDs. Incorporating a developmental perspective into the process of drug development for NDDs could help to overcome some of the current difficulties in identifying and testing new treatments. This paper provides a summary of the proceedings of the 'New Frontiers Meeting' on neurodevelopmental disorders organised by the European College of Neuropsychopharmacology in conjunction with the Innovative Medicines Initiative-sponsored AIMS-2-TRIALS consortium. It brought together experts in developmental genetics, autism, NDDs, and clinical trials from academia and industry, regulators, patient and family associations, and other stakeholders. The meeting sought to provide a platform for focused communication on scientific insights, challenges, and methodologies that might be applicable to the development of CNS treatments from a neurodevelopmental perspective. Multidisciplinary translational consortia to develop basic and clinical research in parallel could be pivotal to advance knowledge in the field. Although implementation of clinical trials for NDDs in paediatric populations is widely acknowledged as essential, safety concerns should guide each aspect of their design. Industry and academia should join forces to improve knowledge of the biology of brain development, identify the optimal timing of interventions, and translate these findings into new drugs, allowing for the needs of users and families, with support from regulatory agencies.
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- 2021
5. Conducting clinical trials in heart failure during (and after) the COVID-19 pandemic: an Expert Consensus Position Paper from the Heart Failure Association (HFA) of the European Society of Cardiology (ESC)
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Petar M. Seferovic, Subodh Verma, Hiroyuki Tsutsui, Jian Zhang, JoAnn Lindenfeld, John G.F. Cleland, Carolyn S.P. Lam, Johann Bauersachs, Eugene Braunwald, Giuseppe M.C. Rosano, Janet Wittes, Javed Butler, Stuart J. Pocock, Mandeep R. Mehra, Gerasimos Filippatos, Marco Metra, John J.V. McMurray, Muhammad Shahzeb Khan, Piotr Ponikowski, Vijay K. Chopra, Stefan D. Anker, Dirk J. van Veldhuisen, Andrew J.S. Coats, Adrian F. Hernandez, Burkert Pieske, Justin A. Ezekowitz, William T. Abraham, Edimar Alcides Bocchi, Tim Friede, John R. Teerlink, Biykem Bozkurt, Milton Packer, Faiez Zannad, Adriaan A. Voors, and Cardiovascular Centre (CVC)
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medicine.medical_specialty ,Social contract ,Clinical trials ,Coronavirus ,COVID-19 ,Heart failure ,Clinical Trials as Topic ,Europe ,Humans ,Informed Consent ,Patient Safety ,Patient Selection ,Research Design ,Betacoronavirus ,Coronavirus Infections ,Heart Failure ,Pandemics ,Pneumonia, Viral ,Clinical Sciences ,Cardiorespiratory Medicine and Haematology ,030204 cardiovascular system & hematology ,03 medical and health sciences ,Patient safety ,0302 clinical medicine ,Internal medicine ,Pandemic ,Medicine ,Viral ,030212 general & internal medicine ,Association (psychology) ,business.industry ,Risk of infection ,Pneumonia ,medicine.disease ,3. Good health ,Clinical trial ,Cardiovascular System & Hematology ,Cardiology ,Position paper ,Cardiology and Cardiovascular Medicine ,business - Abstract
Author(s): Anker, Stefan D; Butler, Javed; Khan, Muhammad Shahzeb; Abraham, William T; Bauersachs, Johann; Bocchi, Edimar; Bozkurt, Biykem; Braunwald, Eugene; Chopra, Vijay K; Cleland, John G; Ezekowitz, Justin; Filippatos, Gerasimos; Friede, Tim; Hernandez, Adrian F; Lam, Carolyn SP; Lindenfeld, JoAnn; McMurray, John JV; Mehra, Mandeep; Metra, Marco; Packer, Milton; Pieske, Burkert; Pocock, Stuart J; Ponikowski, Piotr; Rosano, Giuseppe MC; Teerlink, John R; Tsutsui, Hiroyuki; Van Veldhuisen, Dirk J; Verma, Subodh; Voors, Adriaan A; Wittes, Janet; Zannad, Faiez; Zhang, Jian; Seferovic, Petar; Coats, Andrew JS | Abstract: The coronavirus disease 2019 (COVID-19) pandemic, caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has important implications for the safety of participants in clinical trials and the research staff caring for them and, consequently, for the trials themselves. Patients with heart failure may be at greater risk of infection with COVID-19 and the consequences might also be more serious, but they are also at risk of adverse outcomes if their clinical care is compromised. As physicians and clinical trialists, it is our responsibility to ensure safe and effective care is delivered to trial participants without affecting the integrity of the trial. The social contract with our patients demands no less. Many regulatory authorities from different world regions have issued guidance statements regarding the conduct of clinical trials during this COVID-19 crisis. However, international trials may benefit from expert guidance from a global panel of experts to supplement local advice and regulations, thereby enhancing the safety of participants and the integrity of the trial. Accordingly, the Heart Failure Association of the European Society of Cardiology on 21 and 22 March 2020 conducted web-based meetings with expert clinical trialists in Europe, North America, South America, Australia, and Asia. The main objectives of this Expert Position Paper are to highlight the challenges that this pandemic poses for the conduct of clinical trials in heart failure and to offer advice on how they might be overcome, with some practical examples. While this panel of experts are focused on heart failure clinical trials, these discussions and recommendations may apply to clinical trials in other therapeutic areas.
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- 2020
6. Immunotherapy in older patients with non-small cell lung cancer: Young International Society of Geriatric Oncology position paper
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Fabio Gomes, Nicolò Matteo Luca Battisti, Tiana Kordbacheh, Andrea Luciani, Alastair Greystoke, M. Kiderlen, Melisa L. Wong, and Radiotherapy
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Oncology ,Cancer Research ,medicine.medical_specialty ,Lung Neoplasms ,Immunosenescence ,medicine.medical_treatment ,Antineoplastic Agents ,Review Article ,03 medical and health sciences ,0302 clinical medicine ,SDG 3 - Good Health and Well-being ,Internal medicine ,Carcinoma, Non-Small-Cell Lung ,medicine ,Humans ,Lung cancer ,Immune Checkpoint Inhibitors ,030304 developmental biology ,0303 health sciences ,Chemotherapy ,Clinical Trials as Topic ,business.industry ,Immunotherapy ,Radioimmunotherapy ,medicine.disease ,Clinical trial ,Radiation therapy ,Geriatric oncology ,030220 oncology & carcinogenesis ,Position paper ,Non small cell ,business ,Non-small-cell lung cancer - Abstract
Immunotherapy with checkpoint inhibitors against programmed cell death receptor (PD-1) and programmed cell death ligand (PD-L1) has been implemented in the treatment pathway of patients with non-small cell lung cancer (NSCLC) from locally advanced disease to the metastatic setting. This approach has resulted in improved survival and a more favourable toxicity profile when compared with chemotherapy. Following the successful introduction of single-agent immunotherapy, current clinical trials are focusing on combination treatments with chemotherapy or radiotherapy or even other immunotherapeutic agents. However, most of the data available from these trials are derived from, and therefore might be more applicable to younger and fitter patients rather than older and often frail lung cancer real-world patients. This article provides a detailed review of these immunotherapy agents with a focus on the data available regarding older NSCLC patients and makes recommendations to fill evidence gaps in this patient population.
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- 2020
7. Placebo effects in allergen immunotherapy-An EAACI Task Force Position Paper
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Oliver Pfaar, Mohamed H. Shamji, Glenis Scadding, Ludger Klimek, Stefan Vieths, Ronald van Ree, Antonella Muraro, Jörg Kleine-Tebbe, Bettina Wedi, Karl Christian Bergmann, Anthony J. Frew, Philippe Devillier, Gunter J. Sturm, Carsten Bindslev-Jensen, Roy Gerth van Wijk, Carmen Vidal, Marek Jutel, Peter S. Creticos, Jean Bousquet, Lars Jacobsen, Nikolaos G. Papadopoulos, Stephen R. Durham, Winfried Rief, Ioana Agache, S. Kaul, Manfred Schedlowski, Peter Hellings, APH - Personalized Medicine, AII - Inflammatory diseases, APH - Global Health, Experimental Immunology, Ear, Nose and Throat, and Internal Medicine
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Allergy ,medicine.medical_specialty ,Allergen immunotherapy ,Blinding ,business.industry ,Advisory Committees ,Immunology ,Medizin ,Disease ,Placebo Effect ,Placebo ,medicine.disease ,law.invention ,Clinical trial ,Treatment Outcome ,Double-Blind Method ,Randomized controlled trial ,Desensitization, Immunologic ,law ,Physical therapy ,Humans ,Immunology and Allergy ,Medicine ,Position paper ,business - Abstract
The placebo (Latin "I will please") effect commonly occurs in clinical trials. The psychological and physiological factors associated with patients' expectations about a treatment's positive and negative effects have yet to be well characterized, although a functional prefrontal cortex and intense bidirectional communication between the central nervous system and the immune system appear to be prerequisites for a placebo effect. The use of placebo raises certain ethical issues, especially if patients in a placebo group are denied an effective treatment for a long period of time. The placebo effect appears to be relatively large (up to 77%, relative to pretreatment scores) in controlled clinical trials of allergen immunotherapy (AIT), such as the pivotal, double-blind, placebo-controlled (DBPC) randomized clinical trials currently required by regulatory authorities worldwide. The European Academy of Allergy and Clinical Immunology (EAACI) therefore initiated a Task Force, in order to better understand the placebo effect in AIT and its specific role in comorbidities, blinding issues, adherence, measurement time points, variability and the natural course of the disease. In this Position Paper, the EAACI Task Force highlights several important topics regarding the placebo effect in AIT such as a) regulatory aspects, b) neuroimmunological and psychological mechanisms, c) placebo effect sizes in AIT trials, d) methodological limitations in AIT trial design and e) potential solutions in future AIT trial design. In conclusion, this Position Paper aims to examine the methodological problem of placebo in AIT from different aspects and also to highlight unmet needs and possible solutions for future trials. ispartof: ALLERGY vol:76 issue:3 pages:629-647 ispartof: location:Denmark status: published
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- 2021
8. Position paper from the Japan Thyroid Association task force on the management of low-risk papillary thyroid microcarcinoma (T1aN0M0) in adults
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Megumi Miyakawa, Iwao Sugitani, Fumihiko Furuya, Hiroki Shimura, Yusaku Yoshida, Kazuhiko Horiguchi, Hidekazu Nagano, Naoya Emoto, Toshihiko Kasahara, Katsunori Manaka, Norisato Mitsutake, Hisakazu Shindo, Kenji Iwaku, Susumu Yokoya, Satoru Suzuki, Noriko Makita, Fumihiko Matsumoto, and Junichiro Sato
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Adult ,Pediatrics ,medicine.medical_specialty ,Endocrinology, Diabetes and Metabolism ,Thyroid Gland ,030209 endocrinology & metabolism ,Thyroid carcinoma ,03 medical and health sciences ,0302 clinical medicine ,Endocrinology ,Quality of life ,Japan ,medicine ,Humans ,Thyroid Neoplasms ,Overdiagnosis ,Adverse effect ,Watchful Waiting ,business.industry ,Incidence (epidemiology) ,Thyroid ,Clinical trial ,medicine.anatomical_structure ,Thyroid Cancer, Papillary ,030220 oncology & carcinogenesis ,Position paper ,business - Abstract
The incidence of thyroid carcinoma has been increasing worldwide. This is interpreted as an increase in the incidental detection of papillary thyroid microcarcinomas (PTMCs). However, mortality has not changed, suggesting overdiagnosis and overtreatment. Prospective clinical trials of active surveillance for low-risk PTMC (T1aN0M0) have been conducted in two Japanese institutions since the 1990s. Based on the favorable outcomes of these trials, active surveillance has been gradually adopted worldwide. A task force on the management of PTMC in adults organized by the Japan Thyroid Association therefore conducted a systematic review and has produced the present position paper based on the scientific evidence concerning active surveillance. This paper indicates evidence for the increased incidence of PTMC, favorable surgical outcomes for low-risk PTMC, recommended criteria for diagnosis using fine needle aspiration cytology, and evaluation of lymph node metastasis (LNM), extrathyroidal extension (ETE) and distant metastasis. Active surveillance has also been reported with a low incidence of disease progression and no subsequent recurrence or adverse events on survival if conversion surgery was performed at a slightly advanced stage. Active surveillance is a safe and valid strategy for PTMC, because it might preserve physical quality of life and reduce 10-year medical costs. However, some points should be noted when performing active surveillance. Immediate surgery is needed for PTMC showing high-risk features, such as clinical LNM, ETE or distant metastasis. Active surveillance should be performed under an appropriate medical team and should be continued for life.
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- 2021
9. Harms in Systematic Reviews Paper 1: An introduction to research on harms
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Tianjing Li, Riaz Qureshi, and Evan Mayo-Wilson
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Research Report ,Epidemiology ,Applied psychology ,Psychological intervention ,Research Personnel ,Terminology ,law.invention ,Clinical trial ,Identification (information) ,Systematic review ,Harm ,Randomized controlled trial ,law ,Meta-analysis ,Humans ,Psychology ,Systematic Reviews as Topic - Abstract
Objective : Most systematic reviews of interventions focus on potential benefits. Common methods and assumptions that are appropriate for assessing benefits can be inappropriate for harms. This paper provides a primer on researching harms, particularly in systematic reviews. Study Design and Setting : Commentary describing challenges with assessing harm. Results : Investigators should be familiar with various terminologies used to describe, classify, and group harms. Published reports of clinical trials include limited information about harms, so systematic reviewers should not depend on these studies and journal articles to reach conclusions about harms. Visualizations might improve communication of multiple dimensions of harms such as severity, relatedness, and timing. Conclusion : The terminology, classification, detection, collection, and reporting of harms create unique challenges that take time, expertise, and resources to navigate in both primary studies and evidence syntheses. Systematic reviewers might reach incorrect conclusions if they focus on evidence about harms found in published reports of randomized trials of a particular health problem. Systematic reviews could be improved through better identification and reporting of harms in primary studies and through better training and uptake of appropriate methods for synthesizing evidence about harms.
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- 2022
10. Subgroup analysis and interpretation for phase 3 confirmatory trials: White paper of the EFSPI/PSI working group on subgroup analysis
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Ilya Lipkovich, Aaron Dane, Tom Parke, Amy V. Spencer, and Gerd K. Rosenkranz
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Statistics and Probability ,Context (language use) ,Subgroup analysis ,Affect (psychology) ,01 natural sciences ,010104 statistics & probability ,03 medical and health sciences ,0302 clinical medicine ,White paper ,Commentaries ,Humans ,Pharmacology (medical) ,030212 general & internal medicine ,0101 mathematics ,Set (psychology) ,Pharmacology ,Interpretation (logic) ,Bootstrapping ,Europe ,Clinical trial ,Clinical Trials, Phase III as Topic ,Research Design ,Data Interpretation, Statistical ,Commentary ,Psychology ,Clinical psychology - Abstract
Subgroup by treatment interaction assessments are routinely performed when analysing clinical trials and are particularly important for phase 3 trials where the results may affect regulatory labelling. Interpretation of such interactions is particularly difficult, as on one hand the subgroup finding can be due to chance, but equally such analyses are known to have a low chance of detecting differential treatment effects across subgroup levels, so may overlook important differences in therapeutic efficacy. EMA have therefore issued draft guidance on the use of subgroup analyses in this setting. Although this guidance provided clear proposals on the importance of pre-specification of likely subgroup effects and how to use this when interpreting trial results, it is less clear which analysis methods would be reasonable, and how to interpret apparent subgroup effects in terms of whether further evaluation or action is necessary. A PSI/EFSPI Working Group has therefore been investigating a focused set of analysis approaches to assess treatment effect heterogeneity across subgroups in confirmatory clinical trials that take account of the number of subgroups explored and also investigating the ability of each method to detect such subgroup heterogeneity. This evaluation has shown that the plotting of standardised effects, bias-adjusted bootstrapping method and SIDES method all perform more favourably than traditional approaches such as investigating all subgroup-by-treatment interactions individually or applying a global test of interaction. Therefore, these approaches should be considered to aid interpretation and provide context for observed results from subgroup analyses conducted for phase 3 clinical trials.
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- 2018
11. Incorporating Digital Tools to Improve Clinical Trial Infrastructure: A White Paper From the Digital Engagement Committee of SWOG
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Jonathan Sommers, Mina S. Sedrak, Don S. Dizon, Mark A. Lewis, AnneMarie Ciccarella, Craig R. Nichols, Michael J. Fisch, Jennifer R. Klemp, Wendy Lawton, Elise D. Cook, and Julie R. Gralow
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Medical education ,business.industry ,Cancer therapy ,MEDLINE ,General Medicine ,Clinical trial ,03 medical and health sciences ,0302 clinical medicine ,White paper ,Work (electrical) ,030220 oncology & carcinogenesis ,Political science ,Social media ,The Internet ,Mobile technology ,030212 general & internal medicine ,business - Abstract
Progress toward improvement in cancer therapy relies on clinical trials. Yet, only a minority of eligible patients with cancer enroll as a result of multiple barriers at the patient, investigator, center, and national level. However, the rise of the Internet and mobile technology has created a slew of tools with medical applications, from Web sites to apps to social media platforms, all of which may aide clinicians in our quest to improve the clinical research enterprise. SWOG is one of five members in the National Cancer Institute’s National Clinical Trials Network—the nation’s oldest and largest publicly funded cancer research network—and is taking a leadership role in exploring and testing the promise of digital engagement through the empaneling of the Digital Engagement Committee. This article outlines the mission, principles, and priorities of the Digital Engagement Committee and proposes how this work may inform the use of digital tools for the cancer research community and, hopefully, translate to improved outcomes for our patients.
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- 2018
12. EASL position paper on the use of COVID-19 vaccines in patients with chronic liver diseases, hepatobiliary cancer and liver transplant recipients
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Markus Cornberg, Maria Buti, Daniel Shouval, Paolo Grossi, Christiane S. Eberhardt, and CiiM, Zentrum für individualisierte Infektionsmedizin, Feodor-Lynen-Str.7, 30625 Hannover.
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medicine.medical_specialty ,COVID-19 Vaccines ,Cirrhosis ,Coronavirus disease 2019 (COVID-19) ,medicine.medical_treatment ,Liver transplantation ,Immunocompromised Host ,Pandemic ,Humans ,Medicine ,Intensive care medicine ,Letter to the Editor ,Hepatology ,business.industry ,SARS-CoV-2 ,Liver Diseases ,Immunogenicity ,Vaccination ,COVID-19 ,medicine.disease ,Influenza ,Clinical trial ,Biliary Tract Neoplasms ,Position paper ,Risk Adjustment ,business ,Vaccine ,Liver Transplantation - Abstract
According to a recent World Health Organization estimate, the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic, which originated in China in 2019, has spread globally, infecting nearly 100 million people worldwide by January 2021. Patients with chronic liver diseases (CLD), particularly cirrhosis, hepatobiliary malignancies, candidates for liver transplantation, and immunosuppressed individuals after liver transplantation appear to be at increased risk of infections in general, which in turn translates into increased mortality. This is also the case for SARS-CoV-2 infection, where patients with cirrhosis, in particular, are at high risk of a severe COVID-19 course. Therefore, vaccination against various pathogens including SARS-CoV-2, administered as early as possible in patients with CLD, is an important protective measure. However, due to impaired immune responses in these patients, the immediate and long-term protective response through immunisation may be incomplete. The current SARS-CoV-2 pandemic has led to the exceptionally fast development of several vaccine candidates. A small number of these SARS-CoV-2 vaccine candidates have already undergone phase III, placebo-controlled, clinical trials in healthy individuals with proof of short-term safety, immunogenicity and efficacy. However, although regulatory agencies in the US and Europe have already approved some of these vaccines for clinical use, information on immunogenicity, duration of protection and long-term safety in patients with CLD, cirrhosis, hepatobiliary cancer and liver transplant recipients has yet to be generated. This review summarises the data on vaccine safety, immunogenicity, and efficacy in this patient population in general and discusses the implications of this knowledge on the introduction of the new SARS-CoV-2 vaccines.
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- 2021
13. Defining the vulnerable patient with myeloma—a frailty position paper of the European Myeloma Network
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Alessandra Larocca, Sonja Zweegman, Monika Engelhardt, Thierry Facon, Gordon Cook, Hematology, and CCA - Cancer Treatment and quality of life
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0301 basic medicine ,Cancer Research ,medicine.medical_specialty ,Frail Elderly ,Disease ,03 medical and health sciences ,0302 clinical medicine ,Biomarkers, Tumor ,Humans ,Medicine ,Precision Medicine ,Intensive care medicine ,Multiple myeloma ,Aged ,Aged, 80 and over ,Frailty ,Clinical screening ,business.industry ,Hematology ,Precision medicine ,medicine.disease ,Clinical Practice ,Clinical trial ,030104 developmental biology ,Oncology ,030220 oncology & carcinogenesis ,Perspective ,Position paper ,Multiple Myeloma ,business - Abstract
As the treatment landscape continues to evolve towards the application of precision medicine in multiple myeloma (MM), there is a clear need to identify those patients who are at risk of not achieving the maximum benefit whilst exposed to the highest level of toxicity. This group of patients, defined as frail, is an unmet clinical need. However, how we define such a vulnerable group of patients with MM remains to be clarified. An integral aspect of this is to define the physiological age and capacity of patients with MM to deal with the burden of their disease and it’s treatment. Such assessments may include not only functional and clinical assessments but also laboratory-based biomarkers of frailty, aging and senescent cellular burden. A need to develop, test and validate clinical screening scores before their adoption into clinical practice is mandated. This position paper from the European Myeloma Network aims to review what is known about defining frailty in MM, and how we can advance this knowledge for the design of clinical trials and ultimately how we deliver treatment in the clinic.
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- 2020
14. Therapeutic strategies for severe COVID-19: a position paper from the Italian Society of Infectious and Tropical Diseases (SIMIT)
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Silvia Nozza, Cristina Mussini, Giampiero Carosi, Antonio Cascio, Francesco Mazzotta, Nicola Petrosillo, Marco Falcone, Roberto Parrella, Paolo Grossi, Antonio Chirianni, Carmelo Iacobello, Evangelista Sagnelli, Claudio Maria Mastroianni, Massimo Galli, Marco Tinelli, Marianna Meschiari, Marcello Tavio, Massimo Andreoni, Giovanni Di Perri, V. Portelli, Chiara Iaria, Orlando Armignacco, Caterina Sagnelli, Mussini, C., Falcone, M., Nozza, S., Sagnelli, C., Parrella, R., Meschiari, M., Petrosillo, N., Mastroianni, C., Cascio, A., Iaria, C., Galli, M., Chirianni, A., Sagnelli, E., Iacobello, C., Di Perri, G., Mazzotta, F., Carosi, G., Tinelli, M., Grossi, P., Armignacco, O., Portelli, V., Andreoni, M., Tavio, M., Mussini C., Falcone M., Nozza S., Sagnelli C., Parrella R., Meschiari M., Petrosillo N., Mastroianni C., Cascio A., Iaria C., Galli M., Chirianni A., Sagnelli E., Iacobello C., Di Perri G., Mazzotta F., Carosi G., Tinelli M., Grossi P., Armignacco O., Portelli V., Andreoni M., and Tavio M.
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0301 basic medicine ,Microbiology (medical) ,medicine.medical_specialty ,Coronavirus disease 2019 (COVID-19) ,030106 microbiology ,Coronaviru ,practice guidelines as topic ,coronavirus ,randomized controlled trials as topic ,Guidelines ,medicine.disease_cause ,medical ,law.invention ,03 medical and health sciences ,0302 clinical medicine ,societies ,Randomized controlled trial ,law ,Pandemic ,italy ,medicine ,pneumonia ,030212 general & internal medicine ,Intensive care medicine ,humans ,Societies, Medical ,Coronavirus ,therapy ,SARS-CoV-2 ,business.industry ,COVID-19 ,Pneumonia ,Therapy ,Standard treatment ,General Medicine ,covid-19 ,sars-cov-2 ,societies, medical ,standard of care ,COVID-19 Drug Treatment ,Clinical trial ,Infectious Diseases ,Position paper ,Observational study ,business ,Human - Abstract
Scope Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection has become pandemic, reaching almost one million death worldwide. At present standard treatment for coronavirus disease 2019 (COVID-19) is not well defined because the evidence, either from randomized or observational studies, with conflicting results, has led to rapid changes in treatment guidelines. Our aim was to narratively summarize the available literature on the management of COVID-19 in order to combine current evidence and interpretation of the data by experts who are treating patients in the frontline setting. Methods The panel conducted a detailed review of the literature and eventual press releases from randomized clinical trials for each possible available treatment. Inductive PubMed search waws performed for publications relevant to the topic, including all clinical trials conducted. The result was a flowchart with treatment indications for patients with COVID-19. Implications After 6 months of a pandemic situation and before a possible second coronavirus wave descends on Europe, it is important to evaluate which drugs proved to be effective while also considering that results from many randomized clinical trials are still awaited. Indeed, among treatments for COVID-19, only glucocorticoids have resulted in an association with a significant decrease in mortality in published randomized controlled trials. New therapeutic strategies are urgently needed.
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- 2021
15. Incorporating patient centered benefits as endpoints in randomized trials of maintenance therapies in advanced ovarian cancer: A position paper from the GCIG symptom benefit committee
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Rosalind Glasspool, Jean-Emmanuel Kurtz, Val Gebski, Anne-Sophie Darlington, Jonathan S. Berek, Effi Yeoshoua, Aleksandra Strojna, Iwa Kong, Laura Farrelly, Orgad Rosenblat, Vladyslav Sukhin, Michael Friedlander, Roldano Fossati, Mariana de Paiva Batista, Phillip Harter, Mark Carey, Ting-Chang Chang, Rahul Roy Chowdhury, Sandra Polleis, Marcia Hall, Chyong-Huey Lai, Byung-Ho Nam, Jung Yun Lee, Richard Schwameis, Patricia Roxburgh, Noriko Fujiwara, Jae Weon Kim, Alexi A. Wright, and Xavier Paoletti
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0301 basic medicine ,medicine.medical_specialty ,Prom ,law.invention ,Maintenance Chemotherapy ,03 medical and health sciences ,0302 clinical medicine ,Quality of life (healthcare) ,Randomized controlled trial ,law ,Patient-Centered Care ,Clinical endpoint ,medicine ,Humans ,Progression-free survival ,Patient Reported Outcome Measures ,Intensive care medicine ,Randomized Controlled Trials as Topic ,Ovarian Neoplasms ,business.industry ,Obstetrics and Gynecology ,female genital diseases and pregnancy complications ,Clinical trial ,030104 developmental biology ,Oncology ,030220 oncology & carcinogenesis ,Quality of Life ,Position paper ,Patient-reported outcome ,Female ,business - Abstract
Background Quality of life and patient reported outcome measures (PROMs) are important secondary endpoints and incorporated in most contemporary clinical trials. There have been deficiencies in their assessment and reporting in ovarian cancer clinical trials, particularly in trials of maintenance treatment where they are of particular importance. The Gynecologic Cancer InterGroup (GCIG) symptom benefit committee (SBC) recently convened a brainstorming meeting with representation from all collaborative groups to address questions of how to best incorporate PROMs into trials of maintenance therapies to support the primary endpoint which is usually progression free survival (PFS). These recommendations should harmonize the collection, analysis and reporting of PROM's across future GCIG trials. Methods Through literature review, trials analysis and input from international experts, the SBC identified four relevant topics to address with respect to promoting the role of PROMs to support the PFS endpoint in clinical trials of maintenance treatment for OC. Results The GCIG SBC unanimously accepted the importance of integrating PROM's in future maintenance trials and developed four guiding principles to be considered early in trial design. These include 1) adherence to SPIRIT-PRO guidelines, 2) harmonization of selection, collection and reporting of PROM's; 3) combining Health Related Quality of Life (HRQL) measures with clinical endpoints and 4) common approaches to dealing with incomplete HRQL data. Conclusions Close attention to incorporating HRQL and PROM's is critical to interpret the results of ovarian cancer clinical trials of maintenance therapies. There should be a consistent approach to assessing and reporting patient centered benefits across all GCIG trials to enable cross trial comparisons which can be used to inform practice.
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- 2020
16. Consensus Paper: Strengths and Weaknesses of Animal Models of Spinocerebellar Ataxias and Their Clinical Implications
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Mario Manto, Michael Strupp, Mandi Gandelman, Hirokazu Hirai, Stefan M. Pulst, Jan Cendelin, Harry T. Orr, Filip Tichanek, Jan Tuma, and Marija Cvetanovic
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medicine.medical_specialty ,Consensus ,Neurology ,Cerebellar ataxia ,business.industry ,Cognition ,medicine.disease ,Article ,Clinical trial ,Mice ,Quality of life (healthcare) ,Animal model ,Cerebellum ,Models, Animal ,Quality of Life ,Spinocerebellar ataxia ,Animals ,Spinocerebellar Ataxias ,Medicine ,Neurology (clinical) ,medicine.symptom ,business ,Neuroscience ,Strengths and weaknesses - Abstract
Spinocerebellar ataxias (SCAs) represent a large group of hereditary degenerative diseases of the nervous system, in particular the cerebellum, and other systems that manifest with a variety of progressive motor, cognitive, and behavioral deficits with the leading symptom of cerebellar ataxia. SCAs often lead to severe impairments of the patient's functioning, quality of life, and life expectancy. For SCAs, there are no proven effective pharmacotherapies that improve the symptoms or substantially delay disease progress, i.e., disease-modifying therapies. To study SCA pathogenesis and potential therapies, animal models have been widely used and are an essential part of pre-clinical research. They mainly include mice, but also other vertebrates and invertebrates. Each animal model has its strengths and weaknesses arising from model animal species, type of genetic manipulation, and similarity to human diseases. The types of murine and non-murine models of SCAs, their contribution to the investigation of SCA pathogenesis, pathological phenotype, and therapeutic approaches including their advantages and disadvantages are reviewed in this paper. There is a consensus among the panel of experts that (1) animal models represent valuable tools to improve our understanding of SCAs and discover and assess novel therapies for this group of neurological disorders characterized by diverse mechanisms and differential degenerative progressions, (2) thorough phenotypic assessment of individual animal models is required for studies addressing therapeutic approaches, (3) comparative studies are needed to bring pre-clinical research closer to clinical trials, and (4) mouse models complement cellular and invertebrate models which remain limited in terms of clinical translation for complex neurological disorders such as SCAs.
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- 2021
17. Pathophysiology and management of sensitive skin: position paper from the special interest group on sensitive skin of the International Forum for the Study of Itch (IFSI)
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Misery, L., Weisshaar, E., Brenaut, E., Evers, A.W.M., Huet, F., Ständer, S., Reich, A., Berardesca, E., Serra‐Baldrich, E., Wallengren, J., Linder, D., Fluhr, J.W., Szepietowski, J.C., Maibach, H., Honari, Golara, Le Gall‐Ianotto, Christelle, Takamori, Kenji, Richters, Renée, Centre Hospitalier Régional Universitaire de Brest (CHRU Brest), and Université de Brest (UBO)
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medicine.medical_specialty ,Skin barrier ,media_common.quotation_subject ,[SDV]Life Sciences [q-bio] ,Pain ,Dermatology ,Cosmetics ,Skin Diseases ,Sensitive skin ,030207 dermatology & venereal diseases ,03 medical and health sciences ,0302 clinical medicine ,[SDV.BC.IC]Life Sciences [q-bio]/Cellular Biology/Cell Behavior [q-bio.CB] ,medicine ,Humans ,Paresthesia ,ComputingMilieux_MISCELLANEOUS ,media_common ,integumentary system ,business.industry ,Pruritus ,[SDV.BBM.BM]Life Sciences [q-bio]/Biochemistry, Molecular Biology/Molecular biology ,[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology ,Pathophysiology ,3. Good health ,Clinical trial ,Infectious Diseases ,Public Opinion ,Position paper ,Tingling ,business ,Psychosocial ,030217 neurology & neurosurgery ,[SDV.MHEP.DERM]Life Sciences [q-bio]/Human health and pathology/Dermatology - Abstract
The special interest group on sensitive skin of the International Forum for the Study of Itch previously defined sensitive skin as a syndrome defined by the occurrence of unpleasant sensations (stinging, burning, pain, pruritus and tingling sensations) in response to stimuli that normally should not provoke such sensations. This additional paper focuses on the pathophysiology and the management of sensitive skin. Sensitive skin is not an immunological disorder but is related to alterations of the skin nervous system. Skin barrier abnormalities are frequently associated, but there is no cause and direct relationship. Further studies are needed to better understand the pathophysiology of sensitive skin - as well as the inducing factors. Avoidance of possible triggering factors and the use of well-tolerated cosmetics, especially those containing inhibitors of unpleasant sensations, might be suggested for patients with sensitive skin. The role of psychosocial factors, such as stress or negative expectations, might be relevant for subgroups of patients. To date, there is no clinical trial supporting the use of topical or systemic drugs in sensitive skin. The published data are not sufficient to reach a consensus on sensitive skin management. In general, patients with sensitive skin require a personalized approach, taking into account various biomedical, neural and psychosocial factors affecting sensitive skin.
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- 2020
18. Nutraceutical support in heart failure: a position paper of the International Lipid Expert Panel (ILEP)
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Agata Bielecka-Dabrowa, Arrigo F G Cicero, Nathan D. Wong, Željko Reiner, Dragos Vinereanu, Claudio Ferri, Peter P. Toth, Maciej Banach, Alessandro Colletti, Amirhossein Sahebkar, Dimitri P. Mikhailidis, Stephan von Haehling, Cicero AF, Colletti A, von Haehling S, Vinereanu D, Bielecka-Dabrowa A, Sahebkar A, Toth PP, Reiner Ž, Wong ND, Mikhailidis DP, Ferri C, Banach M, and Kubilius, Raimondas
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medicine.medical_specialty ,Medicine (miscellaneous) ,Disease ,030204 cardiovascular system & hematology ,Ventricular Function, Left ,03 medical and health sciences ,0302 clinical medicine ,Quality of life ,Fatty Acids, Omega-3 ,medicine ,Humans ,030212 general & internal medicine ,Intensive care medicine ,Heart failure, Nutraceuticals, Coenzyme Q10, Herbal drugs, Nutritional supplements ,Coenzyme Q10 ,Heart failure ,Herbal drugs ,Nutraceuticals ,Nutritional supplements ,Heart Failure ,Nutrition and Dietetics ,Ejection fraction ,business.industry ,Stroke Volume ,Evidence-based medicine ,medicine.disease ,3. Good health ,Clinical trial ,Dietary Supplements ,Quality of Life ,Position paper ,business ,Heart failure with preserved ejection fraction - Abstract
Heart failure (HF) is a complex clinical syndrome that represents a major cause of morbidity and mortality in Western countries. Several nutraceuticals have shown interesting clinical results in HF prevention as well as in the treatment of the early stages of the disease, alone or in combination with pharmacological therapy. The aim of the present expert opinion position paper is to summarise the available clinical evidence on the role of phytochemicals in HF prevention and/or treatment that might be considered in those patients not treated optimally as well as in those with low therapy adherence. The level of evidence and the strength of recommendation of particular HF treatment options were weighed up and graded according to predefined scales. A systematic search strategy was developed to identify trials in PubMed (January 1970 to June 2019). The terms ‘nutraceuticals’, ‘dietary supplements’, ‘herbal drug’ and ‘heart failure’ or ‘left verntricular dysfunction’ were used in the literature search. The experts discussed and agreed on the recommendation levels. Available clinical trials reported that the intake of some nutraceuticals (hawthorn, coenzyme Q10,l-carnitine,d-ribose, carnosine, vitamin D, probiotics,n-3 PUFA and beet nitrates) might be associated with improvements in self-perceived quality of life and/or functional parameters such as left ventricular ejection fraction, stroke volume and cardiac output in HF patients, with minimal or no side effects. Those benefits tended to be greater in earlier HF stages. Available clinical evidence supports the usefulness of supplementation with some nutraceuticals to improve HF management in addition to evidence-based pharmacological therapy.
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- 2020
19. Transplant-ineligible newly diagnosed multiple myeloma: Current and future approaches to clinical care: A Young International Society of Geriatric Oncology Review Paper
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Hira S. Mian, Tanya M. Wildes, Melina Boutin, Smith Giri, Ashley E. Rosko, Shakira J. Grant, Nikita Nikita, Nina Rosa Neuendorff, Sonja Zweegman, Jessica L. Krok-Schoen, and Lorenzo Dottorini
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medicine.medical_specialty ,Vulnerable adult ,Frail Elderly ,Frail Older Adults ,Newly diagnosed ,Transplant ineligible ,Article ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Humans ,030212 general & internal medicine ,Clinical care ,Intensive care medicine ,Geriatric Assessment ,Multiple myeloma ,Aged ,Frailty ,business.industry ,medicine.disease ,Europe ,Clinical trial ,Oncology ,Geriatric oncology ,030220 oncology & carcinogenesis ,Geriatrics and Gerontology ,Multiple Myeloma ,business - Abstract
Multiple myeloma is the second most common hematological malignancy in the USA and Europe. Despite improvements in the 5-year and overall survival rates over the past decade, older adults (aged ≥65 years) with multiple myeloma continue to experience disproportionately worse outcomes than their younger counterparts. These differences in outcomes arise from the increased prevalence of vulnerabilities such as medical comorbidities and frailty seen with advancing age that can influence treatment-delivery and tolerance and impact survival. In general, geriatric assessments can help identify those patients more likely to benefit from enhanced toxicity risk-prediction and aid treatment decision-making. Despite the observed benefits of geriatric assessments and other screening frailty tools, provider and systems-level barriers continue to influence the overall perception of the feasibility of geriatric assessments in clinical practice settings. Clinical trials are underway evaluating the efficacy and safety of various multiple myeloma therapies in less fit/frail older adults, with a minority examining fitness-based/risk-adapted approaches. Thus, significant gaps exist in knowing which myeloma therapies are most appropriate for older and more vulnerable adults with multiple myeloma. The purpose of this Review is to discuss how geriatric assessments can be used to guide the management of transplant-ineligible patients; and to highlight frontline therapies for standard-risk and high-risk cytogenetic abnormalities [i.e., t(4;14), t(14;16), and del(17p)] associated with multiple myeloma. We also discuss the current shortcomings of the existing clinical approaches to care and highlight ongoing clinical trials evaluating newer fitness-based approaches to managing transplant-ineligible patients.
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- 2021
20. Clinical Quantification of Myocardial Blood Flow Using PET: Joint Position Paper of the SNMMI Cardiovascular Council and the ASNC
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Panithaya Chareonthaitawee, Henry Gewirtz, Timothy M. Bateman, Vasken Dilsizian, Raymond R. Russell, Ernest V. Garcia, April Mann, Rob Beanlands, James R. Corbett, Thomas H. Schindler, Edward P. Ficaro, Ronald G. Schwartz, Andrew J. Einstein, Gary V. Heller, Sharmila Dorbala, Howard C. Lewin, Shivali Malhotra, Salvador Borges-Neto, Terrence D. Ruddy, Marcelo F. Di Carli, Manuel D. Cerqueira, Piotr J. Slomka, Prem Soman, Daniel S. Berman, Robert A. deKemp, E. Gordon DePuey, and Venkatesh L. Murthy
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Adult ,medicine.medical_specialty ,Cardiology ,Coronary Artery Disease ,030204 cardiovascular system & hematology ,Cardiovascular System ,030218 nuclear medicine & medical imaging ,Myocardial perfusion imaging ,03 medical and health sciences ,0302 clinical medicine ,Japan ,Ammonia ,Reference Values ,Stress, Physiological ,Coronary Circulation ,Internal medicine ,Image Processing, Computer-Assisted ,medicine ,Humans ,Radiology, Nuclear Medicine and imaging ,Societies, Medical ,Clinical Trials as Topic ,Nitrogen Radioisotopes ,medicine.diagnostic_test ,business.industry ,Myocardial Perfusion Imaging ,Reproducibility of Results ,Diagnostic test ,Blood flow ,Middle Aged ,Prognosis ,United States ,Fractional Flow Reserve, Myocardial ,Clinical trial ,Cardiovascular Diseases ,Regional Blood Flow ,Positron-Emission Tomography ,Position paper ,Radiopharmaceuticals ,Nuclear Medicine ,business ,Cardiology and Cardiovascular Medicine ,Rubidium Radioisotopes - Abstract
Radionuclide myocardial perfusion imaging (MPI) is among the most commonly performed diagnostic tests in cardiology. Although the diagnostic and prognostic applications of radionuclide MPI are supported by a wealth of observational and clinical trial data, its performance is limited by two
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- 2017
21. A Smart Mobile Health Tool Versus a Paper Action Plan to Support Self-Management of Chronic Obstructive Pulmonary Disease Exacerbations: Randomized Controlled Trial
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Lonneke M. Boer, Jan H. Vercoulen, Reinier Akkermans, Maarten van der Heijden, Erik Bischoff, Yvonne F. Heijdra, Willem J. J. Assendelft, Peter J. F. Lucas, Tjard Schermer, and Datamanagement & Biometrics
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Male ,medicine.medical_specialty ,Technology Assessment, Biomedical ,Exacerbation ,Health Informatics ,Information technology ,Symptom flare up ,law.invention ,Healthcare improvement science Radboud Institute for Health Sciences [Radboudumc 18] ,Pulmonary Disease, Chronic Obstructive ,03 medical and health sciences ,0302 clinical medicine ,All institutes and research themes of the Radboud University Medical Center ,Randomized controlled trial ,law ,Surveys and Questionnaires ,Health care ,Software Science ,Self-management ,Humans ,Medicine ,COPD ,030212 general & internal medicine ,mHealth ,Disease burden ,Aged ,Original Paper ,business.industry ,Middle Aged ,T58.5-58.64 ,medicine.disease ,Mobile Applications ,Self Efficacy ,Clinical trial ,030228 respiratory system ,Emergency medicine ,Inflammatory diseases Radboud Institute for Health Sciences [Radboudumc 5] ,Female ,Public aspects of medicine ,RA1-1270 ,business - Abstract
Background Many patients with chronic obstructive pulmonary disease (COPD) suffer from exacerbations, a worsening of their respiratory symptoms that warrants medical treatment. Exacerbations are often poorly recognized or managed by patients, leading to increased disease burden and health care costs. Objective This study aimed to examine the effects of a smart mobile health (mHealth) tool that supports COPD patients in the self-management of exacerbations by providing predictions of early exacerbation onset and timely treatment advice without the interference of health care professionals. Methods In a multicenter, 2-arm randomized controlled trial with 12-months follow-up, patients with COPD used the smart mHealth tool (intervention group) or a paper action plan (control group) when they experienced worsening of respiratory symptoms. For our primary outcome exacerbation-free time, expressed as weeks without exacerbation, we used an automated telephone questionnaire system to measure weekly respiratory symptoms and treatment actions. Secondary outcomes were health status, self-efficacy, self-management behavior, health care utilization, and usability. For our analyses, we used negative binomial regression, multilevel logistic regression, and generalized estimating equation regression models. Results Of the 87 patients with COPD recruited from primary and secondary care centers, 43 were randomized to the intervention group. We found no statistically significant differences between the intervention group and the control group in exacerbation-free weeks (mean 30.6, SD 13.3 vs mean 28.0, SD 14.8 weeks, respectively; rate ratio 1.21; 95% CI 0.77-1.91) or in health status, self-efficacy, self-management behavior, and health care utilization. Patients using the mHealth tool valued it as a more supportive tool than patients using the paper action plan. Patients considered the usability of the mHealth tool as good. Conclusions This study did not show beneficial effects of a smart mHealth tool on exacerbation-free time, health status, self-efficacy, self-management behavior, and health care utilization in patients with COPD compared with the use of a paper action plan. Participants were positive about the supportive function and the usability of the mHealth tool. mHealth may be a valuable alternative for COPD patients who prefer a digital tool instead of a paper action plan. Trial Registration ClinicalTrials.gov NCT02553096; https://clinicaltrials.gov/ct2/show/NCT02553096.
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- 2019
22. Unmet Medical Needs and Future Perspectives for Leiomyosarcoma Patients—A Position Paper from the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN)
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Paul H. Huang, Annie Achee, Robin L. Jones, Scott H. Okuno, Roger Wilson, Neeta Somaiah, Rebecca A. Gladdy, Roberta Sanfilippo, Denise K. Reinke, Jonathan C. Trent, Bernd Kasper, Scott M. Schuetze, Breelyn A. Wilky, Matthew L. Hemming, Seth M. Pollack, Brian A. Van Tine, Gerard van Oortmerssen, Matthew Ingham, and Kathrin Schuster
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0301 basic medicine ,Leiomyosarcoma ,Cancer Research ,medicine.medical_specialty ,Review ,Disease ,lcsh:RC254-282 ,Unmet needs ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Patient group ,Intensive care medicine ,SPAEN ,research ,treatment ,NLMSF ,business.industry ,Foundation (evidence) ,leiomyosarcoma ,lcsh:Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,medicine.disease ,body regions ,Clinical trial ,030104 developmental biology ,Oncology ,030220 oncology & carcinogenesis ,Position paper ,Sarcoma ,business - Abstract
Simple Summary In this position paper, we aim to summarize state-of-the-art treatments for patients with leiomyosarcomas in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This white paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN). Abstract As leiomyosarcoma patients are challenged by the development of metastatic disease, effective systemic therapies are the cornerstone of outcome. However, the overall activity of the currently available conventional systemic treatments and the prognosis of patients with advanced or metastatic disease are still poor, making the treatment of this patient group challenging. Therefore, in a joint effort together with patient networks and organizations, namely Sarcoma Patients EuroNet (SPAEN), the international network of sarcoma patients organizations, and the National LeioMyoSarcoma Foundation (NLMSF) in the United States, we aim to summarize state-of-the-art treatments for leiomyosarcoma patients in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This position paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the NLMSF and SPAEN.
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- 2021
23. Defining breakthrough invasive fungal infection-Position paper of the mycoses study group education and research consortium and the European Confederation of Medical Mycology
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Sharon C.-A. Chen, George Richard Thompson, Dimitrios P. Kontoyiannis, C. Orla Morrissey, Oliver A. Cornely, Martin Hoenigl, and Cornelia Lass-Flörl
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0301 basic medicine ,medicine.medical_specialty ,Antifungal Agents ,Medical mycology ,030106 microbiology ,Clinical Sciences ,Antifungal drug ,Dermatology ,Disease ,mucormycosis ,Microbiology ,Article ,030207 dermatology & venereal diseases ,03 medical and health sciences ,0302 clinical medicine ,Risk Factors ,Epidemiology ,medicine ,Mucormycosis ,Humans ,Aspergillosis ,breakthrough ,Treatment Failure ,Intensive care medicine ,relapse ,Clinical Trials as Topic ,refractoriness ,business.industry ,Group education ,General Medicine ,persistence ,invasive fungal disease ,Clinical trial ,Europe ,Mycoses Study Group Education and Research Consortium (MSG-ERC) and the European Confederation of Medical Mycology ,Clinical research ,Infectious Diseases ,Good Health and Well Being ,Mycoses ,Position paper ,business ,Infection ,Invasive Fungal Infections - Abstract
Breakthrough invasive fungal infections (IFI) have emerged as a significant problem in patients receiving systemic antifungals; however, consensus criteria for defining breakthrough IFI are missing. This position paper establishes broadly applicable definitions of breakthrough IFI for clinical research. Representatives of the Mycoses Study Group Education and Research Consortium (MSG-ERC) and the European Confederation of Medical Mycology (ECMM) reviewed the relevant English literature for definitions applied and published through 2018. A draft proposal for definitions was developed, and circulated to all members of the two organizations for comment and suggestions. The authors addressed comments received, and circulated the updated document for approval. Breakthrough IFI was defined as any IFI occurring during exposure to an antifungal drug, including fungi outside the spectrum of activity of an antifungal. The time of breakthrough IFI was defined as the first attributable clinical sign or symptom, mycological finding or radiological feature. The period defining breakthrough IFI depends on pharmacokinetic properties and extends at least until one dosing interval after drug discontinuation. Persistent IFI describes IFI that is unchanged/stable since treatment initiation with ongoing need for antifungal therapy. It is distinct from refractory IFI, defined as progression of disease and therefore similar to non-response to treatment. Relapsed IFI occurs after treatment, and is caused by the same pathogen at the same site, although dissemination can occur. These proposed definitions are intended to support the design of future clinical trials and epidemiological research in clinical mycology, with the ultimate goal of increasing the comparability of clinical trial results.
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- 2019
24. Cognitive remediation for schizophrenia: An expert working group white paper on core techniques
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Christopher R. Bowie, Jason K. Johannesen, Alice M. Saperstein, Joanna M. Fiszdon, Til Wykes, Elizabeth W. Twamley, Jean Pierre Lindenmayer, Alice Medalia, Morris D. Bell, Susan R. McGurk, Torill Ueland, and Rafael Penadés
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Consensus ,Schizophrenia (object-oriented programming) ,Applied psychology ,Cognition ,Cognitive Remediation ,030227 psychiatry ,Variety (cybernetics) ,Clinical trial ,03 medical and health sciences ,Psychiatry and Mental health ,Core (game theory) ,0302 clinical medicine ,White paper ,Cognitive remediation therapy ,Practice Guidelines as Topic ,Schizophrenia ,Effective treatment ,Humans ,Cognitive Dysfunction ,Psychology ,030217 neurology & neurosurgery ,Biological Psychiatry - Abstract
Cognitive remediation is now widely recognized as an effective treatment for cognitive deficits in schizophrenia. Its effects are meaningful, durable, and related to improvements in everyday functional outcomes. As with many therapies, the evolution of cognitive remediation has resulted in treatment programs that use a variety of specific techniques, yet share common core principles. This paper is the product of a cognitive remediation expert working group consensus meeting to identify core features of the treatment and produce recommendations for its design, conduct, reporting, and implementation. Four techniques were identified as core features of cognitive remediation: facilitation by a therapist, cognitive exercise, procedures to develop problem-solving strategies, and procedures to facilitate transfer to real world functioning. Treatment techniques within each of these core features are presented to facilitate decisions for clinical trials and implementation in clinical settings.
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- 2019
25. Quality of life measurement in skin cancer patients: literature review and position paper of the European Academy of Dermatology and Venereology Task Forces on Quality of Life and Patient Oriented Outcomes, Melanoma and Non-Melanoma Skin Cancer
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A.M. Forsea, Åke Svensson, Matthias Augustin, Pavel V Chernyshov, S. Spillekom-vanKoulil, Jacek C Szepietowski, Andrew Yule Finlay, Aimilios Lallas, Lucia Tomas-Aragones, L. Manolache, Anthony Bewley, Francesca Sampogna, Monika Arenbergerova, Mahtab Samimi, Servando E Marron, Gregor Be Jemec, Danylo Halytskyi Lviv National Medical University, Aristotle University of Thessaloniki, University of Zaragoza - Universidad de Zaragoza [Zaragoza], Charles University in Prague, Partenaires INRAE, Infectiologie et Santé Publique (UMR ISP), Institut National de la Recherche Agronomique (INRA)-Université de Tours, Université de Tours, Dali Medical, Skåne University Hospital, Royo Villanova Hospital, Istituto dermopatico dell'immacolata (IDI-IRCCS), Radboud University Medical Center [Nijmegen], Whipps Cross University Hospital, Royal London Hospital, University of Medicine and Pharmacy 'Carol Davila' Bucharest (UMPCD), Zealand University Hospital, Health Sciences Faculty, University of Cape Town, Department of Dermatology, Venereology, and Allergology, Wroclaw Medical University, Universitaetsklinikum Hamburg-Eppendorf = University Medical Center Hamburg-Eppendorf [Hamburg] (UKE), Cardiff University, Institut National de la Recherche Agronomique (INRA)-Université de Tours (UT), and Université de Tours (UT)
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medicine.medical_specialty ,Skin Neoplasms ,MEDLINE ,Context (language use) ,[SDV.CAN]Life Sciences [q-bio]/Cancer ,Dermatology ,030207 dermatology & venereal diseases ,03 medical and health sciences ,0302 clinical medicine ,Quality of life ,medicine ,Humans ,Melanoma ,business.industry ,Cancer ,Dermatology Life Quality Index ,medicine.disease ,humanities ,3. Good health ,Clinical trial ,Europe ,Infectious Diseases ,Treatment Outcome ,030220 oncology & carcinogenesis ,Case-Control Studies ,Quality of Life ,Position paper ,Skin cancer ,business ,[SDV.MHEP]Life Sciences [q-bio]/Human health and pathology ,[SDV.MHEP.DERM]Life Sciences [q-bio]/Human health and pathology/Dermatology - Abstract
International audience; The European Academy of Dermatology and Venereology (EADV) Task Forces (TFs) on Quality of Life (QoL) and Patient Oriented Outcomes, Melanoma and Non-Melanoma Skin Cancer (NMSC) present a review of the literature and position statement on health-related (HR) QoL assessment in skin cancer patients. A literature search was carried out to identify publications since 1980 that included information about the impact of SC on QoL. Generic, dermatology-specific, cancer-specific, SC-specific, facial SC-specific, NMSC-specific, basal cell carcinoma-specific and melanoma-specific QoL questionnaires have been used to assess HRQoL in SC patients. HRQoL was assessed in the context of creation and validation of the HRQoL instruments, clinical trials, comparison of QoL in SC and other cancers, other diseases or controls, HRQoL assessment after treatment, comorbidities, behaviour modification, predictors of QoL and survival, supportive care needs, coping strategies and fear of cancer recurrence. The most widely used instruments for HRQoL assessment in SC patients are the European Organisation for Research and Treatment of Cancer Core Questionnaire (EORTC QLQ-C30), the Functional Assessment of Cancer Therapy-Melanoma (FACT-M), Skin Cancer Index (SCI), Short Form 36 Item Health Survey (SF-36) and the Dermatology Life Quality Index (DLQI). The TFs recommend the use of the cancer-specific EORTC QLQ-C30, especially in late stages of disease, and the melanoma-specific FACT-M and SC-specific SCI questionnaires. These instruments have been well validated and used in several studies. Other HRQoL instruments, also with good basic validation, are not currently recommended because the experience of their use is too limited. Dermatology-specific HRQoL instruments can be used to assess the impact of skin-related problems in SC. The TFs encourage further studies to validate HRQoL instruments for use in different stages of SC, in order to allow more detailed practical recommendations on HRQoL assessment in SC.
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- 2019
26. A White Paper on Collagen Hydrolyzates and Ultrahydrolyzates: Potential Supplements to Support Joint Health in Osteoarthritis?
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Armaghan Mahmoudian, Ursule Kalvaityte, Lacey J. Favazzo, Cyro Scala de Almeida, Michael J. Zuscik, Stan Kubow, Ali Mobasheri, Christina E. Larder, Pieter J. Emans, Perola Grimberg Plapler, Michèle M. Iskandar, Paulo Cesar Hamdan, Luc J. C. van Loon, Ilona Uzieliene, Humane Biologie, RS: NUTRIM - R3 - Respiratory & Age-related Health, RS: CAPHRI - R3 - Functioning, Participating and Rehabilitation, Orthopedie, MUMC+: MA Orthopedie (9), Physiotherapy, Human Physiology and Anatomy, and Human Physiology and Sports Physiotherapy Research Group
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collagen ,medicine.medical_specialty ,Population ,Pain ,Arthritis ,Context (language use) ,Osteoarthritis ,Disease ,METABOLISM ,dietary supplements ,Denatured collagen ,Nutraceutical ,Pharmacotherapy ,Rheumatology ,FOOD ,Complementary and Alternative Medicine (S Kolasinski, Section Editor) ,Osteoarthritis/drug therapy ,Humans ,Medicine ,HYDROLYSATE ,Intensive care medicine ,education ,Nutritional supplement ,education.field_of_study ,business.industry ,Collagen hydrolyzate ,GUT MICROBIOTA ,IN-VITRO DIGESTION ,medicine.disease ,UC-II ,Clinical trial ,Collagen ultra-hydrolyzate ,ORAL TOLERANCE ,II COLLAGEN ,IMMUNE-SYSTEM ,Joint health ,Joint Diseases ,business ,DIETARY-SUPPLEMENTS - Abstract
Purpose of Review Osteoarthritis (OA) is the most common forms of arthritis in the general population, accounting for more pain and functional disability than any other musculoskeletal disease. There are currently no approved disease modifying drugs for OA. In the absence of effective pharmacotherapy, many patients with OA turn to nutritional supplements and nutraceuticals, including collagen derivatives. Collagen hydrolyzates and ultrahydrolyzates are terms used to describe collagens that have been broken down into small peptides and amino acids in the presence of collagenases and high pressure. Recent Findings This article reviews the relevant literature and serves as a White Paper on collagen hydrolyzates and ultrahydrolyzates as emerging supplements often advertised to support joint health in OA. Collagen hydrolyzates have demonstrated some evidence of efficacy in a handful of small scale clinical trials, but their ability to treat and reverse advanced joint disease remains highly speculative, as is the case for other nutritional supplements. Summary The aim of this White Paper is to stimulate research and development of collagen-based supplements for patients with OA and other musculoskeletal diseases at academic and industrial levels. This White Paper does not make any treatment recommendations for OA patients in the clinical context, but simply aims to highlight opportunities for scientific innovation and interdisciplinary collaboration, which are crucial for the development of novel products and nutritional interventions based on the best available and published evidence.
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- 2021
27. White Paper AGA: Gastroparesis: Clinical and Regulatory Insights for Clinical Trials
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Pankaj J. Pasricha, William L. Hasler, Michael Camilleri, and Henry P. Parkman
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medicine.medical_specialty ,Hepatology ,business.industry ,Clinical study design ,media_common.quotation_subject ,Gastroenterology ,Alternative medicine ,medicine.disease ,Article ,Surgery ,Clinical trial ,03 medical and health sciences ,Presentation ,0302 clinical medicine ,White paper ,Drug development ,030220 oncology & carcinogenesis ,medicine ,Clinical endpoint ,030211 gastroenterology & hepatology ,Gastroparesis ,Intensive care medicine ,business ,media_common - Abstract
Gastroparesis continues to represent a large unmet clinical need and a major opportunity for new drug development. This has led to increasing interest by federal funding agencies, regulatory bodies, and industry. This article summarizes the proceedings of the gastroparesis section of the “Drug Development Conference: Clinical Endpoints in Upper GI Disorders” organized by the American Gastroenterological Association in Washington, DC, on October 27–28, 2016. The presentation, diagnosis, and current therapeutic strategies are briefly reviewed, followed by a detailed discussion of the regulatory strategy, recommended endpoints, and future directions.
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- 2017
28. Research Paper: Effect of Play-based Therapy on Metacognitive and Behavioral Aspects of Executive Function: A Randomized, Controlled, Clinical Trial on the Students With Learning Disabilities
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Akram Parand, Samaneh Karamali Esmaili, Narges Shafaroodi, Afsoon Hassani Mehraban, Saeed Akbari-Zardkhaneh, and Masoume Zarei
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Multivariate analysis ,Metacognition ,Behavioral neuroscience ,lcsh:RC321-571 ,03 medical and health sciences ,Cellular and Molecular Neuroscience ,0302 clinical medicine ,Executive function ,Intervention (counseling) ,medicine ,0501 psychology and cognitive sciences ,lcsh:Neurosciences. Biological psychiatry. Neuropsychiatry ,Learning disability ,Play ,05 social sciences ,Cognition ,Clinical trial ,Behavior Rating Inventory of Executive Function ,Neurology (clinical) ,medicine.symptom ,Psychology ,030217 neurology & neurosurgery ,050104 developmental & child psychology ,Clinical psychology ,Research Paper - Abstract
Introduction: Although the effect of educational methods on executive function (EF) is well known, training this function by a playful method is debatable. The current study aimed at investigating if a play-based intervention is effective on metacognitive and behavioral skills of EF in students with specific learning disabilities. Methods: In the current randomized, clinical trial, 49 subjects within the age range of 7 to 11 years with specific learning disabilities were randomly assigned into the intervention (25 subjects; mean age 8.5±1.33 years) and control (24 subjects; mean age 8.7±1.03 years) groups. Subjects in the intervention group received EF group training based on playing activities; subjects in the control group received no intervention. The behavior rating inventory of executive function (BRIEF) was administered to evaluate the behavioral and cognitive aspects of EF. The duration of the intervention was 6 hours per week for 9 weeks. Multivariate analysis of covariance was used to compare mean changes (before and after) in the BRIEF scores between the groups. Results: The assumptions of multivariate analysis of covariance were examined. After controlling pre-test conditions, the intervention and control groups scored significantly differently on both the metacognition (P=0.002; effect size=0.20) and behavior regulation indices (P=0.01; effect size=0.12) of BRIEF. Conclusion: Play-based therapy is effective on the metacognitive and behavioral aspects of EF in students with specific learning disabilities. Professionals can use play-based therapy rather than educational approaches in clinical practice to enhance EF skills.
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- 2017
29. A need to simplify informed consent documents in cancer clinical trials. A position paper of the ARCAD Group
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Cathy Eng, G. Decoster, H.-J. Schmoll, Al B. Benson, Annette K. Larsen, Alberto Sobrero, A. de Gramont, Charles S. Fuchs, Harry Bleiberg, Masashi Fujii, John Zalcberg, Jean-Yves Douillard, Dominique Sprumont, Roberto Labianca, Edith P. Mitchell, Ph. Rougier, and Benoist Chibaudel
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Health Knowledge, Attitudes, Practice ,medicine.medical_specialty ,good clinical practice ,media_common.quotation_subject ,education ,Legibility ,Institution Review Board ,Consent Forms ,03 medical and health sciences ,0302 clinical medicine ,Documentation ,Informed consent ,Neoplasms ,inform consent ,Humans ,Medicine ,030212 general & internal medicine ,Patient participation ,media_common ,Clinical Trials as Topic ,Medical education ,Informed Consent ,business.industry ,clinical trial ,Hematology ,ethics ,Cancérologie ,Clinical trial ,Editor's Choice ,Oncology ,030220 oncology & carcinogenesis ,Family medicine ,oncology ,Practice Guidelines as Topic ,Good clinical practice ,Special Articles ,Position paper ,Patient Participation ,business ,Autonomy ,Hématologie - Abstract
Background: In respect of the principle of autonomy and the right of self-determination, obtaining an informed consent of potential participants before their inclusion in a study is a fundamental ethical obligation. The variations in national laws, regulations, and cultures contribute to complex informed consent documents for patients participating in clinical trials. Currently, only few ethics committees seem willing to address the complexity and the length of these documents and to request investigators and sponsors to revise them in a way to make them understandable for potential participants. The purpose of this work is to focus on the written information in the informed consent documentation for drug development clinical trials and suggests (i) to distinguish between necessary and not essential information, (ii) to define the optimal format allowing the best legibility of those documents., SCOPUS: ar.j, info:eu-repo/semantics/published
- Published
- 2017
30. Effectiveness of Ginseng in Treating Erectile Dysfunction: A Review Paper
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Lee Tiong Chan and Amy Ting Shy Yee
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medicine.medical_specialty ,business.industry ,Sildenafil ,medicine.medical_treatment ,food and beverages ,Penile prosthesis ,medicine.disease ,complex mixtures ,Tadalafil ,Clinical trial ,Ginseng ,chemistry.chemical_compound ,Erectile dysfunction ,chemistry ,Vardenafil ,Diabetes mellitus ,medicine ,business ,Intensive care medicine ,medicine.drug - Abstract
Early identification and treatment of erectile dysfunction (ED) and ED related comorbidities, including hypertension, diabetes mellitus, and hyperlipidemia is crucial in improving the quality of life of men and their partners. To date, the main treatment options for ED can be divided into pharmacological and surgical interventions. These treatments include oral phosphodiesterase type 5 (PDE5) inhibitors (sildenafil, tadalafil, vardenafil), intraurethral or intracavernosal alprostadil, vacuum devices and penile prosthesis. Although considerable advances have been made, there seems to be a high dropout rate of the above treatment due to cultural restrictions and taboos. Subsequently, these patients would seek herbal dietary supplements such as ginseng as an alternative. Even though several studies have proved that ginseng is ideal in treating ED, the published trials on humans were not robust, and there is a lack of exploration of subjective outcomes. This literature review aimed to synthesize and critically appraise clinical and scientific literature on the effects of ginseng in managing ED. In total, 7 studies were included and reviewed. It was concluded that ginseng is a feasible alternative to currently available practices in treating ED due to a significant improvement in International Index of Erectile Function (IIEF)-5 scores and its safety profile. There is a higher likelihood to consider ginseng as an alternative therapy if more studies with larger-scaled clinical trials and higher standards on the safety and efficacy of ginseng are conducted in the future.
- Published
- 2021
31. A pragmatic randomized trial comparing tablet computer informed consent to traditional paper-based methods for an osteoporosis study
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Nicole C. Wright, Amy H. Warriner, Sebastian E. Sattui, Meredith L. Kilgore, Amy S. Mudano, Wilson D. Pace, Walter L. Calmbach, Phillip J. Foster, Jeffrey R. Curtis, Cora E. Lewis, Kenneth G. Saag, and Mary Elkins Melton
- Subjects
medicine.medical_specialty ,Osteoporosis ,Alternative medicine ,Article ,law.invention ,Tablet computer ,03 medical and health sciences ,0302 clinical medicine ,Randomized controlled trial ,Informed consent ,law ,medicine ,030212 general & internal medicine ,Pharmacology ,lcsh:R5-920 ,business.industry ,General Medicine ,Paper based ,16. Peace & justice ,medicine.disease ,Pragmatic clinical trials ,3. Good health ,Clinical trial ,Comprehension ,030220 oncology & carcinogenesis ,Physical therapy ,business ,lcsh:Medicine (General) - Abstract
Objective Methods to improve informed consent efficiency and effectiveness are needed for pragmatic clinical trials. We compared informed consent using a tablet computer to a paper approach to assess comprehension and satisfaction of patients and clinic staff for a future osteoporosis clinical trial. Methods Nine community-based practices identified and recruited patients to compare the informed consent processes (tablet vs. paper) in a mock osteoporosis clinical trial. The tablet informed consent included an animation summarizing the trial, complete informed consent document, and questions to assess and reinforce comprehension of the study. Participants were women age ≥55 years with ≥1 year of alendronate use. We surveyed participants to assess comprehension and satisfaction and office staff for satisfaction and perceived time demands. Results The nine practices enrolled 33 participants. There was not a significant difference in comprehension between the tablet vs. paper informed consent [mean (SD) tablet: 12.2 (1.0) vs. paper: 11.4 (1.7)]. Office staff preferred the tablet to the paper informed consent for identifying potential study participants (two-sided t-test p = 0.02) despite an increased perceived time spent to complete the tablet process [tablet: 28.3 min (SD 16.3) vs. paper: 19.0 min (SD 6.9); p = 0.08]. Conclusions Although, there were no significant differences in participant satisfaction and comprehension with the tablet informed consent compared to a paper informed consent, patients and office staff trended towards greater satisfaction with the tablet informed consent. Larger studies are needed to further evaluate the utility of electronic informed consent in pragmatic clinical trials.
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- 2016
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32. Adolescents and young adults (AYA) with cancer: a position paper from the AYA Working Group of the European Society for Medical Oncology (ESMO) and the European Society for Paediatric Oncology (SIOPE)
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Nathalie Gaspar, Emmanouil Saloustros, Andrea C. Ferrari, J. De Munter, O. Smith, Lars Hjorth, Dan Stark, W.T.A. van der Graaf, Fedro A. Peccatori, L. Soanes, J.-Y. Douillard, Lorna A Fern, K. Derwich, Giannis Mountzios, A. Blondeel, Stefan S. Bielack, Valérie Laurence, I. Bozovic-Spasojevic, and S. Jezdic
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Oncology ,Cancer Research ,medicine.medical_specialty ,Standard of care ,Adolescent ,Accrual ,Medical Oncology ,Young Adult ,Multidisciplinary approach ,Neoplasms ,Internal medicine ,Humans ,Medicine ,Young adult ,Child ,Aged ,Paediatric oncology ,business.industry ,Cancer ,medicine.disease ,humanities ,Europe ,Clinical trial ,Position paper ,business - Abstract
It is well recognised that adolescents and young adults (AYA) with cancer have inequitable access to oncology services that provide expert cancer care and consider their unique needs. Subsequently, survival gains in this patient population have improved only modestly compared with older adults and children with cancer. In 2015, the European Society for Medical Oncology (ESMO) and the European Society for Paediatric Oncology (SIOPE) established the joint Cancer in AYA Working Group in order to increase awareness among adult and paediatric oncology communities, enhance knowledge on specific issues in AYA and ultimately improve the standard of care for AYA with cancer across Europe. This manuscript reflects the position of this working group regarding current AYA cancer care, the challenges to be addressed and possible solutions. Key challenges include the lack of specific biological understanding of AYA cancers, the lack of access to specialised centres with age-appropriate multidisciplinary care and the lack of available clinical trials with novel therapeutics. Key recommendations include diversifying interprofessional cooperation in AYA care and specific measures to improve trial accrual, including centralising care where that is the best means to achieve trial accrual. This defines a common vision that can lead to improved outcomes for AYA with cancer in Europe.
- Published
- 2021
33. We need more patient and public reviews on research papers-and the resources to do so
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Dana M Lewis and Emma Doble
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Medical education ,Financial Management ,Hospital bed ,business.industry ,Perspective (graphical) ,General Medicine ,Research process ,Quality Improvement ,Clinical trial ,Work (electrical) ,Health care ,Health Resources ,Humans ,Patient Participation ,Psychology ,business ,Medical literature ,Quality of Health Care - Abstract
Historically, patients have always been considered the passive recipients of healthcare. This way of thinking affected everything from how people were cared for in a clinician’s office or hospital bed, to how they participated in clinical trials. It’s also meant that patients have previously had no role in the production or review of medical literature after research has been completed. However, this is changing, and now patients and members of the public are increasingly involved in new and meaningful ways at more steps in the research process, including as potential reviewers of medical papers. This has enormous benefits for science and healthcare. But patients and members of the public are not always provided with the relevant resources to participate effectively and efficiently, and this is something that journals need to work on. In 2014, The BMJ started soliciting patient and public (or P&P) reviews of papers.1 The role of patients and public reviewers in health research cannot be underestimated, with patients and members of the public bringing a unique and valuable perspective to research and peer review.2 However, as with traditional novice peer reviewers, patients and public reviewers …
- Published
- 2021
34. Position Paper of the European Society of Cardiology Working Group Cellular Biology of the Heart: cell-based therapies for myocardial repair and regeneration in ischemic heart disease and heart failure
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Christine L. Mummery, Cinzia Perrino, Jonathan Leor, James T. Willerson, Linda W. van Laake, Felix B. Engel, Ulf Landmesser, Rainer Schulz, Péter Ferdinandy, Rosalinda Madonna, Stefan Janssens, Sean M. Davidson, Kirsti Ytrehus, Joost P.G. Sluijter, Sandrine Lecour, Derek J. Hausenloy, Thomas Eschenhagen, Madonna, Rosalinda, Van Laake, Linda W, Davidson, Sean M, Engel, Felix B, Hausenloy, Derek J, Lecour, Sandrine, Leor, Jonathan, Perrino, Cinzia, Schulz, Rainer, Ytrehus, Kirsti, Landmesser, Ulf, Mummery, Christine L, Janssens, Stefan, Willerson, Jame, Eschenhagen, Thoma, Ferdinandy, Péter, and Sluijter, Joost P. G.
- Subjects
0301 basic medicine ,medicine.medical_specialty ,Cell- and Tissue-Based Therapy ,Myocardial Ischemia ,Context (language use) ,Review ,Disease ,Cell therapy ,03 medical and health sciences ,Medical ,Heart Regeneration ,Internal medicine ,Journal Article ,medicine ,Humans ,Regeneration ,Cell-based therapy ,Heart Failure ,Heart repair ,Ischaemic heart disease ,Cell Tracking ,Clinical Trials as Topic ,Data Accuracy ,Ethics, Medical ,Heart ,Patient Safety ,Patient Selection ,Stem Cell Transplantation ,Stroke Volume ,Treatment Outcome ,cardiovascular diseases ,Ethics ,Medicine(all) ,business.industry ,Regeneration (biology) ,Stroke volume ,medicine.disease ,Cell biology ,Clinical trial ,030104 developmental biology ,Current Opinion ,Heart failure ,Cardiology ,Position paper ,Cardiology and Cardiovascular Medicine ,business - Abstract
Despite improvements in modern cardiovascular therapy, the morbidity and mortality of ischaemic heart disease (IHD) and heart failure (HF) remain significant in Europe and worldwide. Patients with IHD may benefit from therapies that would accelerate natural processes of postnatal collateral vessel formation and/or muscle regeneration. Here, we discuss the use of cells in the context of heart repair, and the most relevant results and current limitations from clinical trials using cell-based therapies to treat IHD and HF. We identify and discuss promising potential new therapeutic strategies that include ex vivo cell-mediated gene therapy, the use of biomaterials and cell-free therapies aimed at increasing the success rates of therapy for IHD and HF. The overall aim of this Position Paper of the ESC Working Group Cellular Biology of the Heart is to provide recommendations on how to improve the therapeutic application of cell-based therapies for cardiac regeneration and repair.
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- 2016
35. A bibliometric study of the top cited papers related to periodontal regeneration
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Andy Wai Kan Yeung, W. Keung Leung, and H. N. Liu
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medicine.medical_specialty ,Impact factor ,business.industry ,Study Type ,Guided Periodontal Tissue Regeneration ,Clinical trial ,Systematic review ,Bibliometrics ,Family medicine ,Medicine ,Animals ,Regeneration ,Journal Impact Factor ,business ,General Dentistry - Abstract
This report identifies the top cited papers in the field of periodontal regeneration since inception of the concept. Using the H-classics approach, 132 papers published between 1970 and 2012 were identified, with 230.0 ± 175.6 (mean ± SD) citations and 10.4 ± 11.5 citations/year. There were 46 clinical reports, 28 animal studies, 23 in vitro studies, 30 reviews, 3 systematic reviews, and 2 combined animal and in vitro studies. Analysis of covariance showed that institution number (≥3, P = 0.011), journal impact factor at publication (>3.0, P = 0.001) and study type (in vitro/reviews vs. clinical trials/animal studies, P = 0.024) were significantly associated with citations/year. This study has characterized the most influential literature in the field of periodontal regeneration and serves as a quick reference resource.
- Published
- 2021
36. When is lack of scientific integrity a reason for retracting a paper? A case study
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Jess G. Fiedorowicz, Albert F.G. Leentjens, James L. Levenson, RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience, Psychiatrie & Neuropsychologie, and MUMC+: MA Med Staf Spec Psychiatrie (9)
- Subjects
Randomized control trial ,Blinding ,Scientific Misconduct ,law.invention ,03 medical and health sciences ,Misconduct ,Retraction of Publication as Topic ,0302 clinical medicine ,Randomized controlled trial ,law ,Intervention (counseling) ,Humans ,030212 general & internal medicine ,Scientific misconduct ,Ethical reflection ,Randomized Controlled Trials as Topic ,Publishing ,Medical education ,Homeopathy ,Retraction ,Clinical trial ,Psychiatry and Mental health ,Clinical Psychology ,Research Design ,Spite ,Periodicals as Topic ,Psychology ,030217 neurology & neurosurgery ,CLINICAL-TRIALS - Abstract
Objective The journal received a request to retract a paper reporting the results of a triple-blind randomized placebo-controlled trial. The present and immmediate past editors expand on the journal's decision not to retract this paper in spite of undisputable evidence of scientific misconduct on behalf of one of the investigators. Methods The editors present an ethical reflection on the request to retract this randomized clinical trial with consideration of relevant guidelines from the Committee on Publication Ethics (COPE) and the International Committee of Medical Journal Editors (ICMJE) applied to the unique contextual issues of this case. Results In this case, scientific misconduct by a blinded provider of a homeopathy intervention attempted to undermine the study blind. As part of the study, the integrity of the study blind was assessed. Neither participants nor homeopaths were able to identify whether the participant was assigned to homeopathic medicine or placebo. Central to the decision not to retract the paper was the fact that the rigorous scientific design provided evidence that the outcome of the study was not affected by the misconduct. The misconduct itself was thought to be insufficient reason to retract the paper. Conclusion Retracting a paper of which the outcome is still valid was in itself considered unethical, as it takes away the opportunity to benefit from its results, rendering the whole study useless. In such cases, scientific misconduct is better handled through other professional channels.
- Published
- 2021
37. Research Paper: Clomiphene Citrate to Inducte Ovulation in Females With Unexplained Infertility: A Randomized, Controlled, Clinical Trial
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Mona Abdel-Gawad, Ahmed E.H. Elbohoty, Nayrouz Gozaf, Amr A. Nadim, and Nashwa M. Ezzat Eman M.G. El-Said
- Subjects
medicine.medical_specialty ,business.industry ,Obstetrics ,media_common.quotation_subject ,Clinical pregnancy ,Confidence interval ,Clinical trial ,Primary outcome ,Relative risk ,Number needed to treat ,Medicine ,business ,Ovulation ,media_common ,Unexplained infertility - Abstract
Objectives: The current study aimed at comparing the efficacy of Clomiphene Citrate (CC) for the expectant management of unexplained infertility in females over 3 successive cycles. Materials: The present randomized, controlled, clinical trial was carried out at Ain Shams University Maternity Hospital. Females with unexplained infertility for at least 12 months of unprotected regular marital life were enrolled. Eligible females were randomly assigned into one of the 2 following groups: group 1 received 100 mg CC once a day for 5 days, and group 2 was expectantly followed up without induction of ovulation. The primary outcome was the clinical pregnancy rate. Results: A total of 113 females were enrolled in the current trial. The mean age of the subjects was 25.3±3.1 years; ranged 20 to 33. The clinical pregnancy rate was slightly, but significantly, higher in CC group compared with the controls; both per case (7/57 vs. 4/56, 12.3% vs. 7.1%, respectively; P=0.357; Relative Risk (RR)=1.72; 95% Confidence Interval (CI)=0.53, 5.55; Number Needed to Treat (NNT)=19) and per cycle (7/163 vs. 4/160, 4.3% vs. 2.5%, respectively; P=0.374; RR=1.72; 95% CI=0.51, 5.75; NNT=56). Discussion: CC seems to be not effective in improving clinical pregnancy outcome compared with observation alone in females with unexplained infertility.
- Published
- 2019
38. Moving forward from drug-centred to patient-centred research: A white paper initiated by EORTC and developed together with the BioMed Alliance members
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Guy Brusselle, Elsa Mateus, Barbara Casadei, Colm O'Morain, Rik Lories, Denis Lacombe, and Kate Hill
- Subjects
Pulmonary and Respiratory Medicine ,Medical education ,business.industry ,Process (engineering) ,Clinical trial ,Europe ,03 medical and health sciences ,0302 clinical medicine ,Alliance ,White paper ,Phenotype ,030228 respiratory system ,Research Design ,Drug Design ,Patient-Centered Care ,Medicine ,Humans ,030212 general & internal medicine ,Registries ,Precision Medicine ,business ,Drug Approval ,Patient centred ,Biomarkers ,Randomized Controlled Trials as Topic - Abstract
This paper discusses how to restructure the process of clinical research to maximise the potential of precision medicinehttp://ow.ly/1ZCc30nuw2a
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- 2018
39. Surgical quality assurance in head and neck cancer trials: an EORTC Head and Neck Cancer Group position paper based on the EORTC 1420 'Best of' and 24954 'larynx preservation' study
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Christian Simon, C. René Leemans, Piero Nicolai, Petri Koivunen, Marc Hamoir, Dietmar Thurnher, Andreas Dietz, Philippe Ceruse, Guy Andry, Vincent Grégoire, Keith K.A. Hunter, Lisa Licitra, Hisham Mehanna, Carmela Caballero, Giuseppe Spriano, Jean-Pascal Machiels, UCL - SSS/IREC/MIRO - Pôle d'imagerie moléculaire, radiothérapie et oncologie, UCL - (SLuc) Centre du cancer, and UCL - (SLuc) Unité d'oncologie médicale
- Subjects
Cancer Research ,medicine.medical_specialty ,Clinical trials ,EORTC ,Head and neck cancer ,Quality assurance ,Surgery ,Quality management ,Quality Assurance, Health Care ,medicine.medical_treatment ,03 medical and health sciences ,0302 clinical medicine ,Surgical oncology ,Medicine ,Humans ,Medical physics ,030223 otorhinolaryngology ,Protocol (science) ,business.industry ,medicine.disease ,Quality Improvement ,Radiation therapy ,Clinical trial ,Oncology ,Head and Neck Neoplasms ,030220 oncology & carcinogenesis ,Position paper ,business - Abstract
Quality improvement of care for patients with head and neck cancer remains a constant objective for the multidisciplinary team of physicians managing these patients. The purpose of quality assurance (QA) for head and neck surgical oncology and surgical trials however differs. While QA for the general head and neck patient aims to improve global outcome through structural changes of health-care systems, QA for surgical trials pursues the goal to help providing meaningful results from a clinical trial through the definition of structure, process and outcome measures within the trial. Establishing a QA program for surgical trials is challenging largely due to the variation in the execution of surgical techniques. Within this article, we describe the surgical QA program, which was developed for the phase III European Organisation for Research and Treatment of Cancer (EORTC) 1420 study, a trial assessing swallowing function after transoral surgery compared with radiation therapy. We propose based on our experience to further develop surgical QA for surgical clinical trials by introducing two separate components, one adaptable and one non-adaptable. The adaptable is tailored to the scientific question and specific procedure; the non-adaptable consists of minimal structural requirements of the clinical unit to participate in surgical trials at EORTC as well as guidelines and incentives for protocol adherence based on our experience in EORTC 24954. Finally, we strongly believe that surgical QA designed for clinical trials may serve as a basis for the development of QA surgical guidelines in clinical practice.
- Published
- 2018
40. Nutritional Support in Cancer Patients: A Position Paper from the Italian Society of Medical Oncology (AIOM) and the Italian Society of Artificial Nutrition and Metabolism (SINPE)
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Paolo Pedrazzoli, Giordano D. Beretta, Mariateresa Nardi, Agostino Paccagnella, Alessandro Laviano, Carmine Pinto, Emanuele Cereda, Cecilia Gavazzi, Vittorina Zagonel, and Riccardo Caccialanza
- Subjects
0301 basic medicine ,medicine.medical_specialty ,Pediatrics ,Palliative care ,Alternative medicine ,Review ,Clinical nutrition ,03 medical and health sciences ,0302 clinical medicine ,Quality of life (healthcare) ,Medicine ,Nutritional support ,Intensive care medicine ,Cancer ,030109 nutrition & dietetics ,business.industry ,Malnutrition ,medicine.disease ,Clinical trial ,Oncology ,030220 oncology & carcinogenesis ,Position paper ,business - Abstract
Malnutrition is a frequent problem in cancer patients, which leads to prolonged hospitalization, a higher degree of treatment-related toxicity, reduced response to cancer treatment, impaired quality of life and a worse overall prognosis. The attitude towards this issue varies considerably and many malnourished patients receive inadequate nutritional support. We reviewed available data present in the literature, together with the guidelines issued by scientific societies and health authorities, on the nutritional management of patients with cancer, in order to make suitable and concise practical recommendations for appropriate nutritional support in this patient population. Evidence from the literature suggests that nutritional screening should be performed using validated tools (the Nutritional Risk Screening 2002 [NRS 2002], the Malnutrition Universal Screening Tool [MUST], the Malnutrition Screening Tool [MST] and the Mini Nutritional Assessment [MNA]), both at diagnosis and at regular time points during the course of disease according to tumor type, stage and treatment. Patients at nutritional risk should be promptly referred for comprehensive nutritional assessment and support to clinical nutrition services or medical personnel with documented skills in clinical nutrition, specifically for cancer patients. Nutritional intervention should be actively managed and targeted for each patient; it should comprise personalized dietary counseling and/or artificial nutrition according to spontaneous food intake, tolerance and effectiveness. Nutritional support may be integrated into palliative care programs. “Alternative hypocaloric anti-cancer diets” (e.g. macrobiotic or vegan diets) should not be recommended as they may worsen nutritional status. Well-designed clinical trials are needed to further our knowledge of the nutritional support required in different care settings for cancer patients.
- Published
- 2016
41. HIV cure research in the time of COVID-19 - Antiretroviral therapy treatment interruption trials: A discussion paper
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Simon Collins, Steven G. Deeks, Ole Schmeltz Søgaard, Jerome Amir Singh, D Kelly, John Frater, Marina Caskey, L Vanderkerckhove, Sarah Fidler, and Sharon R Lewin
- Subjects
0301 basic medicine ,medicine.medical_specialty ,Coronavirus disease 2019 (COVID-19) ,Epidemiology ,Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) ,Immunology ,Human immunodeficiency virus (HIV) ,Context (language use) ,medicine.disease_cause ,Microbiology ,03 medical and health sciences ,Viewpoint ,0302 clinical medicine ,Virology ,Pandemic ,medicine ,030212 general & internal medicine ,Intensive care medicine ,business.industry ,Analytical treatment interruption (ATI) COVID-19 ,SARS-CoV-2 ,HIV cure ,Public Health, Environmental and Occupational Health ,Antiretroviral therapy ,QR1-502 ,Clinical trial ,030104 developmental biology ,Infectious Diseases ,Treatment interruption ,Public aspects of medicine ,RA1-1270 ,business - Abstract
This discussion paper addresses the safety of HIV cure studies, particularly those involving stopping antiretroviral therapy, known as an analytic treatment interruption (ATI) in the context of the SARS-CoV-2 pandemic. More than 30 studies listed on ClinicalTrials.gov include an ATI and many others were planned to begin over the next 12 months but most were halted due to the COVID-19 pandemic. We consider the ethics, risks and practical considerations to be taken into account before re-opening HIV cure clinical trials, noting the specific risks of ATI in the context of circulating SARS-CoV-2.
- Published
- 2021
42. Harms in Systematic Reviews Paper 2: Methods used to assess harms are neglected in systematic reviews of gabapentin
- Author
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Tianjing Li, Thanitsara Rittiphairoj, Riaz Qureshi, Eliseo Guallar, Evan Mayo-Wilson, and Mara McAdams-DeMarco
- Subjects
Gabapentin ,Epidemiology ,Applied psychology ,Guideline ,Clinical trial ,Harm ,Systematic review ,Meta-analysis ,medicine ,Humans ,Psychology ,medicine.drug ,Systematic Reviews as Topic - Abstract
Objective : We compared methods used with current recommendations for synthesizing harms in systematic reviews and meta-analyses (SRMAs) of gabapentin. Study Design & Setting : We followed recommended systematic review practices. We selected reliable SRMAs of gabapentin (i.e., met a pre-defined list of methodological criteria) that assessed at least one harm. We extracted and compared methods in four areas: pre-specification, searching, analysis, and reporting. Whereas our focus in this paper is on the methods used, Part 2 examines the results for harms across reviews. Results : We screened 4320 records and identified 157 SRMAs of gabapentin, 70 of which were reliable. Most reliable reviews (51/70; 73%) reported following a general guideline for SRMA conduct or reporting, but none reported following recommendations specifically for synthesizing harms. Across all domains assessed, review methods were designed to address questions of benefit and rarely included the additional methods that are recommended for evaluating harms. Conclusion : Approaches to assessing harms in SRMAs we examined are tokenistic and unlikely to produce valid summaries of harms to guide decisions. A paradigm shift is needed. At a minimal, reviewers should describe any limitations to their assessment of harms and provide clearer descriptions of methods for synthesizing harms.
- Published
- 2021
43. Reporting transparency and completeness in trials: Paper 1: Introduction - Better reporting for disruptive clinical trials using routinely collected data
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Brett D. Thombs, Lars G. Hemkens, and Edmund Juszczak
- Subjects
Clinical trial ,Epidemiology ,business.industry ,Research Design ,Completeness (order theory) ,Medicine ,Humans ,Accounting ,business ,Transparency (behavior) ,Checklist ,Routinely Collected Health Data - Published
- 2021
44. ANMCO POSITION PAPER: cardio-oncology in the COVID era (CO and CO)
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Furio Colivicchi, Paolo Trambaiolo, Luigi Tarantini, Stefania Angela Di Fusco, Fabiana Lucà, Michele Massimo Gulizia, Vincenzo Quagliariello, Giuseppina Gallucci, Stefano Oliva, Iris Parrini, Nicola Maurea, Maria Laura Canale, Fabio Turazza, Giulia Russo, Chiara Lestuzzi, Irma Bisceglia, and Domenico Gabrielli
- Subjects
medicine.medical_specialty ,Telemedicine ,business.industry ,SARS-CoV-2 ,Risk of infection ,COVID-19 ,Disease ,Telehealth ,Articles ,Cardiovascular disease ,Clinical trial ,Cardio-oncology ,Cardiovascular imaging ,Pandemic ,Health care ,Global health ,Medicine ,AcademicSubjects/MED00200 ,Cardiology and Cardiovascular Medicine ,business ,Intensive care medicine ,Biomarkers ,Cancer - Abstract
The COVID-19 pandemic and its impact on patients with cancer and cardiovascular disease have confirmed the particular vulnerability of these populations. Indeed, not only a higher risk of contracting the infection has been reported but also an increased occurrence of a more severe course and unfavourable outcome. Beyond the direct consequences of COVID-19 infection, the pandemic has an enormous impact on global health systems. Screening programmes and non-urgent tests have been postponed; clinical trials have suffered a setback. Similarly, in the area of cardiology care, a significant decline in STEMI accesses and an increase in cases of late presenting heart attacks with increased mortality and complication rates have been reported. Health care systems must therefore get ready to tackle the ‘rebound effect’ that will likely show a relative increase in the short- and medium-term incidence of diseases such as heart failure, myocardial infarction, arrhythmias, and cardio- and cerebrovascular complications. Scientific societies are taking action to provide general guidance and recommendations aimed at mitigating the unfavourable outcomes of this pandemic emergency. Cardio-oncology, as an emerging discipline, is more flexible in modulating care pathways and represents a beacon of innovation in the development of multi-specialty patient management. In the era of the COVID-19 pandemic, cardio-oncology has rapidly modified its clinical care pathways and implemented flexible monitoring protocols that include targeted use of cardiac imaging, increased use of biomarkers, and telemedicine systems. The goal of these strategic adjustments is to minimize the risk of infection for providers and patients while maintaining standards of care for the treatment of oncologic and cardiovascular diseases. The aim of this document is to evaluate the impact of the pandemic on the management of cardio-oncologic patients with the-state-of-the-art knowledge about severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and coronavirus disease (COVID-19) in order to optimize medical strategies during and after the pandemic.
- Published
- 2021
45. Evolving Intersection Between Inherited Cancer Genetics and Therapeutic Clinical Trials in Prostate Cancer: A White Paper From the Germline Genetics Working Group of the Prostate Cancer Clinical Trials Consortium
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Rana R. McKay, Himisha Beltran, Emmanuel S. Antonarakis, Akash Patnaik, Charles J. Ryan, Channing J. Paller, Tomasz M. Beer, Maria I. Carlo, Mary-Ellen Taplin, Edward M. Schaeffer, Daniel J. George, Noah D. Kauff, Joshi J. Alumkal, Walter M. Stadler, Alicia K. Morgans, Celestia S. Higano, Veda N. Giri, Elisabeth I. Heath, Heather H. Cheng, Jacob Vinson, and Wassim Abida
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0301 basic medicine ,Genetics ,Cancer Research ,education.field_of_study ,medicine.diagnostic_test ,business.industry ,Population ,medicine.disease ,Germline ,Article ,Clinical trial ,03 medical and health sciences ,Prostate cancer ,030104 developmental biology ,0302 clinical medicine ,White paper ,Oncology ,030220 oncology & carcinogenesis ,Cancer genetics ,Advanced disease ,medicine ,business ,education ,Genetic testing - Abstract
Purpose Advances in germline genetics, and related therapeutic opportunities, present new opportunities and challenges in prostate cancer. The Prostate Cancer Clinical Trials Consortium Germline Genetics Working Group was established to address genetic testing for men with prostate cancer, especially those with advanced disease undergoing testing for treatment-related objectives and clinical trials. Methods The Prostate Cancer Clinical Trials Consortium Germline Genetics Working Group met monthly to discuss the current state of genetic testing of men with prostate cancer for therapeutic or clinical trial purposes. We assessed current institutional practices, developed a framework to address unique challenges in this population, and identified areas of future research. Results Genetic testing practices in men with prostate cancer vary across institutions; however, there were several areas of agreement. The group recognized the clinical benefits of expanding germline genetic testing, beyond cancer risk assessment, for the goal of treatment selection or clinical trial eligibility determination. Genetic testing for treatment selection should ensure patients receive appropriate pretest education and consent and occur under auspices of a research study whenever feasible. Providers offering genetic testing should be able to interpret results and recommend post-test genetic counseling for patients. When performing tumor (somatic) genomic profiling, providers should discuss the potential for uncovering germline mutations and recommend appropriate genetic counseling. In addition, family members may benefit from cascade testing and early cancer screening and prevention strategies. Conclusion As germline genetic testing is incorporated into practice, further development is needed in establishing prompt testing for time-sensitive treatment decisions, integrating cascade testing for family, ensuring equitable access to testing, and elucidating the role of less-characterized germline DNA damage repair genes, individual gene-level biologic consequences, and treatment response prediction in advanced disease.
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- 2018
46. Endpoints and design of clinical trials in patients with decompensated cirrhosis: Position paper of the LiverHope Consortium
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E. Palacio, Koos de Wit, Daniela Campion, Salvatore Piano, Elisa Pose, T. Lanzillotti, Carlos Jiménez, Sara Montagnese, Patrick S. Kamath, Rajeshwar P. Mookerjee, Miquel Gómez i Serra, Ruben Hernaez, M. Aban, Marta Carol, G. Nicolao, Claire Francoz, Giacomo Zaccherini, Hugh Watson, Isabel Graupera, M.T. Chiappa, Jonel Trebicka, Macarena Simón-Talero, Frank Erhard Uschner, Cristina Solé, Alejandro Forner, Ulrich Beuers, F. Durand, Paolo Caraceni, Victor Vargas, Marta Cervera, Carlo Alessandria, Adrià Juanola, V. Esnault, Jeltje Helder, Marko Korenjak, Olivier Roux, Laura Napoleone, Ferran Torres, S. Graf-Dirmeier, Judit Pich, Ann T. Ma, Maria Martha Bernardi, M. Pérez-Guasch, Núria Fabrellas, Pere Ginès, Maricela Quintana López, Elsa Solà, Emma Avitabile, Aleksander Krag, Juan G. Abraldes, Gastroenterology and Hepatology, Graduate School, Tytgat Institute for Liver and Intestinal Research, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Elsa Solà, Elisa Pose, Daniela Campion, Salvatore Piano, Olivier Roux, Macarena Simon-Talero, Frank Uschner, Koos de Wit, Giacomo Zaccherini, Carlo Alessandria, Ulrich Beuers, Paolo Caraceni, Claire Francoz, Rajeshwar P. Mookerjee, Jonel Trebicka, Victor Vargas, Miquel Serra, Ferran Torres, Sara Montagnese, Aleksander Krag, Ruben Hernaez, Marko Korenjak, Hugh Watson, Juan G. Abraldes, Patrick S. Kamath, Pere Ginès, LiverHope Consortium Investigators, Institut Català de la Salut, [Solà E, Pose E] Liver Unit, Hospital Clínic De Barcelona, Barcelona, School of Medicine and Health Sciences University of Barcelona, Spain. Institut d´Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEReHD), Barcelona, Spain. [Campion D] Division of Gastroenterology and Hepatology, Città della Salute e della Scienza Hospital, University of Turin, Turin, Italy. [Piano S] Unit of Internal Medicine and Hepatology (UIMH), Department of Medicine - DIMED, University of Padova, Padova, Italy. [Roux O] Hepatology and Liver Intensive Care Unit, Hospital Beaujon, APHP, Clichy, France. [Simon-Talero M] Unitat del Fetge, Vall d'Hebron Hospital Universitari, Barcelona, Spain. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. Universitat Autònoma de Barcelona, Bellaterra, Spain. Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBEReHD), Barcelona, Spain, and Vall d'Hebron Barcelona Hospital Campus
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Liver Cirrhosis ,0301 basic medicine ,Cirrhosis ,administración de los servicios de salud::gestión de la atención al paciente::tratamiento de las enfermedades [ATENCIÓN DE SALUD] ,enfermedades del sistema digestivo::enfermedades hepáticas::cirrosis hepática [ENFERMEDADES] ,Digestive System Diseases::Liver Diseases::Liver Cirrhosis [DISEASES] ,Trasplantament hepàtic ,Disease ,Severity of Illness Index ,0302 clinical medicine ,Clinical trials ,Quality of life ,ACLF ,Ascites ,Secondary Prevention ,Liver transplant ,Otros calificadores::/terapia [Otros calificadores] ,Hepatic encephalopathy ,Clinical Trials as Topic ,education.field_of_study ,Disease Management ,3. Good health ,Clinical trial ,Europe ,Natural history ,Hepatic cirrhosis ,Research Design ,Qualitat de vida ,Ascite ,Disease Progression ,030211 gastroenterology & hepatology ,AKI ,ascites ,cirrhosis ,clinical trials ,endpoints ,hepatic encephalopathy ,hyponatremia ,infections ,liver transplant ,quality of life ,medicine.symptom ,Infection ,Hyponatremia ,técnicas de investigación::métodos::diseño de la investigación [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS] ,medicine.medical_specialty ,Consensus ,Cirrosi hepàtica ,Endpoint Determination ,Population ,Infections ,03 medical and health sciences ,Investigative Techniques::Methods::Research Design [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT] ,Hypertension, Portal ,medicine ,Humans ,Intensive care medicine ,education ,Health Services Administration::Patient Care Management::Disease Management [HEALTH CARE] ,Cirrosi hepàtica - Tractament ,Cirrhosi ,Hepatology ,business.industry ,Other subheadings::/therapy [Other subheadings] ,Endpoint ,medicine.disease ,Assaigs clínics - Disseny ,030104 developmental biology ,Etiology ,Endpoints ,business ,Hepatic transplantation - Abstract
Clinical trials; Liver transplant; Quality of life Ensayos clínicos; Trasplante de hígado; Calidad de vida Assaigs clínics; Trasplantament de fetge; Qualitat de vida Management of decompensated cirrhosis is currently geared towards the treatment of complications once they occur. To date there is no established disease-modifying therapy aimed at halting progression of the disease and preventing the development of complications in patients with decompensated cirrhosis. The design of clinical trials to investigate new therapies for patients with decompensated cirrhosis is complex. The population of patients with decompensated cirrhosis is heterogeneous (i.e., different etiologies, comorbidities and disease severity), leading to the inclusion of diverse populations in clinical trials. In addition, primary endpoints selected for trials that include patients with decompensated cirrhosis are not homogeneous and at times may not be appropriate. This leads to difficulties in comparing results obtained from different trials. Against this background, the LiverHope Consortium organized a meeting of experts, the goal of which was to develop recommendations for the design of clinical trials and to define appropriate endpoints, both for trials aimed at modifying the natural history and preventing progression of decompensated cirrhosis, as well as for trials aimed at managing the individual complications of cirrhosis.
- Published
- 2021
47. Allergen exposure chambers: harmonizing current concepts and projecting the needs for the future - an EAACI Position Paper
- Author
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Friedrich Horak, P. Zieglmayer, Robert L. Jacobs, Lars Jacobsen, Moises A. Calderon, D. Larenas-Linnemann, J. Yang, T. Zuberbier, Oliver Pfaar, Torben Sigsgaard, S. Kaul, Hendrik Nolte, Jakob Hjort Bønløkke, Mark Larché, Cynthia G. Rather, K. C. Bergmann, L. Peoples, Ralph Mösges, S. Thaarup, Ronald L. Rabin, Anne K. Ellis, Philippe Devillier, E. Angjeli, F. De Blay, Charles P. Andrews, Pascal Demoly, Piyush Patel, R. Gerth van Wijk, Anne-Marie Salapatek, Jens M. Hohlfeld, Marek Jutel, Publica, CHU Strasbourg, Mitochondrie, stress oxydant et protection musculaire (MSP), Université de Strasbourg (UNISTRA), Hôpital Foch [Suresnes], Laboratoire de recherche sur les mécanismes moléculaires et pharmacologiques de l’obstruction bronchique (LOBIP), Université de Versailles Saint-Quentin-en-Yvelines (UVSQ), Département pneumologie et addictologie [Montpellier], Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier)-Hôpital Arnaud de Villeneuve, Institut Pierre Louis d'Epidémiologie et de Santé Publique (iPLESP), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM), and Internal Medicine
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0301 basic medicine ,medicine.medical_specialty ,Standardization ,Clinical immunology ,Immunology ,education ,allergic ,Marketing authorization ,Unmet needs ,03 medical and health sciences ,0302 clinical medicine ,rhinitis ,Hypersensitivity ,Immunology and Allergy ,Medicine ,Humans ,Medical physics ,allergen exposure chambers ,[SDV.IMM.ALL]Life Sciences [q-bio]/Immunology/Allergology ,validation ,Inhalation Exposure ,clinical trials ,Task force ,business.industry ,Health Policy ,Reproducibility of Results ,Allergens ,respiratory system ,Environment, Controlled ,3. Good health ,Clinical trial ,030104 developmental biology ,030228 respiratory system ,Desensitization, Immunologic ,Position paper ,ALLERGEN EXPOSURE ,business - Abstract
BACKGOUND: Allergen exposure chambers (AEC) are clinical facilities allowing for controlled exposure of subjects to allergens in an enclosed environment. AEC have contributed towards characterizing the pathophysiology of respiratory allergic diseases and the pharmacological properties of new therapies. In addition, they are complementary to and offer some advantages over traditional multi-centre field trials for evaluation of novel therapeutics. To date, AEC studies conducted have been monocentric and have followed protocols unique to each center. Because there are technical differences among AEC, it may be necessary to define parameters to standardize the AEC so that studies may be extrapolated for driving basic immunological research and for marketing authorization purposes by regulatory authorities.METHODS: For this task force initiative of the European Academy of Allergy and Clinical Immunology (EAACI) experts from academia and regulatory agencies met with chamber operators to list technical, clinical and regulatory unmet needs as well as the prerequisites for clinical validation.RESULTS: The latter covered the validation process, standardization of challenges and outcomes, intra- and inter-chamber variability and reproducibility, in addition to comparability with field trials and specifics of paediatric trials and regulatory issues.CONCLUSION: This EAACI Position Paper aims to harmonize current concepts in AEC and to project unmet needs with the intent to enhance progress towards use of these facilities in determining safety and efficacy of new therapeutics in the future. This article is protected by copyright. All rights reserved.
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- 2017
48. GCP-compliant digital archiving of paper-based patient records of clinical trial subjects: a key issues paper
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Sebastian C Semler, Mathias Freudigmann, Gabriele Schwarz, Guido Scharf, Christian D Kohl, Paul Schmücker, and Insa Bruns
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business.industry ,media_common.quotation_subject ,General Medicine ,Certification ,Paper based ,medicine.disease ,Key issues ,Clinical trial ,Good clinical practice ,Medicine ,Quality (business) ,Source document ,Medical emergency ,business ,Digitization ,media_common - Abstract
Many healthcare providers digitize their paper-based patient records from routine care, destroying originals (replacing scanning). Current regulations are unclear as to which paper-based patient records may be destroyed after digitization if the respective patients participate in clinical trials. GCP-compliant destruction is possible if both sponsors and authorities recognize the digital copies as source documents. Recognition should be based on digitized paper-based patient records complying with the requirements for certified copies defined in ‘Note for Guidance – CPMP/ICH/135/95’. This paper describes principles by which digitized patient records can be recognized as GCP-compliant certified copies, allowing the paper-based originals to be destroyed. A prerequisite is written proof that the digitization process implemented is controlled and its outcome quality is permanently monitored.
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- 2013
49. ANMCO Position Paper: direct oral anticoagulants for stroke prevention in atrial fibrillation: clinical scenarios and future perspectives
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Iolanda Enea, Giuseppe Di Tano, Michele Massimo Gulizia, Andrea Di Lenarda, Luigi Moschini, Federico Nardi, Stefania Angela Di Fusco, Maurizio Giuseppe Abrignani, Giovanna Geraci, Furio Colivicchi, Paolo Verdecchia, and Carmine Riccio
- Subjects
medicine.medical_specialty ,030204 cardiovascular system & hematology ,Dabigatran ,03 medical and health sciences ,chemistry.chemical_compound ,Anticoagulation ,0302 clinical medicine ,Rivaroxaban ,Edoxaban ,medicine ,Apixaban ,030212 general & internal medicine ,Intensive care medicine ,business.industry ,Atrial fibrillation ,Articles ,medicine.disease ,Clinical trial ,chemistry ,Position paper ,Observational study ,Medical emergency ,Cardiology and Cardiovascular Medicine ,business ,medicine.drug - Abstract
It is now 4 years since the introduction of the new direct oral anticoagulants into clinical practice. Therefore, the Italian Association of Hospital Cardiologists (ANMCO) has deemed necessary to update the previous position paper on the prevention of thrombo-embolic complications in patients with non-valvular atrial fibrillation, which was published in 2013. All available scientific evidence has been reviewed, focusing on data derived from both clinical trials and observational registries. In addition, all issues relevant to the practical clinical management of oral anticoagulation with the new direct inhibitors have been considered. Specific clinical pathways for optimal use of oral anticoagulation with the new directly acting agents are also developed and proposed for clinical implementation. Special attention is finally paid to the development of clinical algorithms for medium and long-term follow-up of patients treated with new oral direct anticoagulants.
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- 2017
50. Behavioral features in Prader-Willi syndrome (PWS) : consensus paper from the International PWS Clinical Trial Consortium
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Theresa V. Strong, Lauren Schwartz, Elizabeth Roof, Assumpta Caixàs, Kate Woodcock, Louise Gallagher, Stewart L. Einfeld, Elisabeth M. Dykens, Parisa Salehi, Bonnie P. Taylor, Anastasia Dimitropoulos, Lauren J. Rice, Anthony J. Holland, Jessica Duis, Schwartz, Lauren [0000-0002-4022-1196], and Apollo - University of Cambridge Repository
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Temper outbursts ,congenital, hereditary, and neonatal diseases and abnormalities ,Consensus ,Cognitive Neuroscience ,Neurosciences. Biological psychiatry. Neuropsychiatry ,Review ,Hyperphagia ,Anxiety ,Patient advocacy ,Pathology and Forensic Medicine ,03 medical and health sciences ,0302 clinical medicine ,Quality of life (healthcare) ,Patient vignettes ,Social cognition ,Intervention (counseling) ,medicine ,Humans ,030304 developmental biology ,0303 health sciences ,Behavior ,Obsessive-compulsive ,Neuropsychology ,nutritional and metabolic diseases ,nervous system diseases ,Clinical trial ,Treatment study ,Rigidity ,Pediatrics, Perinatology and Child Health ,Quality of Life ,Neurology (clinical) ,medicine.symptom ,Prader-Willi syndrome ,Psychology ,030217 neurology & neurosurgery ,Obsessive–compulsive ,RC321-571 ,Clinical psychology - Abstract
Funder: Foundation for Prader-Willi Research; doi: http://dx.doi.org/10.13039/100002889, Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder associated with a characteristic behavioral phenotype that includes severe hyperphagia and a variety of other behavioral challenges such as temper outbursts and anxiety. These behaviors have a significant and dramatic impact on the daily functioning and quality of life for the person with PWS and their families. To date, effective therapies addressing these behavioral challenges have proven elusive, but several potential treatments are on the horizon. However, a limiting factor for treatment studies in PWS is the lack of consensus in the field regarding how to best define and measure the complex and interrelated behavioral features of this syndrome. The International PWS Clinical Trials Consortium (PWS-CTC, www.pwsctc.org) includes expert PWS scientists, clinicians, and patient advocacy organization representatives focused on facilitating clinical trials in this rare disease. To address the above gap in the field, members of the PWS-CTC “Behavior Outcomes Working Group” sought to develop a unified understanding of the key behavioral features in PWS and build a consensus regarding their definition and description. The primary focus of this paper is to present consensus definitions and descriptions of key phenotypic PWS behaviors including hyperphagia, temper outbursts, anxiety, obsessive–compulsive behaviors, rigidity, and social cognition deficits. Patient vignettes are provided to illustrate the interrelatedness and impact of these behaviors. We also review some available assessment tools as well as new instruments in development which may be useful in measuring these behavioral features in PWS.
- Published
- 2021
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