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Purpose: In cluster headache, patients often describe a ramp up period with initially milder headache and then build up to a persistent and severe pattern. It is possible that within the early ramp up, patients may be more treatment responsive. We propose to study if response to therapy for prevention of cluster headache is dependent on timing from onset of cluster cycle. Methods: We will conduct a prospective observational cohort study, where we examine the relationship between timing of start of preventive medication and likelihood of response to treatment. We will look at individuals exposed within 10 days of onset of cluster cycle, versus those exposed after 10 days, to preventive medication. Results: We plan to enroll patients into the study and present data on completion. Conclusion: We aim to study the relationship between cluster headache onset and response to therapy. We propose that there is some data to suggest that earlier in cycle cluster headache may be more easily prevented, and we aim to study this relationship systematically. This is an important question that can influence treatment decisions in clinical practice, as well as clinical trial design. [ABSTRACT FROM AUTHOR]

Abstract Background The progressive course of pulmonary fibrosis (PPF) is observed with variable prevalence in different entities of fibrosing interstitial lung disease (fILD). PPF is characterised by worsening respiratory symptoms, declining lung function and increasing extent of fibrosis on high-resolution computer tomography. In Germany, data are limited on the characteristics and management of such patients. Methods/Design INSIGHTS-ILD is a prospective observational longitudinal registry designed to describe characteristics, management and course of newly diagnosed (incident) and prevalent patients with fILD on the long term. The registry uses a non-probability sampling approach to collect data on characteristics, therapeutic interventions, health-related quality of life and health economic parameters. It is planned to include 900 patients in ambulatory care in about 30 expert sites over three years. The study has been initiated in December 2021, and currently (January 2023) follows 360 patients. Discussion The registry is expected to provide much-needed data on the characteristics, management, and trajectories of patients fILD in Germany. The start of the study comes at a time when new treatment options are available for PPF. We hypothesize that PPF represents a broad clinical phenotype that is differentially influenced by inflammatory and fibrotic pathomechanisms that need to be treated with anti-inflammatory and/or anti-fibrotic treatment strategies. This registry will allow comparisons with other countries. Gap analyses based on current guidelines for management of these patients will be possible. Trial registration DRKS00027389 (registered on 7.12.2021), BfArM NIS 7562.

Granulomatous and lymphocytic interstitial lung disease (gl-ILD) is a major cause of morbidity and mortality among patients with common variable immunodeficiency. Corticosteroids are recommended as first-line treatment for gl-ILD, but evidence for their efficacy is lacking. This study analyzed the effect of high-dose corticosteroids (≥0.3 mg/kg prednisone equivalent) on gl-ILD, measured by high-resolution computed tomography (HRCT) scans, and pulmonary function test (PFT) results. Patients who had received high-dose corticosteroids but no other immunosuppressive therapy at the time (n = 56) and who underwent repeated HRCT scanning or PFT (n = 39) during the retrospective and/or prospective phase of the Study of Interstitial Lung Disease in Primary Antibody Deficiency (STILPAD) were included in the analysis. Patients without any immunosuppressive treatment were selected as controls (n = 23). HRCT scans were blinded, randomized, and scored using the Hartman score. Differences between the baseline and follow-up HRCT scans and PFT were analyzed. Treatment with high-dose corticosteroids significantly improved HRCT scores and forced vital capacity. Carbon monoxide diffusion capacity significantly improved in both groups. Of 18 patients, for whom extended follow-up data was available, 13 achieved a long-term, maintenance therapy independent remission. All patients with relapse were retreated with corticosteroids, but only one-fifth of them responded. Two opportunistic infections were found in the corticosteroid treatment group, while overall infection rate was similar between cohorts. Induction therapy with high-dose corticosteroids improved HRCT scans and PFT results of patients with gl-ILD and achieved long-term remission in 42% of patients. It was not associated with major side effects. Low-dose maintenance therapy provided no benefit and efficacy was poor in relapsing disease. [ABSTRACT FROM AUTHOR]

Background: The progressive course of pulmonary fibrosis (PPF) is observed with variable prevalence in different entities of fibrosing interstitial lung disease (fILD). PPF is characterised by worsening respiratory symptoms, declining lung function and increasing extent of fibrosis on high-resolution computer tomography. In Germany, data are limited on the characteristics and management of such patients. Methods/Design: INSIGHTS-ILD is a prospective observational longitudinal registry designed to describe characteristics, management and course of newly diagnosed (incident) and prevalent patients with fILD on the long term. The registry uses a non-probability sampling approach to collect data on characteristics, therapeutic interventions, health-related quality of life and health economic parameters. It is planned to include 900 patients in ambulatory care in about 30 expert sites over three years. The study has been initiated in December 2021, and currently (January 2023) follows 360 patients. Discussion: The registry is expected to provide much-needed data on the characteristics, management, and trajectories of patients fILD in Germany. The start of the study comes at a time when new treatment options are available for PPF. We hypothesize that PPF represents a broad clinical phenotype that is differentially influenced by inflammatory and fibrotic pathomechanisms that need to be treated with anti-inflammatory and/or anti-fibrotic treatment strategies. This registry will allow comparisons with other countries. Gap analyses based on current guidelines for management of these patients will be possible. Trial registration DRKS00027389 (registered on 7.12.2021), BfArM NIS 7562. [ABSTRACT FROM AUTHOR]

To compare maternal and perinatal outcomes between randomized trials and observational studies in which conservative management was performed for more than 48 h in patients with early-onset severe preeclampsia. We searched PubMed, LILACS, Cochrane and Google Scholar. The studies were divided in two groups: randomized and observational studies, from 1990 to 2018 that included patients with severe preeclampsia before 34 weeks of gestation with pregnancy prolongation ≥48 h but that did not include fetal growth restriction or HELLP syndrome at the beginning. The main variables recorded were maternal and perinatal complications. Forty-four studies met the inclusion criteria, and 5 of these were randomized. The average pregnancy prolongation was 9 days, with no difference between groups. Maternal complications were significantly more common in observational studies, RR = 0.71, 95% CI (0.54–0.93), p =.009. Perinatal complications were also significantly more common in observational studies (RR = 0.89, 95% CI (0.80–0.98), p =.01) at the expense of stillbirth and neonatal deaths. The percentages of cesarean sections were significantly higher in randomized studies, RR = 1.54, 95% CI (1.46–1.64). There were 2 maternal deaths, both in observational studies. Observational studies in which conservative management of early-onset preeclampsia is performed and do not include patients with fetal growth restriction or patients with HELLP syndrome and where at least 2 days of pregnancy prolongation is achieved are associated with significantly more maternal and perinatal complications. [ABSTRACT FROM AUTHOR]

Background and purpose: Biochemical recurrence after prostatectomy is commonly treated with salvage radiotherapy (SRT). In this prospective observational study we investigated the PSA decay rate, determined by predefined serial PSA measurements during SRT, as a predictor for treatment outcome. Materials and methods: Between 2013 and 2016, 214 patients were included in the study. The prescribed dose to the prostate bed was 70 Gy in 35 fractions (7 weeks) without hormonal treatment. PSA was measured weekly during SRT. Assuming first order kinetics, a PSA decay-rate constant (k) was calculated for 196 eligible patients. The ability of k to predict disease progression was compared with known clinical prediction parameters using Cox regression, logistic regression and ROC analyses. Disease progression was defined as continuously rising PSA after SRT, PSA increase by ≥0.2 ng/ml above nadir after SRT, hormonal treatment or clinical progression. Results: After a median follow up of 4.7 years the estimated failure-free survival at 5 years was 56%. The PSA decay-rate constant (k) was found to be the strongest predictor of disease progression in both uni-and multivariable analyses. Conclusion: The addition of k to established clinical variables significantly improves the possibility to predict treatment outcome after SRT and could be used to personalize future therapies.

In the current paper, we will focus on requirements to ensure big data can advance the outcomes of our patients suffering from kidney disease. The associated ethical question is whether and how we as a nephrology community can and should encourage the collection of big data of our patients. We identify some ethical reflections on the use of big data, and their importance and relevance. Furthermore, we balance advantages and pitfalls and discuss requirements to make legitimate and ethical use of big data possible. The collection, organization and curation of data come upfront in the pipeline prior to any analyses. Great care must therefore be taken to ensure quality of the data at this stage, to avoid the garbage in garbage out problem and suboptimal patient care as a consequence of such analyses. Access to the data should be organized so that correct and efficient use of data is possible. This means that data must be stored safely, so that only those entitled to do so can access them. At the same time, those who are entitled to access the data should be able to do so in an efficient way, so as not to hinder relevant research. Analysis of observational data is itself prone to many errors and biases. Each of these biases can finally result in provision of low-quality medical care. Secure platforms should therefore also ensure correct methodology is used to interpret the available data. This requires close collaboration of a skilled workforce of experts in medical research and data scientists. Only then will our patients be able to benefit fully from the potential of AI and big data., (© The Author(s) 2024. Published by Oxford University Press on behalf of the ERA.)

Ultrasound-guided intermediate cervical plexus blockade with perivascular infiltration of the carotid artery bifurcation perivacular block (PVB) is a reliable technique for regional anesthesia in carotid endarterectomy (CEA). We investigated the effect of the carotid bifurcation level (CBL) on PVB efficacy and safety in patients undergoing CEA. This prospective observational cohort study included 447 consecutive CEA patients who received PVB over a 6-y period. Vascular and neurologic puncture-related complications were recorded. The CBL was localized at the low level (C4 and C5 vertebra, low-level [LL] group) in 381 (85.2%) patients and at the high level (C2 and C3 vertebra, high-level [HL] group) in 66 (14.8%) patients. Local anesthetic supplementation by surgeons was necessary in 64 (14.3%) patients in the LL group and 38 (59.4%) patients in the HL group (p < 0.001) and was associated with a higher rate of central neurologic complications in the HL group (p = 0.031). Therefore, the efficacy of the PVB may be influenced by the CBL. [ABSTRACT FROM AUTHOR]

The main aim of this study is to systematically record Cerebrolysin treatment modalities and concomitant medication, according to local standards, in patients with moderate to severe neurological deficits after acute ischemic stroke and to assess the impact of these parameters on therapy outcome during early rehabilitation (day 21) and on day 90. An open observational treatment design based on the principles of high-quality comparative effectiveness research (HQCER) has been chosen to capture the therapies as applied in real-world clinical practice. HQCER opens a new horizon for strengthening the validity of the results from observational trials, thereby enhancing the associated level of evidence. Rigorous pre-specification of analytical procedures and tight risk-based centralized monitoring were additional measures to improve the impact of the observational approach. The value for real-world studies has become obvious, and such studies based on comparative effectiveness designs supplement the classical study designs by enabling the inclusion of larger proband numbers and more statistical reliability for practical use. [ABSTRACT FROM AUTHOR]

Abstract Background Chronic obstructive pulmonary disease (COPD) is the fifth leading cause of death in China with a reported prevalence of 8.2% people aged ≥40 years. It is recommended that Chinese physicians follow Global Initiative for Chronic Obstructive Lung Disease (GOLD) and national guidelines, yet many patients with COPD in China remain undiagnosed. Furthermore, missed diagnoses and a lack of standardized diagnosis and treatment remain significant problems. The situation is further complicated by a lack of large-scale, long-term, prospective studies of real-world outcomes, including exacerbation rates, disease severity, efficacy of treatment, and compliance of COPD patients in China. Methods/design The REALizing and improving management of stable COPD in China (REAL) study is a 52-week multi-center, prospective, observational trial. REAL aims to recruit approximately 5000 outpatients aged ≥40 years with a clinical diagnosis of COPD per GOLD 2016. Outpatients will be consecutively recruited from approximately 50 tertiary and secondary hospitals randomly selected across six geographic regions to provide a representative population. Patients will receive conventional medical care as determined by their treating physicians. The primary objective is to evaluate COPD patient outcomes including lung function, health status, exacerbations, hospitalization rate, and dyspnea following 1 year of current clinical practice. Secondary objectives are to assess disease severity, treatment patterns, adherence to medication, and associated risk factors. Data will be collected at two study visits, at patients’ usual care visits, and by telephone interview every 3 months. Discussion Knowledge of COPD among physicians in China is poor. The REAL study will provide reliable information on COPD management, outcomes, and risk factors that may help improve the standard of care in China. Patient recruitment began on 30 June 2017 and the estimated primary completion date is 30 July 2019. Trial registration ClinicalTrials.gov identifier: NCT03131362. Registered on 20 March 2017.

Background: Eribulin shows some activity in controlling brain metastasis in breast cancer. Methods: This observational, multicenter study evaluated brain disease control rates, survival and safety in patients with brain metastatic breast cancer treated with eribulin in clinical practice. Results: A total of 34 patients were enrolled (mean age 49 years, 91% with visceral metastases) and 29 were evaluable for brain disease. Fourteen achieved disease control and showed a longer time without progression: 10 months (95% CI: 2.3-17.7) versus 4 months (95% CI: 3.3-4.7) in the control group (p = 0.029). Patients with clinical benefits at 6 months had longer survival. Leukopenia and neutropenia were the most frequent grade 3-4 toxicities. Conclusion: Eribulin confirms its effectiveness in patients with brain metastatic breast cancer. Further studies on larger cohorts are needed to confirm the results. [ABSTRACT FROM AUTHOR]

Abstract Background Functional dyspepsia (FD) is a gastrointestinal disorder characterized by recurrent and diverse symptoms and pathophysiology that remains unexplained following routine clinical investigation. Enzynorm®f is a pharmaceutical preparation comprising fixed amounts of pepsin of biological origin and organically bound acid in the form of amino acid hydrochloride. It is traditionally used as a mild agent to support gastric function and to stimulate the stomach’s proteolytic activities in FD. Methods In a non-interventional, observational, post-marketing surveillance study, patients with an established diagnosis of FD were treated with a fixed combination of pepsin and amino acid hydrochloride taken as tablets three times daily for 6 weeks. The primary objective of this study was to assess the change in symptoms using the validated Gastrointestinal Symptom Score (GIS©). Secondary objectives included patients’ assessment of their gastrointestinal symptoms as well as treatment safety and tolerability. Results A total of 97 patients (mean age 58.4 ± 13.9 years; 63.2% females) were included in the study, with 72 data having GIS© score data at baseline and at 6 weeks, and 34 also at 3 weeks. The overall GIS© sum score decreased by 4.1 (p

Background: Human epidermal growth factor receptor 2 (HER2) is a promising therapeutic target in metastatic colorectal cancer (mCRC). This study was to evaluate the efficacy and safety of pyrotinib alone or pyrotinib with trastuzumab in patients with HER2-positive mCRC., Patients and Methods: In this prospective observational study, patients with HER2 positive, Ras Sarcoma Viral Oncogene Homolog (RAS) wild type mCRC who received at least one standard treatment of palliative chemotherapy were enrolled. Patients were treated with oral pyrotinib alone or pyrotinib with trastuzumab. The primary endpoint was progression free survival (PFS), and the secondary endpoints were overall survival (OS), confirmed objective response rate (ORR), and safety. This trial is registered with chitcr.org, number ChiCTR2100046381., Results: From February 15, 2021, to January 10, 2023, 32 patients were enrolled in this study. Twenty (62.5%) patients were treated with pyrotinib, while 12 (37.5%) received pyrotinib and trastuzumab. As of June 24, 2023, with a median follow-up of 11.0 months, the median PFS was 5.7 months (95%CI 4.5-10.2), while OS was not evaluable (NE), ORR and disease control rate (DCR were 34.4% and 87.5%. Patients' PFS in the pyrotinib plus trastuzumab subgroup and pyrotinib monotherapy group were 8.6 and 5.5 months, OS was not evaluable (NE) and 10.9 months, ORR was 50.0% and 25.0%, respectively. Most treatment-related adverse events (TRAEs) were grade 1-2, diarrhea was the most frequent TRAE (81.3%, 26/32). Grade 3 TRAEs occurred in 11 patients: 9 for diarrhea, 1 for nausea, and 1 for oral mucositis., Conclusion: Pyrotinib with or without trastuzumab showed promising anti-tumor activity and acceptable toxicities in treatment-refractory, HER2-positive mCRC., Competing Interests: Disclosure This study was funded by the Special Clinical Research Program of Shanghai Municipal Health Commission Health Industry (No. 202040222). All authors state that they have no conflicts of interest., (Copyright © 2023. Published by Elsevier Inc.)

Background: Chronic obstructive pulmonary disease (COPD) is the fifth leading cause of death in China with a reported prevalence of 8.2% people aged ≥40 years. It is recommended that Chinese physicians follow Global Initiative for Chronic Obstructive Lung Disease (GOLD) and national guidelines, yet many patients with COPD in China remain undiagnosed. Furthermore, missed diagnoses and a lack of standardized diagnosis and treatment remain significant problems. The situation is further complicated by a lack of large-scale, long-term, prospective studies of real-world outcomes, including exacerbation rates, disease severity, efficacy of treatment, and compliance of COPD patients in China.Methods/design: The REALizing and improving management of stable COPD in China (REAL) study is a 52-week multi-center, prospective, observational trial. REAL aims to recruit approximately 5000 outpatients aged ≥40 years with a clinical diagnosis of COPD per GOLD 2016. Outpatients will be consecutively recruited from approximately 50 tertiary and secondary hospitals randomly selected across six geographic regions to provide a representative population. Patients will receive conventional medical care as determined by their treating physicians. The primary objective is to evaluate COPD patient outcomes including lung function, health status, exacerbations, hospitalization rate, and dyspnea following 1 year of current clinical practice. Secondary objectives are to assess disease severity, treatment patterns, adherence to medication, and associated risk factors. Data will be collected at two study visits, at patients' usual care visits, and by telephone interview every 3 months.Discussion: Knowledge of COPD among physicians in China is poor. The REAL study will provide reliable information on COPD management, outcomes, and risk factors that may help improve the standard of care in China. Patient recruitment began on 30 June 2017 and the estimated primary completion date is 30 July 2019.Trial Registration: ClinicalTrials.gov identifier: NCT03131362. Registered on 20 March 2017. [ABSTRACT FROM AUTHOR]

Background: There is limited understanding of the feasibility of conducting long-term research among undiagnosed (pre-symptomatic) adults at risk to develop Huntington disease (HD), while protecting their emotional well-being and safety. Objective: To assess pre-specified events pertaining to emotional well-being, safety, and feasibility among healthy consenting adults at risk for developing HD who have chosen not to undergo genetic testing. Methods: PHAROS research participants prospectively reported the occurrence of events pertaining to psychological distress (psychiatric evaluations, depression, suicidality) and feasibility (maintaining confidentiality, study attrition). PHAROS enrolled 1001 participants. Results: Events pertaining to psychological distress were reported by 35% of participants. The most common events included heightened suicide risk (26%), new onset depression (12%), and new mental health evaluation (9%); all occurred significantly more frequently among participants with expanded trinucleotide CAG repeats (≥37). Five deaths occurred, none related to suicide. Forty-one percent of participants reported self-disclosure of their HD at-risk status, and 15% reported that someone else (usually a family member) had done so. Confidentiality of CAG test results was maintained by investigators. The withdrawal rate was largely uniform over the study period and did not differ significantly by gender or CAG status. Conclusions: The potentially vulnerable research participants in PHAROS showed good emotional tolerability and safety. Individual CAG data were not disclosed, and confidentiality about disclosure of at-risk HD status was well maintained by others (family, friends, etc.). Long-term research participation of adults at risk for HD who choose not to undergo pre-symptomatic DNA testing is well tolerated, safe and feasible. [ABSTRACT FROM AUTHOR]

Clinical drug trials in Irkutsk Scientific Center of Surgery and Traumatology began in 2005. In 2013, the laboratory of clinical trials was established in the science department of clinical surgery. We analyzed the experience of Irkutsk Scientific Center of Surgery and Traumatology in international multicentre trials. Participation in international multicentre trials allows the Irkutsk Scientific Center of Surgery and Traumatology to get affiliated with the publications in first quartile scientific journals. The experience of the laboratory in clinical trials is projected into practical work of other institutes - Hospital of Irkutsk Scientific Center SB RAS and Irkutsk Regional Clinical Hospital. The important objectives of the laboratory of clinical trials are developing the protocols, performing drug clinical trials and medical device clinical trials. The laboratory carries out admission of patients for observational trial PROTOCOL. One of the fields of work of the laboratory is the prospect of clinical testing of developed clinical technologies.

Purpose: The OVA-YOND study is the first prospective, non-interventional trial designed to evaluate trabectedin (1.1 mg/m2) plus PLD (30 mg/m2) in patients with platinum-sensitive recurrent ovarian cancer (ROC), given according to the marketing authorization in real-life clinical practice across Germany.Methods: Eligible patients were adults with platinum-sensitive ROC, pretreated with ≥ 1 platinum-containing regimen/s. The primary endpoint was to assess safety/tolerability of the combination.Results: Seventy-seven patients with platinum-sensitive relapse from 31 sites were evaluated. Patients received a median of 6 cycles (range 1-21) with 39 patients (50.6%) receiving ≥ 6 cycles. Median treatment duration was 4.2 months (range 0.7-18.8), mostly on an outpatient basis (88.3% of patients). Most common grade 3/4 trabectedin-related adverse events (AEs) were leukopenia (18.2%), neutropenia (15.6%), thrombocytopenia (9.1%), alanine (7.8%) and aspartate aminotransferase (6.5%) increase, and nausea/vomiting (5.2% each). Neutropenia (18.2%), leukopenia (15.6%), thrombocytopenia (10.4%), and nausea/vomiting (5.2% each) were the most frequent grade 3/4 PLD-related AEs. No deaths attributed to drug-related AEs or unexpected AEs occurred. Five patients (6.5%) had a complete response and 19 patients (24.7%) achieved a partial response for an objective response rate of 31.2% with median response duration of 6.25 months. Sixteen patients (20.8%) had disease stabilization for a disease control rate of 51.9%. Median progression-free survival was 6.3 months and median overall survival was 16.4 months.Conclusion: Trabectedin plus PLD confer clinically meaningful benefit to pre-treated patients with platinum-sensitive ROC, being comparable to those previously observed in selected populations from clinical trials and with a manageable safety profile. [ABSTRACT FROM AUTHOR]

Objectives: Aerococcus urinae and Aerococcus sanguinicola cause urinary tract infections (UTIs) and antibiotic treatment recommendations are solely based on in vitro findings and limited clinical experience. Our objective was to investigate the effectiveness of different treatment strategies in aerococcal UTI through a prospective observational study.Methods: Urine samples with aerococci were identified and patients were enrolled. The aerococci were subjected to Etests. Information on clinical symptoms, and the treatment given, was collected. Patients were interviewed after the conclusion of treatment to assess clinical cure and a control urine culture assessed the microbiological cure.Results: Of 31,629 urine samples, 144 grew aerococci and fulfilled the inclusion criteria. 91 patients gave consent and the 72 patients with UTI were assessed for treatment outcome. 53 patients had A. urinae UTI, while 19 had A. sanguinicola UTI. Nitrofurantoin was most commonly prescribed, achieving clinical and microbiological success in 71/76% of cases of A. urinae UTI, and 42/50% of cases of A. sanguinicola UTI. Pivmecillinam achieved success in patients with A. urinae cystitis and ciprofloxacin in patients with pyelonephritis.Conclusions: Our results support that nitrofurantoin is a valid option for the treatment of cystitis caused by A. urinae. [ABSTRACT FROM AUTHOR]

Background: Functional dyspepsia (FD) is a gastrointestinal disorder characterized by recurrent and diverse symptoms and pathophysiology that remains unexplained following routine clinical investigation. Enzynorm®f is a pharmaceutical preparation comprising fixed amounts of pepsin of biological origin and organically bound acid in the form of amino acid hydrochloride. It is traditionally used as a mild agent to support gastric function and to stimulate the stomach's proteolytic activities in FD.Methods: In a non-interventional, observational, post-marketing surveillance study, patients with an established diagnosis of FD were treated with a fixed combination of pepsin and amino acid hydrochloride taken as tablets three times daily for 6 weeks. The primary objective of this study was to assess the change in symptoms using the validated Gastrointestinal Symptom Score (GIS©). Secondary objectives included patients' assessment of their gastrointestinal symptoms as well as treatment safety and tolerability.Results: A total of 97 patients (mean age 58.4 ± 13.9 years; 63.2% females) were included in the study, with 72 data having GIS© score data at baseline and at 6 weeks, and 34 also at 3 weeks. The overall GIS© sum score decreased by 4.1 (p < 0.0001) from 11.6 (±4.8) at baseline to 7.4 (± 4.6) reflecting an improvement of clinical symptomatology after 6 weeks of treatment. In a subgroup of 70 patients who had FD meeting the Rome III criteria a GIS© score reduction of ≥50% was observed after 3 weeks treatment in 24% and in 30.8% after 6 weeks. Adverse events were mostly gastrointestinal in nature and consistent with the underlying disease; no unexpected adverse reactions were reported. Twenty-seven patients discontinued the study, mostly because of gastrointestinal symptoms.Conclusion: The results of this study support the efficacy of a fixed combination of pepsin and amino acid hydrochloride for the treatment of patients with FD and also suggest good to moderate treatment tolerability. These findings should be further explored in a randomised, placebo-controlled clinical trial.Clinical Trial Registration: This study has been retrospectively registered in the ClinicalTrials.gov registry, trial identifier NCT03076411 . [ABSTRACT FROM AUTHOR]

Huntington disease ( HD) is a late onset ultimately fatal neurodegenerative disorder caused by a cytosine-adenine-guanine ( CAG) triplet repeat expansion in the Huntingtin gene which was discovered in 1993. The PHAROS study is a unique observational study of 1001 individuals at risk for HD who had not been previously tested for HD and who had no plans to do so. In this cohort, 104 (10%) individuals changed their minds and chose to be tested during the course of the study but outside of the study protocol. Baseline behavioral scores, especially apathy, were more strongly associated with later genetic testing than motor and chorea scores, particularly among subjects with expanded CAG repeat length. In the CAG expanded group, those choosing to be tested were older and had more chorea and higher scores on the behavioral section of the unified Huntington's disease rating scale at baseline than those not choosing to be tested. Following genetic testing, 56% of subjects with CAG < 37 had less depression when compared to prior to testing, but depression generally stayed the same or increased for 64% of subjects in the expanded group. This finding suggests that approaches to testing must continue to be cautious, with appropriate medical, psychological and social support. [ABSTRACT FROM AUTHOR]

Recent reports indicate that total hip arthroplasty (THA) can be further accelerated and might be managed as day-case. This could provide benefits to health care expenditure, lower patient risks and change patient satisfaction. We evaluated the feasibility, efficacy, safety and patient satisfaction of same-day (<12 hours) THA operation and discharge.~Introduction~Background~Consecutive patients were screened for eligibility (primary THA, ASA 1 or 2 physical status) and the presence of a support person to follow the patient for 48 hours postoperatively in an observational study. THA was managed as day-case (<12 hours) through patient selection, preoperative patient education programme, light spinal anaesthesia, low opioid usage, and early mobilisation. Efficacy and logistics were analysed and compared to current accelerated practice. Patient satisfaction was compared to existing fast-track THA and elective surgery cohorts.~Methods~Methods~29 consecutive patients were screened and 20 participated (69%). 17/20 patients (85%) were released on the day of surgery. There were no serious adverse events or complications, and no infections or readmission at 6-week follow-up. Patient overall satisfaction rating (1-5) was high (4.75), higher than in a 301-patient cohort (4.53) (THA and total knee arthroplasty) from the same centre (p = 0.23), and higher than in a national, elective surgery cohort (4.3) (p = 0.04).~Results~Results~We report the feasibility of day-case THA. Safety, complications and overall patient acceptance were satisfactory. Light spinal anaesthesia facilitated quick transit through postanaesthesia care unit to early mobilisation. Support persons and patient education may play a part in high patient acceptance of day-case surgery.~Conclusions~Conclusions [ABSTRACT FROM AUTHOR]

Background Fragile X syndrome (FXS), caused by a mutation of the FMR1 gene on the X chromosome, is the most common inherited form of intellectual disability and autism spectrum disorders. Comprehensive data are lacking, however, on the characteristics and management patients with FXS in Germany. Methods/design EXPLAIN is a prospective, observational, longitudinal registry with a non-probability sampling approach. It collects data on patient characteristics, therapeutic interventions, psychosocial parameters (including those of family members and caregivers), quality of life of caregiver and patient, caregiver burden, and health economic parameters, such as hospitalisation time. It is designed to include data from 300 patients in ambulatory care from about 50 centres that employ psychiatrists, paediatricians, neurologists, and other relevant specialists, in Germany. The study was initiated in March, 2013. Patients will be followed for at least two years. Discussion The registry is expected to provide much-needed data on the characteristics and management of patients with FXS in Germany. It will also allow comparisons with other countries, and will enable gap analyses based on current guidelines for management of these patients. [ABSTRACT FROM AUTHOR]

Objective of this observational trial is to examine the effects of quetiapine in comparison with olanzapine and risperidone on clinical outcomes and quality of life in patients with schizophrenia and schizoaffective disorder in routine care. 374 adult persons with schizophrenia or schizoaffective disorder prescribed antipsychotic maintenance therapy with quetiapine, olanzapine, or risperidone at discharge from inpatient treatment were included. Clinical and psychosocial outcomes were assessed before discharge and at 6, 12, 18, and 24 months. Statistical analyses were conducted by mixed-effects regression models for longitudinal data. The propensity score method was used to control for selection bias. Patients discharged on olanzapine had significantly lower hospital readmissions than those receiving quetiapine or risperidone. The average chlorpromazine equivalent dose of quetiapine was higher than in patients treated with olanzapine or risperidone. No further significant differences between treatment groups were found. Quetiapine and risperidone are less effective in preventing the need for psychiatric inpatient care than olanzapine, and higher chlorpromazine equivalent doses of quetiapine are needed to obtain clinical effects similar to those of olanzapine and risperidone. [ABSTRACT FROM AUTHOR]

Background: Randomized controlled efficacy trials (RCTs), the scientific gold standard, are required for regulatory approval of Alzheimer's disease (AD) interventions, yet provide limited information regarding real-world therapeutic effectiveness. Objective: To compare the nature of evidence regarding the combination of approved AD treatments from RCTs versus long-term observational controlled studies (LTOCs). Methods: Comparisons of strengths, limitations, and evidence level for monotherapy [cholinesterase inhibitor (ChEI) or memantine] and combination therapy (ChEI + memantine) in RCTs versus LTOCs. Results: RCTs examined highly selected populations over months. LTOCs collected data across multiple AD stages in large populations over many years. RCTs and LTOCs show similar patterns favoring combination over monotherapy over placebo/no treatment. Long-term combination therapy compared to monotherapy reduced cognitive and functional decline and delayed time to nursing home admission. Persistent treatment was associated with slower decline. While LTOCs used control groups, adjusted for multiple covariates, had higher external validity, and favorable ethical, practical and cost considerations, their limitations included potential selection bias due to lack of placebo comparisons and randomization. Conclusions: Naturalistic LTOCs provide complementary long-term level II evidence to complement level I evidence from short-term RCTs regarding therapeutic effectiveness in AD that may otherwise be unobtainable. A coordinated strategy/consortium to pool LTOC data from multiple centers to estimate long-term comparative effectiveness, risks/benefits, and costs of AD treatments is needed. Copyright © 2012 S. Karger AG, Basel [ABSTRACT FROM AUTHOR]

Aim: Controversies and disagreement exist on conventional treatment strategies of hemorrhoids due to relapse, inefficacy, and complications. We intend to evaluate the role of individualized homeopathic treatment in hemorrhoids., Materials and Methods: In this prospective, open, observational trial, hemorrhoids patients were treated using five standardized scales measuring complaints severity and anoscopic score. It was conducted at two homeopathic hospitals in India, during from mid-July 2014 to mid-July 2015. Patients were intervened as per individualized homeopathic principles and followed up every month up to 6 months., Results: Total 73 were screened, 52 enrolled, 38 completed, 14 dropped out. Intention to treat population ( n : = 52) was analyzed in the end. Statistically significant reductions of mean bleeding (month 3: -21.8, 95% confidence interval [CI]: -30.3, -13.3, P : < 0.00001, d = 0.787; month 6: -25.5, 95% CI -35.4, -15.6, P : < 0.00001, d = 0.775), pain (month 3: -21.3, 95% CI -28.6, -14.0, P : < 0.00001, d = 0.851; month 6: -27.6, 95% CI -35.6, -19.6, P : < 0.00001, d = 1.003), heaviness visual analog scales (VASs) (month 3: -8.1, 95% CI -13.9, -2.3, P : = 0.008, d = 0.609; month 6: -12.1, 95% CI -19.1, -5.1, P : = 0.001, d = 0.693), and anoscopic score (month 3: -0.4, 95% CI -0.6, -0.2, P : < 0.0001, d = 0.760; month 6: -0.5, 95% CI -0.7, -0.3, P : < 0.0001, d = 0.703) were achieved. Itching VASs reduced significantly only after 6 months (-8.1, 95% CI -14.6, -1.6, P : = 0.017, d = 0.586). No significant lowering of discharge VASs was achieved after 3 and 6 months., Conclusion: Under classical homeopathic treatment, hemorrhoids patients improved considerably in symptoms severity and anoscopic scores. However, being observational trial, our study cannot provide efficacy data. Controlled studies are required. Trial Reg. CTRI/2015/07/005958., Competing Interests: Conflicts of Interest: None declared.

Objectives: The effects of guided self-help interventions for parents of children with ADHD have already been proven in randomized controlled trials. The objective of this study was to assess the effectiveness of this novel form of intervention under routine care conditions in a nationwide trial., Method: Registered pediatricians as well as child and youth psychiatrists enrolled 274 children between 6 and 12 years old (83.6% male) diagnosed with ADHD to a self-help program for parents of children with ADHD. The program lasted for 1 year and consisted of eight booklets with advice for parenting children with ADHD as well as complementary telephone consultations (14 calls, up to 20 minutes each). The course of the ADHD symptoms and the comorbid symptoms as well as the development of the child’s individual problems were assessed in a pre-post design., Results: 63% of the enrolled parents adhered to the program until the end. The families who cancelled the program did not differ concerning the severity of ADHD symptoms, but they did more often show an impaired familial and social background, and their children received pharmacological treatment more often. Three-fourths of the children who completed the program had received pharmacological treatment at the beginning of the program. The children had more severe ADHD symptoms than a clinical control group. During the intervention, ADHD symptoms as well as psychosocial functioning improved with large effect sizes of d>0.9. Additionally, comorbid oppositional and emotional symptoms decreased., Conclusions: These results indicate that guided self-help programs for families with children with ADHD are effective, also as an addition to pharmacological treatment.