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Background: Data and Safety Monitoring is integral to quality assurance of clinical trials. Although monitoring is a core legal component of regulated clinical trials, non-regulated trials are not mandated to incorporate monitoring. Consequently, the monitoring process has been underutilised and underreported in this setting. This research report outlines the development and plans for implementing a bespoke Clinical Monitoring Strategy within the ' Rehabilitation Strategies Following Oesophagogastric and Hepatopancreaticobiliary Cancer (ReStOre II) Trial' , a non-regulated trial comparing a 12-week multidisciplinary programme of rehabilitation to standard care in a cohort of 120 cancer survivors., Methods: This research note provides a detailed overview of the ReStOre II Clinical Monitoring Strategy and describes the development of the strategy pre and post awarding of the grant. The strategy consists of the establishment and implementation of a comprehensive trial governance structure, inclusive of a Trial Management Group, Trial Steering Committee Meeting, and Independent Data Monitoring Committee. In addition, external trial monitoring by the Clinical Research Facility at St James's Hospital. Three monitoring visits will be conducted during the trial; i) site initiation visit, ii) interim monitoring visit, and iii) close our visit., Results: The Clinical Monitoring Strategy has been finalised and is currently being implemented within the ReStOre II Trial. Two site initiation visits and one interim monitoring visit have been completed to date., Conclusion: This research note provides a template for implementation of a Clinical Monitoring Strategy in a non-regulated clinical trial., Registration: ReStOre II Trial: https://clinicaltrials.gov/ct2/show/NCT03958019., Competing Interests: No competing interests were disclosed., (Copyright: © 2024 O'Neill L et al.)

Introduction: Asthma is a pathology that remains severe and is inadequately controlled in 4% of patients. Identification of multiple pathophysiological mechanisms has led to the development of biomedicines, of which there are currently five available in France, with a safety profile that appears favorable but remains uncertain due to a lack of real-life experience with these new molecules., State of Knowledge: Although relatively benign, the adverse effects of biologics are diverse. Headache, joint pain, skin reactions at the injection site, fever and asthenia are commonly observed during the different treatments. Ophthalmological complications seem restricted to dupilumab, with numerous cases of keratitis and conjunctivitis in patients with atopic dermatitis. Several respiratory complications have also been observed, essentially consisting in pharyngitis and other upper respiratory infections. Hypereosinophilia may occur, mainly with dupilumab, requiring investigation of systemic repercussions or vasculitis. Allergic reactions are uncommon but require careful monitoring during initial injections., Conclusion: Biologics for severe asthma are recent drugs with a favorable safety profile, but with little real-life experience, justifying increased vigilance by prescribing physicians., (Copyright © 2024 SPLF. Published by Elsevier Masson SAS. All rights reserved.)

Background: The impact of inflammatory bowel disease (IBD) on caregivers has rarely been investigated. Our work aims to explore the burden of IBD on the different aspects of caregivers' lives., Methods: We conducted an online survey via the social network of the French IBD patient organization addressed to patients' caregivers who were asked to fill in a questionnaire covering the impact of the disease on different aspects of their lives. Impacts were measured by a visual analog scale (VAS). A VAS score ≥5 of 10 was considered significantly high. We then performed uni- and multivariate analyses of predictors of higher impact on parents and partners, separately., Results: A total of 853 caregivers participated, predominantly women (77%). The mean age was 48.5 years. Their relationship with the patient was mainly parents in 57.1% and partners in 30.6%. The type of IBD was Crohn's disease in 63% and ulcerative colitis in 35%. The psychological burden was the highest among parents and was mainly correlated with a lack of knowledge about IBD and professional compromise (P < .05). The impact on leisure was the highest among partners and was affected by psychological, sexual, and professional burdens (P < .05). About 50% of partners experienced an absent sexual relationship for weeks/months and decreased libido regardless of IBD type. Professional impact was greater in parents compared with partners, and 36% of caregivers needed work arrangements., Conclusions: IBD has a high impact on different aspects of life of patients' caregivers. Therefore, interventions to reduce the disease burden in this population are needed., (© The Author(s) 2023. Published by Oxford University Press on behalf of Crohn’s & Colitis Foundation. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Aims: In patients with ulcerative colitis (UC), no biomarker is available to help the physician to choose the most suitable biotherapy. The primary objective of this pilot study was to assess the feasibility of identification of α4β7- and TNF-expressing cells, to predict the response to vedolizumab using confocal laser endoscopy (CLE)., Methods: Patients with moderate-to-severe UC, naïve of biotherapy, received vedolizumab. Clinical evaluation was performed at each infusion. Endoscopic evaluation was performed before inclusion and at week 22. Fresh colonic biopsies were stained using FITC-labelled vedolizumab and Alexa fluor-labelled adalimumab and ex vivo dual-band CLE images were acquired. Blood samples were collected to measure trough concentrations of vedolizumab and to determine absolute counts of T and B cells subpopulations, NK cells and monocytes., Results: Nineteen patients were enrolled in the study and received at least one dose of vedolizumab. Clinical remission and endoscopic improvement were observed in 58% of whom 5 patients (45%) had an endoscopic subscore of 0. In terms of clinical response and remission, endoscopic improvement and histologic response, FITC-conjugated vedolizumab staining tended to be higher in responder patients compared to non-responders at week 22. A threshold value of 6 positive FITC-vedolizumab staining areas detected by CLE seemed informative to discriminate the responders and non-responders. The results were similar in terms of clinical remission and endoscopic improvement with a sensitivity of 78% and a specificity of 85% (p = 0.05). Trough concentrations and blood immune cells were not associated with responses to vedolizumab., Conclusion: This pilot study demonstrate that dual-band CLE is feasible to detect α4β7- and TNF-expressing cells. Positive α4β7 staining seems to be associated with clinical and endoscopic remission in UC patients treated by anti-α4β7-integrin, subject to validation by larger-scale studies. Clinical-trial.gov: NCT02878083., Competing Interests: I have read the journal’s policy and the authors of this manuscript have the following competing interests: CTP (2) has received personal fees from Takeda for lecture and research grant from MSD. MF (2) has received personal lecture fees from Takeda, Abbvie and MSD. GB (5) has received personal fees from Takeda, Abbvie and MSD for lecture and advisory board. AB has received personal fees from Takeda, Abbvie and MSD for lecture and advisory board and research grants from Takeda and Medtronic. LQ (1), AF (3), TD, JB, TO, MA, MD, SBa, SBl, CB, MAV, RJ and MN have declared that no conflict of interest exist. This does not alter our adherence to PLOS ONE policies on sharing data and materials., (Copyright: © 2024 Quénéhervé et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.)

Facing with an increasing demand for transition to adult care management, our home parenteral nutrition (HPN) team designed an adolescent therapeutic educational program (ATEP) specifically intended for adolescents on long-term HPN. The aim of this study was to report on the first sessions of this program., Methods: The ATEP is designed in three sessions of five consecutive days, during school holidays over the year. It includes group sessions on catheter handling, disconnecting and connecting the PN and catheter dressing, dealing with unforeseen events (e.g., fever or catheter injury), but also sessions with psychologist, social worker, sports teacher, fashion specialist, meeting with adults who received HPN since childhood. Specific course for the accompanying parents were also provided. Six months after the last session, a 3-day trip to the attraction park "le Futuroscope," Poitiers, France, was organized without any parental presence., Results: After 3 ATEP courses, a total of 16 adolescents have been enrolled. They were aged between 13 and 17 years (median 14 IQR: 14-16.25). All were on long term HPN started during the neonatal period except for four who started PN at a median age of 10 years old (IQR: 1-10). At the time of the ATEP, their median PNDI was 105% (IQR: 95.5-120.8) while receiving a median of six infusions per week (IQR: 5-7). Thirteen received Taurolidine lock procedure. After the ATEP, 11 adolescents could be considered as fully autonomous, 4 as partially autonomous and one failed to gain any autonomy. Course evaluation by adolescents or parents was good to excellent., Conclusion: Through the holistic and multiprofessional approach of this training and the group cohesion, the adolescents were not only able to handle catheter care and PN connections but were able to understand and accept better their illness and project themselves into their own future., Competing Interests: The authors declare no conflict of interest., (© 2024 The Authors. JPGN Reports published by Wiley Periodicals LLC. on behalf of the European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition.)