Cardiovascular events observed among patients in the etrasimod clinical programme: an integrated safety analysis of patients with moderately to severely active ulcerative colitis.

Objective: Etrasimod is an oral, once-daily, selective sphingosine 1-phosphate (S1P) receptor modulator for the treatment of moderately to severely active ulcerative colitis (UC). S1P receptor expression on cardiac cells is involved in cardiac conduction. We report cardiovascular treatment-emergent adverse events (TEAEs) associated with S1P receptor modulators and other cardiovascular events in the etrasimod UC clinical programme.

Comparison of the lethal triad and the lethal diamond in severe trauma patients: a multicenter cohort.

Background: To reduce the number of deaths caused by exsanguination, the initial management of severe trauma aims to prevent, if not limit, the lethal triad, which consists of acidosis, coagulopathy, and hypothermia. Recently, several studies have suggested adding hypocalcemia to the lethal triad to form the lethal diamond, but the evidence supporting this change is limited. Therefore, the aim of this study was to compare the lethal triad and lethal diamond for their respective associations with 24-h mortality in severe trauma patients receiving transfusion.

Body composition is associated with changes in health-related quality of life in patients with knee and/or hip osteoarthritis.

Objective: The objective of this study was to analyse the association between body composition and changes in health-related quality of life (HRQoL) of patients followed for hip and knee osteoarthritis (OA).

Methods: Longitudinal data from the Knee and Hip OsteoArthritis Long-term Assessments (KHOALA) cohort, a multicentre cohort of 878 patients with symptomatic knee and/or hip OA, were used. The main outcome criteria were changes in patient-reported outcomes measures, the Study Short Form-36 (physical functioning, pain, mental health and vitality) and the OsteoArthritis Knee and Hip Quality Of Life (OAKHQOL)(physical activity, pain and mental health).

Tatton-Brown-Rahman syndrome: a new multiple endocrine neoplasia syndrome with intellectual disability?

We describe for the first time the case of a woman presenting with Tatton-Brown-Rahman syndrome (TBRS) and multiple endocrine neoplasia (MEN). She developed primary hyperparathyroidism at age 13, a pituitary cyst at age 14, adrenal tumor at age 21, and metastatic insulinoma at age 34. In addition, she showed intellectual disability, obesity, multiple lipomas, facial dysmorphia, hemihypertrophy and kyphoscoliosis.

Free medial sural artery perforator flap for soft tissue defects in the hand: a cadaver study.

We carried out a cadaver study using 10 fresh-frozen adult legs and hands to explore technical feasibility and reproducibility of the free Medial Sural Artery Perforator flap and its applicability for covering soft tissue defects in the hand. A mean of 2 cutaneous perforators (1-4) were found. Each flap had a pedicle composed of a main perforator that arose from the medial sural artery.

Nanopore Sequencing as a Cutting-Edge Technology for Medulloblastoma Classification.

Background: Medulloblastoma (MB) is one of the most prevalent embryonal malignant brain tumors. Current classification organizes these tumors into four molecular subgroups (WNT, SHH, Group 3, and Group 4 MB). Recently, a comprehensive classification has been established, identifying numerous subtypes, some of which exhibit a poor prognosis.

A randomized phase 2 study of efmarodocokin alfa, an IL-22 agonist, versus vedolizumab in patients with ulcerative colitis.

Background And Aims: Efmarodocokin alfa is an interleukin (IL)-22 agonist, with favorable pharmacokinetic (PK) properties and an acceptable safety profile. This study further explored the therapeutic potential of efmarodocokin alfa compared to vedolizumab in patients with ulcerative colitis (UC).

Methods: This randomized phase 2 trial evaluated the efficacy, safety, PK, and pharmacodynamics of 3 doses of efmarodocokin alfa administered intravenously every 4 weeks (30 μg/kg [n=43], 60 μg/kg [n=44], and 90 μg/kg [n=43]) compared with placebo (n=22) and with vedolizumab (n=43) in the treatment of moderate to severe UC.

Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy.

Context: Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial.

Acute Severe Ulcerative Colitis: An International Delphi Consensus on Clinical Trial Design and Endpoints.

Background & Aims: Interventional clinical trials in ASUC are characterised by substantial heterogeneity due to a lack of consensus in several key areas of trial design - this impedes clinical research efforts to identify novel therapies. The objective of this initiative was to achieve the first consensus and provide clear position statements on ASUC trial design.

Methods: A modified Delphi consensus approach was employed with a panel of twenty clinicians with international representation and expertise in ASUC trial design and delivery.

Central nervous system complications of immune checkpoint inhibitors: A comprehensive review.

The ever-increasing use of immune checkpoint inhibitors (ICIs) has significantly improved cancer management, but at the cost of frequent immunologic side effects. Among them, neurologic immune-related adverse events (nirAEs) are less common but pose a challenge to clinicians due to their severity, heterogeneous nature and nonspecific clinical presentation, making diagnosis complex. The prognosis of these nirAEs, especially those related to the central nervous system (CNS), correlates with their rapid recognition and therapeutic management.

Vasodilator drugs and heart-related outcomes in systemic sclerosis: an exploratory analysis.

Background And Aims: Systemic sclerosis (SSc) is an autoimmune connective disease characterised by excessive extracellular matrix deposition and widespread skin and internal organ fibrosis including various cardiac manifestations. Heart involvement is one of the leading causes of death among patients with SSc. In this study, we aimed to assess the effect of various vasodilator treatments.

Methods of nivolumab administration in advanced melanoma: A comparison of patients' clinical outcomes treated with flat dose or weight-adjusted dose, a multicenter observational study.

Background: Nivolumab obtained approval in advanced melanoma (AM) with weight-adjusted dose (WAD) administration (3 mg/kg/2 weeks). In 2018, the dosage regimen was changed to flat dose (FD) administration (240 mg/2 weeks or 480 mg/4 weeks) based on a modeling study, without clinical data.

Methods: AM patients have been prospectively included in the French national multicenter MelBase database since 2013.

Disease-modifying treatment and disability progression in subclasses of patients with primary progressive MS: results from the Big MS Data Network.

Background: Effectiveness of disease-modifying treatment (DMT) in people affected by primary progressive multiple sclerosis (PPMS) is limited. Whether specific subgroups may benefit more from DMT in a real-world setting remains unclear. Our aim was to investigate the potential effect of DMT on disability worsening among patients with PPMS stratified by different disability trajectories.

Standards for Quality Improvement Registries on Abdominal Aortic Aneurysm Repair: A Delphi Consensus Report From VASCUNET and International Consortium of Vascular Registries.

Objective: Outcome registries in vascular surgery are increasingly used to drive quality improvement by vascular societies. The VASCUNET collaboration, within the European Society for Vascular Surgery (ESVS), and the International Consortium of Vascular Registries (ICVR) developed a set of variables for quality improvement registries on abdominal aortic aneurysm (AAA) repair as a registry standard.

Methods: Representatives from international vascular registries within VASCUNET, ICVR, and other nations with established registries were invited to provide the variables.

Association between education level and access to disease-modifying treatment in patients with multiple sclerosis in France.

Background: We hypothesized that differences in access to disease-modifying treatments (DMTs) could explain the association between socioeconomic status and disability progression in multiple sclerosis (MS).

Objective: This study aimed to analyze the association between education level and DMT use in France.

Methods: All patients from OFSEP network with MS onset over 1996-2014 and aged ⩾ 25 years at onset were included.

Association between education level and disability progression in patients with multiple sclerosis in France.

Background: Studies have reported an association between socioeconomic status and disability progression in multiple sclerosis (MS), but findings using the pre-MS individual socioeconomic status are missing.

Objective: The objective was to investigate the association between education level and disability progression.

Methods: All Observatoire Français de la Sclérose en Plaques (OFSEP) patients with MS clinical onset over 1960-2014, and aged ⩾25 years at MS onset were included.

Raising the bar in ulcerative colitis management.

Ulcerative colitis (UC) is a chronic inflammatory bowel disease characterized by growing incidence and prevalence around the world in the last few decades. The range of available existing treatment and strategies for its management is being implemented. Given the introduction of newly developed molecules and the lack of specific guidelines, drug positioning may represent a tough clinical challenge.

Development and Validation of a Sexual Quality of Life Score for Youths with Inflammatory Bowel Disease.

Background And Aims: Inflammatory Bowel Disease (IBD) are known to impact patients 'sexual lives. The aim of this study is to create and validate a sexual QoL designed for youth with IBD.

Methods: This study was conducted in two phases: development of the score and validation of the items.

Patient Safety and Perception of Quality in University Dental Hospitals: A French National Survey.

Objectives: Patient safety is poorly developed in dentistry. The aim of this study was to evaluate the level of patient safety perception and quality culture in French university dental hospitals.

Methods: A national survey was performed using a questionnaire that was sent electronically to dental students, teachers, senior professionals, and paramedics of the university dental clinics that volunteered to participate.