Impaired Exercise Capacity in High-Risk Diabetic Cardiomyopathy: The ARISE-HF Cardiopulmonary Exercise Testing Subanalysis.

Background: Objective indices of functional capacity in patients with diabetic cardiomyopathy and stage B heart failure (HF) have not been comprehensively defined. We sought to characterize the cardiopulmonary exercise characteristics of individuals with diabetic cardiomyopathy at high risk for overt HF.

Methods: The relationships from cardiopulmonary exercise testing with clinical and laboratory characteristics of participants with diabetic cardiomyopathy were evaluated using baseline data from the ARISE-HF trial (Aldose Reductase Inhibition for Stabilization of Exercise Capacity in Heart Failure).

Single cell profiling of circulating autoreactive CD4 T cells from patients with autoimmune liver diseases suggests tissue imprinting.

Autoimmune liver diseases (AILD) involve dysregulated CD4 T cell responses against liver self-antigens, but how these autoreactive T cells relate to liver tissue pathology remains unclear. Here we perform single-cell transcriptomic and T cell receptor analyses of circulating, self-antigen-specific CD4 T cells from patients with AILD and identify a subset of liver-autoreactive CD4 T cells with a distinct B-helper transcriptional profile characterized by PD-1, TIGIT and HLA-DR expression. These cells share clonal relationships with expanded intrahepatic T cells and exhibit transcriptional signatures overlapping with tissue-resident T cells in chronically inflamed environments.

Comparison of two strategies of glucocorticoid withdrawal in patients with rheumatoid arthritis in low disease activity (STAR): a randomised, placebo- controlled, double-blind trial.

Objectives: To compare two strategies-a hydrocortisone replacement strategy and a prednisone tapering strategy-for their success in glucocorticoid discontinuation in patients with rheumatoid arthritis (RA) with low disease activity (LDA).

Methods: The Strategies for glucocorticoid TApering in Rheumatoid arthritis (STAR) study was a double- blind, double-placebo randomised controlled trial including patients with RA receiving a stable dose of glucocorticoid 5 mg/day for ≥3 months and were in LDA for ≥3 months. Patients were randomly assigned in a 1:1 ratio to either replace prednisone with 20 mg/day of hydrocortisone for 3 months, then reduce to 10 mg/day for 3 months before discontinuation or to taper prednisone by 1 mg/day every month until complete discontinuation, contingent on maintaining LDA.

Near-universal prevalence of central adiposity in heart failure with preserved ejection fraction: the PARAGON-HF trial.

Background And Aims: An expansion of fat mass is an integral feature of patients with heart failure and preserved ejection fraction (HFpEF). While body mass index (BMI) is the most common anthropometric measure, a measure of central adiposity-the waist-to-height ratio (WHtR)-focuses on body fat content and distribution; is not distorted by bone or muscle mass, sex, or ethnicity; and may be particularly relevant in HFpEF.

Methods: The PARAGON-HF trial randomized 4796 patients with heart failure and ejection fraction ≥45% to valsartan or sacubitril/valsartan.

The nucleolin antagonist N6L and paclitaxel combination treatment could be a new promising therapeutic strategy for pancreatic ductal adenocarcinoma therapy.

Pancreatic cancer (PCa) is one of the most devastating cancers with few clinical signs and no truly effective therapy. In recent years, our team has demonstrated that nucleolin antagonists such as N6L could be a therapeutic alternative for this disease. In order to study a possible clinic development of N6L (multivalent pseudopeptide), we undertook to study the effect of combination of N6L with chemotherapies classically used for PCa on the survival of pancreatic cancer cells.

Occurrence and management of thrombosis recurrence and bleeding in low-molecular-weight heparin-treated patients with cancer-associated thrombosis: a French nationwide cohort study.

Background: Rates of venous thromboembolism (VTE) recurrence and bleeding remain high in patients with cancer who are prescribed anticoagulants (ACs) such as low-molecular-weight heparin (LMWH) after an initial VTE event.

Objectives: To identify patient characteristics associated with VTE recurrence and bleeding in patients receiving LMWH for cancer-associated VTE and to explore secondary AC management and clinical outcomes in these patients.

Methods: An observational study was conducted using nationwide French data for adults with active cancer who were hospitalized with VTE in 2013-2018 and were reimbursed for LMWH ≤ 30 days after hospital discharge.

Identification of the conditions and minimum requirements necessary for the release of autologous fresh CAR T-cell products under hospital exemption: a position paper from the WP-bioproduction of the UNITC consortium.

The accessibility of CAR-T cells in centralized production models faces significant challenges, primarily stemming from logistical complexities and prohibitive costs. However, European Regulation EC No. 1394/2007 introduced a pivotal provision known as the hospital exemption.

Communicating scientific evidence: drugs for Alzheimer's disease as a case study.

This paper reviews the scientific evidence on new anti-amyloid monoclonal antibodies for treating Alzheimer's disease as a case study for improving scientific evidence communication. We introduce five guidelines condensed from the biomedical evidence literature but adapted to the short format of science communication in e.g.

Effectiveness and safety of biosimilars in pediatric inflammatory bowel diseases: an observational longitudinal study on the French National Health Data System.

Background: Data on biosimilar use in pediatric inflammatory bowel diseases (IBD) are scarce compared to the status of studies in adults, resulting in limitations in its treatment. We compared effectiveness and safety of biosimilars versus originators in this population.

Methods: We used data from the French National Health Data System to identify children (less than 18 years old at treatment initiation) initiating treatment with a biosimilar or the originator infliximab or adalimumab for Crohn's disease (CD) or ulcerative colitis (UC), from first biosimilar launch (January 2015 and October 2018, respectively) to 31 December 2022.

In vivo Safety and Feasibility of a Computed Tomography-Guided Robotic Device for Percutaneous Needle Placement in Bone.

Purpose: To evaluate the feasibility and accuracy of a robotic device used clinically in soft tissues (abdomen and lung), modified in design and workflow, to perform needle insertion in percutaneous bone procedures.

Methods: The primary objective was safety (severe complications) of robotic-assisted insertion in this new application. Secondary objectives were feasibility (placement technical success), performance (acceptable insertions rate), accuracy (lateral deviation), number of intermediate CT-scans and tolerance (minor/moderate complications).

Patient satisfaction with radio-frequency identification (RFID) tag localization compared with wire localization for nonpalpable breast lesions: the RFID trial.

Background: Most breast cancers are detected at an early stage in which case conservative surgery is indicated. An accurate preoperative localization technique is essential for conservative surgery of non-palpable breast lesions. Currently, the gold standard technique is wire localization (WL).

Data management and sharing.

Guided by the FAIR principles (Findable, Accessible, Interoperable, Reusable), responsible data sharing requires well-organized, high-quality datasets. However, researchers often struggle with implementing Data Management and Sharing Plans (DMSPs) due to lack of knowledge on how to do this, time constraints, legal, technical and financial challenges, particularly concerning data ownership and privacy. While patients support data sharing, researchers and funders may hesitate, fearing the loss of intellectual property or competitive advantage.

Low-dose aspirin versus placebo in postpartum venous thromboembolism: a multi-national, pilot, randomised, placebo-controlled trial.

Background: Despite the morbidity and mortality of venous thromboembolism, there is little evidence to guide postpartum thromboprophylaxis in patients at moderate risk. We aimed to assess the feasibility of conducting a double-blind, randomised trial of aspirin versus placebo in postpartum individuals with two or more venous thromboembolism risk factors, mild-to-moderate thrombophilia, or both.

Methods: The pilot PARTUM trial, a multi-national, randomised, double-blind, placebo-controlled trial, was conducted in seven centres across Canada, France, Ireland, and the Netherlands.

Beta-variant recombinant SARS CoV-2 vaccine induces durable cross-reactive antibodies against Omicron BA variants.

Background: We previously reported the safety and immunogenicity data from a randomized trial comparing the booster responses of vaccinees who received monovalent (MV) recombinant protein Beta-variant (MVB.1.351) and MV ancestral protein (MVD614) vaccines with AS03 adjuvant (Sanofi/GSK) to booster response of vaccinees who received mRNA MV ancestral strain BNT162b2 vaccine (Pfizer-BioNTech).

Incomplete reporting of adverse events in duloxetine trials: a meta-research survey of randomized controlled trials versus placebo.

Objective: Relying on published data alone might be insufficient for meta-analyses to be reliable and trustworthy since selective outcome reporting is common, especially for adverse events. We investigated the existence of selective reporting and its potential for bias in a case study exploring adverse events of duloxetine in adults.

Study Design And Setting: We systematically searched all previous meta-analyses/pooled analyses on duloxetine published on PubMed for 7 indications approved by the American and European health authorities.

Perifoveal vascular anomalous complex and telangiectatic capillaries: An overview of two entities potentially sharing a common pathophysiology.

Focal capillary ectasia in the macular region can manifest in distinct clinical scenarios, which can be categorized into 2 main entities: perifoveal vascular anomalous complex (PVAC) and telangiectatic capillaries (TelCaps). PVAC represents a primary, idiopathic condition, whereas TelCaps occur secondary to underlying vascular disorders, including diabetic macular edema and retinal vein occlusion. We provide a comprehensive analysis of these 2 entities, encompassing their clinical presentations, multimodal imaging findings, histological evidence, and differential diagnosis from other retinal microvascular abnormalities, such as Type 1 macular telangiectasia, adult-onset Coats disease, Type 3 macular neovascularization in age-related macular degeneration, and retinal arterial macroaneurysms.

Perspectives on the use of decellularized/devitalized and lyophilized human perinatal tissues for bone repair: Advantages and remaining challenges.

Human amniotic membrane (hAM) has been extensively used for several decades as a bioactive scaffold for regenerative medicine. In its cryopreserved form-one of the main storage formats-the presence of viable cells has often been questioned. Furthermore, there is little published evidence of the role of endogenous amniotic cells from cryopreserved hAM in tissue repair.

A phase II double-blind multicentre, placebo-controlled trial to assess the efficacy and safety of alpelisib (BYL719) in paediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP): the SESAM study protocol.

Introduction: The megalencephaly capillary malformation polymicrogyria (MCAP syndrome) results from mosaic gain-of-function variants. The main clinical features are macrocephaly, somatic overgrowth, neurodevelopmental delay and brain anomalies. Alpelisib (Vijoice) is a recently FDA-approved PI3Kα-specific inhibitor for patients with PIK3CA-related overgrowth spectrum (PROS).

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.

Background And Objectives: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.

Cathinone metabolism and biliary excretion in an ex-vivo pig liver model: Example of 4-Cl-PVP and eutylone.

Objective: Recently, the pig liver model perfused ex vivo using a normothermic machine perfusion (NMP) has been proposed as a suitable model to study xenobiotic metabolism and biliary excretion. The aim of our study is to describe the metabolism of NPS such as cathinones (with a focus on 4-Cl-PVP and eutylone) in blood and bile, using a normothermic perfused pig liver model.

Methods: Livers (n = 4) from male large white pigs, 3-4 months of age and weighing approximately 75-80 kg, were harvested and reperfused onto an NMP (LiverAssist®, XVIVO) using autologous whole blood at 38 °C.

Prevalence rate of high-grade cytological lesions of the cervix in women aged under 25 years in French Guiana.

Introduction: The incidence of cervical cancer in French Guiana is among the highest worldwide. It ranks 5th among all cancers and is the 2nd most common cancer in women after breast cancer. The primary objective of this study was to establish the proportion of high-grade cytological lesions of the cervix in women under 25 years of age who underwent a cervico-uterine smear (CUS) in French Guiana over a 10-year period.

Efficacy of a vein visualisation device for facilitating peripheral venous line placement in adult patients with sickle cell disease: A randomised clinical trial.

Background: Intravenous (IV) access is often required for the treatment of vaso-occlusive crises in patients with sickle cell disease, but can be particularly challenging due to recurrent venous damage. The AccuVein® device, uses near-infrared light technology to visualise veins for easier venepuncture.

Methods: A randomised, controlled trial of the efficacy of the AccuVeinAV400® device in the replacement of peripheral venous lines during a vaso-occlusive crisis was conducted at two centres in France.

Intracerebroventricular anaerobic dopamine in Parkinson's disease with L-dopa-related complications: a phase 1/2 randomized-controlled trial.

Continuous compensation for cerebral dopamine deficiency represents an ideal treatment for Parkinson's disease. Dopamine does not cross the digestive and blood-brain barriers and is rapidly oxidized. The new concept is the intracerebroventricular administration of anaerobic dopamine (A-dopamine) using an abdominal pump connected to a subcutaneous catheter implanted in the third ventricle, near the striatum.