Proximal protection devices for carotid artery stenting - A benchtop assessment of flow reversal performance.

Background And Purpose: Proximal protection devices, such as TransCarotid Artery Revascularization (TCAR, SilkRoad Medical, Sunnyvale), aim to yield better outcomes in carotid artery stenting (CAS) than distal protection devices by preventing plaque embolization to the brain. However, transfemoral catheters may not fully reverse flow from the external carotid artery (ECA) to the internal carotid artery (ICA). We assess a new balloon-sheath device, Femoral Flow Reversal Access for Carotid Artery Stenting (FFRACAS), for this purpose.

Impact of Sjögren's disease on quality of sexual life using the Qualisex score.

Objective: This study aimed to assess the impact of Sjögren disease (SjD) on the quality of sexual life and its determinants using the Qualisex questionnaire.

Methods: The Qualisex questionnaire was administered to participants within the ASSESS cohort, a French national multicentric prospective cohort of individuals with SjD. Patients' characteristics and psychometric evaluations were also collected.

Impact of frailty and older age on weaning from invasive ventilation: a secondary analysis of the WEAN SAFE study.

Objective: To understand the impact of both frailty and chronologic age on outcomes of weaning from invasive mechanical ventilation (MV).

Methods: The study population consisted of patients enrolled in the 'WorldwidE. AssessmeNt of Separation of pAtients From ventilatory assistancE (WEAN SAFE) study.

A multicenter Phase II randomized, placebo-controlled single-blind trial with the SV2A ligand seletracetam in photosensitive epilepsy patients.

The objective of this study was to evaluate the effect of seletracetam (SEL), a potent modulator of synaptic vesicle glycoprotein 2A (SV2A), in patients with photoparoxysmal EEG response (PPR) to intermittent photic stimulation (IPS) as proof-of-principle of efficacy in patients with epilepsy. In this multicenter, single-blind Phase II study, adults with photosensitive epilepsy, with/without concomitant antiseizure medication therapy, underwent IPS under 3 eye conditions (at eye closure, eyes closed and eyes open) after a single oral dose of placebo (day - 1) or SEL (day 1; 0.5, 1, 2, 4, 10, or 20 mg).

Inter-Rater Disagreements in Applying the Montreal Classification for Crohn's Disease: The Five-Nations Survey Study.

Background: The Montreal classification has been widely used in Crohn's disease since 2005 to categorize patients by the age of onset (A), disease location (L), behavior (B), and upper gastrointestinal tract and perianal involvement. With evolving management paradigms in Crohn's disease, we aimed to assess the performance of gastroenterologists in applying the Montreal classification.

Methods: An online survey was conducted among participants at an international educational conference on inflammatory bowel diseases.

Inflammatory Potential of the Diet and Risk of Crohn's Disease and Ulcerative Colitis.

Background: Association between dietary factors and the risk of developing inflammatory bowel disease (IBD) has been studied extensively. However, identification of deleterious dietary patterns merits further study.

Aim: To investigate the risk of developing Crohn's disease (CD) and ulcerative colitis (UC) according to the inflammatory score of the diet (ISD) in the multinational European Prospective Investigation into Cancer and Nutrition (EPIC) cohort.

Clinical practice recommendations for the diagnosis and management of X-linked hypophosphataemia.

X-linked hypophosphataemia (XLH) is a rare metabolic bone disorder caused by pathogenic variants in the PHEX gene, which is predominantly expressed in osteoblasts, osteocytes and odontoblasts. XLH is characterized by increased synthesis of the bone-derived phosphaturic hormone fibroblast growth factor 23 (FGF23), which results in renal phosphate wasting with consecutive hypophosphataemia, rickets, osteomalacia, disproportionate short stature, oral manifestations, pseudofractures, craniosynostosis, enthesopathies and osteoarthritis. Patients with XLH should be provided with multidisciplinary care organized by a metabolic bone expert.

FRED-EPI study: Safety and efficacy of FRED/FRED Jr aneurysm treatment in current clinical practice.

Objective: Flow diversion is increasingly used as an endovascular treatment for intracranial aneurysms. FRED-EPI is a prospective, multicenter, French study, conducted to analyze the safety and efficacy of aneurysm treatment with FRED/FRED Jr (Microvention, AlisoViejo, CA, USA) in current clinical practice.

Patients And Methods: Patients with intracranial aneurysms treated with FRED and FRED Jr who agreed to participate were prospectively and consecutively included in all French centers using these devices.

Outcomes and challenges in adolescents living with HIV in France: a nationwide cohort study over 35 years (1985-2020).

Objective: Most data published on adolescents living with HIV (ALH) have been collected before the large diffusion of second-generation integrase strand transfer inhibitors (INSTI) among the pediatric population. We analyzed the nationwide ANRS-MIE CO10 Pediatric cohort to assess the changes over time in health and social outcomes of French ALH.

Design: The cohort enrolled children born in France since 1985 and, from 2005, children diagnosed with HIV at ≤13 years, including those born abroad if antiretroviral-naive at first medical care in France.

Association of a remote monitoring programme with all-cause mortality and hospitalizations in patients with heart failure: National-scale, real-world evidence from a 3-year propensity score analysis of the TELESAT-HF study.

Aims: To examine the association of a remote monitoring programme (RMP) with all-cause mortality and hospital admissions for heart failure (HF) within the French healthcare system.

Methods And Results: A national-scale, real-world, propensity-weighted cohort study was conducted using the SNDS French database from August 2018 to December 2022 (NCT06312501). Patients receiving standard of care (SoC) were compared with those receiving RMP (Satelia® Cardio, NP Medical).

Harnessing the tumor microenvironment: targeted cancer therapies through modulation of epithelial-mesenchymal transition.

The tumor microenvironment (TME) is integral to cancer progression, impacting metastasis and treatment response. It consists of diverse cell types, extracellular matrix components, and signaling molecules that interact to promote tumor growth and therapeutic resistance. Elucidating the intricate interactions between cancer cells and the TME is crucial in understanding cancer progression and therapeutic challenges.

Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.

Importance: There is a lack of long-term efficacy and safety data on hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) and on RNA interference (RNAi) therapeutics in general. This study presents the longest-term data to date on patisiran for hATTR-PN.

Objective: To present the long-term efficacy and safety of patisiran in adults with hATTR-PN.

Structural variation in nebulin and its implications on phenotype and inheritance: establishing a dominant distal phenotype caused by large deletions.

Introduction: Structural variants (SVs) of the nebulin gene (), including intragenic duplications, deletions, and copy number variation of the triplicate region, are an established cause of recessively inherited nemaline myopathies and related neuromuscular disorders. Large deletions have been shown to cause dominantly inherited distal myopathies. Here we provide an overview of 35 families with muscle disorders caused by such SVs in .

Minimal invasive biopsies are highly sensitive for amyloid detection in hereditary transthyretin amyloidosis with polyneuropathy.

Objective: To assess the effectiveness of labial minor salivary gland biopsy (LSGB) alone or in combination with punch skin biopsy (SB) for the detection of amyloid deposits in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).

Methods: In this single-center retrospective study, Congo red staining of minimal invasive LSGB (4 mm) and SB (3 mm) was assessed in ATTRv-PN patients consecutively evaluated between 2012 and 2023.

Results: Histopathological data of 171 ATTRv-PN, including 49 early-onset p.

Usefulness of Cerebrospinal Fluid Alzheimer's disease biomarkers in older patients: Evidence from a national multicenter prospective study.

Background: The use of cerebrospinal (CSF) biomarkers in the diagnosis of Alzheimer's disease (AD) has been gaining interest in clinical practice. Although their usefulness has been demonstrated, their potential value in older patients remains debated.

Objectives: To assess whether knowledge of the results of CSF AD biomarkers was associated with the same gain in diagnostic confidence in older adults > 80 than in younger patients.

Microsporidiosis in Patients with Autoimmune Diseases Undergoing Monoclonal Antibody Associated Therapy.

We present Enterocytozoon bieneusi infection in four patients with autoimmune diseases undergoing prolonged monoclonal antibody therapies. Two patients suffered from inflammatory bowel disease and received anti-TNF therapies, whereas two other patients suffered from systemic lupus erythematosus with renal involvement and received anti-CD20 or anti-BLyS protein therapies. Three out of four patients consulted for diarrhea with abdominal pain without intestinal inflammation or bleeding at the time of sampling.

Comprehensive exploration of unexplained dyspnoea in subjects with normal ejection fraction and low natriuretic peptides.

Background: Unexplained exertional dyspnoea without significant elevation of natriuretic peptides is common. One of the causes might be early heart failure with preserved ejection fraction (HFpEF).

Aims: This study aimed to characterize patients with exertional dyspnoea and normal/near-to-normal N-terminal pro-brain natriuretic peptide (NT-proBNP) levels with regard to early stages of HFpEF and non-cardiac causes.

Efficacy and safety of the PHIL embolic agent in the treatment of intracranial dural arteriovenous fistulas: results of the PHIL-dAVF study.

Background And Purpose: Embolization is the first-line treatment for dural arteriovenous fistulas (dAVF). The precipitating hydrophobic injectable liquid (PHIL) embolic agent is a non-adhesive copolymer with specific features and endovascular behavior. This study assessed its safety and efficacy in a prospective real-life cohort.

High-Dose Cyproterone Acetate and Intracranial Meningioma: Impact of the Risk Minimisation Measures Implemented in France in 2018-2019.

Purpose: To measure the impact of national regulatory actions implemented in France in August 2018 and June 2019 to reduce the risk of meningioma associated with the use of cyproterone acetate (CPA).

Methods: Using the French National Healthcare database, we calculated the monthly number of CPA users among cisgender women, men and transgender women in 2010-2021, the monthly proportion of users with cerebral imaging screening, and the annual rate of meningioma surgery associated with CPA use. CPA discontinuations and switches were analysed.

PDE11A is a phenotype modulator of Primary Bilateral Macronodular Adrenal Hyperplasia: results of a 334 patients' series.

Purpose: Primary bilateral macronodular adrenal hyperplasia (PBMAH), the most common cause of Cushing's syndrome due to bilateral nodules, is a heterogeneous disease at the clinical, hormonal and morphological levels. ARMC5 inactivating pathogenic variants are causative of PBMAH and rare variants of PDE11A have been associated with PBMAH.

Methods: Leukocyte DNA of 354 PBMAH index cases was sequenced for ARMC5 and PDE11A genes by Next generation sequencing (NGS).

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.

National Cohort of Compassionate Use of Meropenem-Vaborbactam: No Benefit over Meropenem for .

Background: Meropenem-vaborbactam (MEM-VAB) is a novel carbapenem-beta-lactamase-inhibitor combination that demonstrates activity against carbapenem-resistant (CR) Gram-negative bacteria, and more specifically KPC-producers, since vaborbactam is an effective inhibitor of KPC enzymes in vitro. This study aimed to describe the initial uses and efficacy of MEM-VAB for compassionate treatment during the first 21 months following its early access in France.

Method: A national multicenter retrospective study was conducted, including all patients who received at least one dose of MEM-VAB between 20 July 2020, and 5 April 2022.