Nusinersen treatment in adults with severe spinal muscular atrophy: A real-life retrospective observational cohort study.

Background And Objectives: Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease due to homozygous loss-of-function of the survival motor neuron gene SMN1 with absence of the functional SMN protein. Nusinersen, a costly intrathecally administered drug approved in 2017 in Europe, induces alternative splicing of the SMN2 gene, which then produces functional SMN protein, whose amount generally increases with the number of SMN2 gene copies.

Methods: We retrospectively collected data from consecutive wheelchair-bound adults with SMA managed at a single center in 2018-2020.

Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic.

Background And Purpose: Data on interruption of enzyme replacement therapy (ERT) are scarce in late onset Pompe disease. Due to the COVID-19 crisis, eight neuromuscular reference centers in France were obligated to stop the treatment for 31 patients.

Methods: We collected the motor and respiratory data from our French registry, before COVID-19 and at treatment restart.

Smoking and obesity among long-haul truck drivers in France.

Introduction: This study examines the health status of long-haul truck drivers (LHTDs) and more specifically smoking and obesity, in France.

Methods: A total of 373 French and other LHTDs were randomly interviewed at six highway rest stops. Variables recorded were self-reported sociodemographic characteristics (age, gender, marital status, and nationality), behavioral variables (consumption of sodas, daily exercise, smoking status), weight and height.

Factors Associated With Treatment Failure in Moderately Severe Community-Acquired Pneumonia: A Secondary Analysis of a Randomized Clinical Trial.

Importance: Failure of treatment is the most serious complication in community-acquired pneumonia (CAP).

Objective: To assess the potential risk factors for treatment failure in clinically stable patients with CAP.

Design, Setting, And Participants: This secondary analysis assesses data from a randomized clinical trial on CAP (Pneumonia Short Treatment [PTC] trial) conducted from December 19, 2013, to February 1, 2018.

Bench testing of tracheostomy tube-related insults using an instrumented manikin.

Purpose: Choosing the right tracheal tube for the right patient is a daily preoccupation for intensivists and emergency physicians. Tracheal tubes can generate severe complications, which are chiefly due to the pressures applied by the tube to the trachea. We designed a bench study to assess the frequency of pressure levels likely to cause tracheal injury.

Post-operative hallux varus: a review of treatment methods.

Purpose: Hallux varus is a classical complication of hallux valgus surgery with a medium rate of 6%.

Material And Methods: Methods of treatment are MTP1 fusion or conservative joint operations. Hallux varus results from imbalance between excessive medial capsule retraction or tensioning and excessive lateral laxity or soft-tissue release but also and mainly from an over displacement of the first metatarsal which reduces the intermetatarsal angle and thus leads to medial displacement of the great toe.

Biomechanical study comparing the energy cost of human bipedalism versus zenkutsu-dachi stepping of a karateka.

Traditional stepping in the zenkutsu-dachi stance of the Shotokan style of karate is very physically demanding. It requires considerably more effort than what is expended during conventional human bipedalism. We performed a biomechanical study to analyze and compare these two types of gaits when performed by a highly experienced karateka.

International retrospective natural history study of -related congenital muscular dystrophy.

Muscular dystrophies due to heterozygous pathogenic variants in gene cover a broad spectrum of clinical presentations and severity with an age of onset ranging from the neonatal period to adulthood. The natural history of these conditions is not well defined, particularly in patients with congenital or early onset who arguably present with the highest disease burden. Thus the definition of natural history endpoints along with clinically revelant outcome measures is essential to establishing both clinical care planning and clinical trial readiness for this patient group.

Bone mineral density at extremely low weight in patients with anorexia nervosa.

Objective: Low bone mineral density (BMD) is a frequent and invalidating consequence of chronic undernourishment in patients with anorexia nervosa (AN). The aim of this study was to assess prevalence and clinic-biological correlates of low BMD and fractures in extremely undernourished inpatients with AN.

Design: Retrospective cohort study.

Dalbavancin treatment for prosthetic joint infections in real-life: a national cohort study and literature review.

Objectives: Dalbavancin is a long-lasting lipoglycopeptide active against Gram-positive bacteria, especially methicillin-resistant staphylococci. Few data are available on dalbavancin use for treatment of prosthetic joint infections (PJIs). We describe a cohort of patients treated for PJI with dalbavancin and review the literature regarding this condition.

Mortality and Neurologic Sequelae in Influenza-Associated Encephalopathy: Retrospective Multicenter PICU Cohort in France.

Objectives: To describe and estimate the mortality rate of severe influenza-associated encephalopathy/encephalitis among children admitted to PICUs.

Design: Multicenter retrospective study.

Setting: Twelve French PICUs.

Treatment of bone and joint infections by ceftazidime/avibactam and ceftolozane/tazobactam: a cohort study.

Objectives: Ceftazidime/avibactam (C/A) and ceftolozane/tazobactam (C/T) are two novel antibacterials with known efficacy against Gram-negative bacteria (GNB). We aimed to describe the efficacy and safety of surgical management combined with C/A or C/T treatment for bone and joint infections (BJIs).

Methods: We conducted an observational, bicentric study of patients treated with C/A or C/T for a BJI between May 2016 and June 2019.

Pediatric Acute Alcohol Intoxication Admitted to the ICU: A Review of 102 Cases in France.

Objectives: To describe the profile and clinical outcomes of children (<18 yr) admitted to intensive care for acute alcohol intoxication, with special attention to complications and to the subgroup that required intubation.

Design: Retrospective observational study.

Setting: Seven pediatric and three adult ICUs in France.

Discontinuing β-lactam treatment after 3 days for patients with community-acquired pneumonia in non-critical care wards (PTC): a double-blind, randomised, placebo-controlled, non-inferiority trial.

Background: Shortening the duration of antibiotic therapy for patients admitted to hospital with community-acquired pneumonia should help reduce antibiotic consumption and thus bacterial resistance, adverse events, and related costs. We aimed to assess the need for an additional 5-day course of β-lactam therapy among patients with community-acquired pneumonia who were stable after 3 days of treatment.

Methods: We did this double-blind, randomised, placebo-controlled, non-inferiority trial (the Pneumonia Short Treatment [PTC]) in 16 centres in France.

Bloodstream infections in older population: epidemiology, outcome, and impact of multidrug resistance.

Bloodstream infections (BSIs) among older patients are frequent with high rate of mortality. Infections with multidrug-resistant organisms (MDRO) are associated with higher mortality than with susceptible microorganisms. We aimed to evaluate the prevalence of MDRO infection during BSI in older population and the factors associated with unfavorable outcome.

Susceptibility Testing Is Key for the Success of Cefiderocol Treatment: A Retrospective Cohort Study.

Cefiderocol is a novel siderophore cephalosporin, which has proven in vitro activity against carbapenem-resistant (CR) Gram-negative pathogens and stability towards all carbapenemases. The aim of this study was to describe the first cases of prescriptions and the efficacy of cefiderocol for compassionate use in the 2 months following its access in France. We performed a national retrospective study of all patients who received at least one dose of cefiderocol from 2 November 2018 to 5 November 2019.

Clinical Cutaneous Features of Patients Infected With SARS-CoV-2 Hospitalized for Pneumonia: A Cross-sectional Study.

Background: Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is the cause of a current pandemic worldwide. This virus can reach all organs and disturbs the immune system, leading to a cytokine storm in severe forms. We aimed to report cutaneous features among coronavirus disease 2019 (COVID-19) hospitalized patients.

A strange New Year's Eve: triggers in Kleine-Levin syndrome.

Kleine-Levin syndrome is a rare neurological disease of unknown cause beginning typically during adolescence, characterized by remittent-relapsing episodes of severe hypersomnia associated with cognitive and behavioral disturbances. Triggering factors at Kleine-Levin syndrome onset include infection, sleep deprivation, as well as alcohol, drug, and substance intake. A young woman had 6 episodes over 2 years, including hypersomnia, confusion, derealization, cognitive impairment, anxiety, feeling of being scrutinized, anorexia (and sweet craving once) but no hypersexuality.