Evaluation of professional practices in the use of mexiletine for the management of childhood myotonia in French pediatric neuromuscular centers (MEXI-PEDI survey).

Background: Myotonia is the main feature of both myotonic dystrophy (DM) and non-dystrophic myotonia (NDM). It is felt as stiffness, pain, fatigue, and weakness. In France, mexiletine, a non-selective voltage-gated sodium channel blocker, is approved for the treatment of myotonia in adults with NDM, and it has a temporary recommendation for use in the symptomatic treatment of DM in adults.

Pseudotumoural optic neuritis in myelin oligodendrocyte glycoprotein antibody-associated disease in children: Pseudotumoural optic neuritis in MOG antibody-associated disease.

Myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOGAD) is a neuro-inflammatory condition affecting adults and children. The presentations vary and include acute disseminated encephalomyelitis, optic neuritis or transverse myelitis. Optic neuritis associated with anti-MOG antibodies is typically bilateral, anterior and initially severe but usually resolves quickly and completely.

From pediatric emergency department to pediatric intensive care unit: a retrospective study in a French Tertiary University hospital.

Objective: The management of a child presenting with a critical medical or surgical condition is a scarce event in the pediatric emergency department (PED). In this one year retrospective study, we have tried to better characterize the profile and care pathway of children who had been transferred to the neonatal or pediatric intensive care or critical care units (PICCU) after a visit to the PED, or died in PED.

Methods: Retrospective study of children who has been transferred to PICCU from the two PED of Marseille's University Hospital from the 1 of January 2022 until the 31 of December 2022.

Primary adrenal insufficiency in children excluding congenital adrenal hyperplasia: insights from 33-year single-center experience in Tunisia.

Background: Primary adrenal insufficiency (PAI) is a rare but potentially life-threatening condition. Congenital adrenal hyperplasia (CAH) is the most common cause of PAI in children. To date, numerous non-CAH causes have been identified through genetic analysis but they remain poorly characterized.

Food protein induced enterocolitis syndrome: French practices assessment in children.

Background: Food protein-induced enterocolitis syndrome (FPIES) is a specific non IgE-mediated food allergy. The international consensus guidelines defined diagnosis criteria and management plan in 2017.

Objectives: To assess practices regarding FPIES in France and in French-speaking countries, according to those guidelines.

Intersectoral professionals' understanding of the prevention of sexual violence against children, their role and that of the GP: A study using semi-structured interviews: Prevention of sexual violence against children.

Background: In 2020, the WHO reported a European prevalence of 9.6 % of sexual abuse among children, and called on every country to improve prevention of such violence.

Objective: To explore the understanding of an intersectional sample of professionals of their role and that of the general practitioner (GP) in the primary and secondary prevention of sexual violence against children (SVAC).

Early osteopathic manipulative treatment to prevent cranial positional deformities: A randomized controlled trial.

Background: Cranial positional deformities occur frequently in the first months of life.

Objective: To evaluate the effectiveness of osteopathic manipulative treatment (OMT) in preventing the onset of positional plagiocephaly and brachycephaly at 4 months in at-risk newborns.

Methods: This single-center, randomized, controlled open-label study, with blinded assessment of the endpoints, was carried out at the maternity unit of Montpellier University Hospital (France) between 2019 and 2022.

The role of nutritional vitamin D in CKD-MBD in children and adults with CKD, on dialysis and after kidney transplantation - a European consensus statement.

Vitamin D deficiency is common in patients with chronic kidney disease (CKD) and associates with poor outcomes. Current clinical practice guidelines recommend supplementation with nutritional vitamin D as for the general population. However, recent large-scale, clinical trials in the general population failed to demonstrate a benefit of vitamin D supplementation on skeletal or non-skeletal outcomes, fueling a debate on the rationale for screening for and correcting vitamin D deficiency, both in non-CKD and CKD populations.

Sequential and Time-Controlled Sol-Gel Transitions by Mechanical Switching of Molecular Tweezers.

Controlling the motion of molecular machines to influence higher-order structures is well-established in biological systems but remains a significant challenge for synthetic analogs. Herein, we aim to harness the mechanical switching of switchable molecular tweezers to modulate their self-assembly and produce stimuli-responsive organogels. We report a series of terpy(Pt-salphen) molecular tweezers functionalized with alkyl chains that act as low-molecular-weight gelators (LMWGs) in their open conformation.

Healthy Lifestyle in the First 1000 Days and Overweight and Obesity Throughout Childhood.

Objectives: Investigate associations of different family healthy lifestyle scores (HLS) during the first 1000 days with childhood overweight and obesity (OWOB).

Methods: Cohort-specific analyses were conducted on participants (n = 25 006) from 4 European birth cohorts (The study on the pre- and early postnatal determinants of child health and development [EDEN], Elfe, France; Generation R, the Netherlands; and Lifeways, Ireland). Three composite HLSs were calculated: a maternal pregnancy HLS based on prepregnancy body mass index (BMI) and diet quality, physical activity, smoking status, and alcohol consumption during pregnancy; a parental pregnancy HLS additionally considering paternal BMI and smoking status; and an infancy HLS based on breastfeeding duration, age of solid food introduction, and exposure to passive smoking.

EXPLORATORY STRUCTURAL NEUROIMAGING EXAMINATION OF IMPULSIVITY IN SEVERE ALCOHOL USE DISORDER: PERSISTENT IMPLICATION OF THE VENTRAL STRIATUM.

Background: While Alcohol Use Disorder (AUD) is frequently associated with impulsivity, its structural brain substrates are still poorly defined. The triadic model of addiction postulates that impulsive behavior is regulated by an amygdalo-striatal impulsive subcomponent, a prefrontal and cerebellar reflective subcomponent, and an insular regulatory subcomponent. The objective of this study was thus to examine the relationships between self-evaluated impulsivity and structural brain abnormalities in patients with severe AUD (sAUD) using the triadic model as a theoretical framework.

Effects of blood-brain barrier opening using ultrasound on tauopathies: A systematic review.

Unlabelled: Blood-brain barrier opening with ultrasound can potentiate drug efficacy in the treatment of brain pathologies and also provides therapeutic effects on its own. It is an innovative tool to transiently, repeatedly and safely open the barrier, with studies showing beneficial effects in both preclinical models for Alzheimer's disease and recent clinical studies. The first preclinical and clinical work has mainly shown a decrease in amyloid burden in mice models and in patients.

Brain inflammation and cognitive decline induced by spinal cord injury can be reversed by spinal cord cell transplants.

Spinal cord injuries (SCIs) impact between 250,000 and 500,000 people worldwide annually, often resulting from road accidents or falls. These injuries frequently lead to lasting disabilities, with the severity depending on the injury's extent and location. Emerging research also links SCIs to cognitive impairments due to brain inflammation.

Nivolumab plus ipilimumab versus nivolumab in microsatellite instability-high metastatic colorectal cancer (CheckMate 8HW): a randomised, open-label, phase 3 trial.

Background: CheckMate 8HW prespecified dual primary endpoints, assessed in patients with centrally confirmed microsatellite instability-high or mismatch repair-deficient status: progression-free survival with nivolumab plus ipilimumab compared with chemotherapy as first-line therapy and progression-free survival with nivolumab plus ipilimumab compared with nivolumab alone, regardless of previous systemic treatment for metastatic disease. In our previous report, nivolumab plus ipilimumab showed superior progression-free survival versus chemotherapy in first-line microsatellite instability-high or mismatch repair-deficient metastatic colorectal cancer in the CheckMate 8HW trial. Here, we report results from the prespecified interim analysis for the other primary endpoint of progression-free survival for nivolumab plus ipilimumab versus nivolumab across all treatment lines.

Bone Stress Injury Epidemiology and Risk Factors in Female Off-Road Runners: A Systematic Review.

Background: Off-road running is a growing sport with little research investigating injury profiles of female participants. Bone stress injuries (BSIs) are a particularly detrimental injury with little known about their incidence and risk factors in female off-road runners.

Objective: Collate and review the available evidence reporting epidemiological data and risk factors associated with BSI in female off-road runners.

Perilymph sampling in Mongolian gerbil, technical note and procedure evaluation.

Objective: The aim of this study is to detail and evaluate the surgical procedure for perilymph sampling from the cochlear apex in the Mongolian gerbil.

Design: Perilymph sampling from the cochlear apex was performed one to three time in 12 male gerbils aged 8 to 12 months via the submandibular route. 11 of them were previously implanted with intracochlear implants loaded with dexamethasone and placed in the scala tympani, the 12th was used to collect control samples.

VAE-Surv: A novel approach for genetic-based clustering and prognosis prediction in myelodysplastic syndromes.

Background And Objectives: Several computational pipelines for biomedical data have been proposed to stratify patients and to predict their prognosis through survival analysis. However, these analyses are usually performed independently, without integrating the information derived from each of them. Clustering of survival data is an underexplored problem, and current approaches are limited for biomedical applications, whose data are usually heterogeneous and multimodal, with poor scalability for high-dimensionality.

Clinical outcomes and management following progressive disease with anti-PD-(L)1 therapy in patients with advanced Merkel Cell Carcinoma.

Aim: Merkel Cell Carcinoma (MCC) is a rare skin cancer with a rising incidence worldwide. Anti-programmed death-1/ligand-1 (anti-PD-(L)1) therapies are effective for the treatment of advanced MCC. This study examines patterns of response / progression of advanced MCC to anti-PD-(L)1 therapies and describes subsequent management.

Antibiotics use decreases survival in cutaneous squamous cell carcinoma patients receiving immune checkpoint inhibitors.

Introduction: Immune checkpoint inhibitors (ICI) have become the first-line therapy in patients with advanced cutaneous squamous cell carcinoma (cSCC). Antibiotics (ATB) have been reported to reduce ICI response in cancers, but this has not been evaluated in cSCC.

Aim: To evaluate ATB exposure at the onset of ICI in cSCC patients and to analyze its impact on outcome.

Cancer-specific utility instrument for health economic evaluations: A synopsis of the EORTC QLU-C10D user manual and current validity evidence.

The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility - Core 10 Dimensions (QLU-C10D) adds a preference-based scoring algorithm to the EORTC measurement portfolio. It is built on the most widely used health-related quality of life (HRQoL) measure in oncology, the EORTC Quality of Life Questionnaire - Core 30 (QLQ-C30), allowing for the calculation of both HRQoL profiles and health utilities. This is an important advancement for integrating cancer-specific values into health economic evaluations and decision making, offering greater content validity and statistical power than some generic measures.

Health networking on cancer in the European Union: a 'green paper' by the EU Joint Action on Networks of Expertise (JANE).

Health networking is in principle a formidable instrument to address many challenges posed by cancer, one of the two most common and most lethal non-communicable chronic diseases. The European Union (EU)'s Beating Cancer Plan foresaw the addition of new health networks to the four already existing European Reference Networks on rare cancers: the Network of Comprehensive Cancer Centres and several networks of expertise (NoEs), which will be shortly deployed on items as complex and poor-prognosis cancers, palliative care, survivorship, personalised primary and secondary prevention, omic technologies, hi-tech medical resources, and cancers in adolescents and young adults. The community of experts of the EU Joint Action, due to build such NoEs, has drafted this 'green paper', incorporating 13 open questions, in an effort to foster discussion on some open questions about health networking on cancer in the EU.

Interaction of cesium compounds with abundant inorganic compounds of atmosphere: Effect on cloud formation potential and settling.

Experiments were conducted in controlled laboratory conditions to determine the size-resolved CCN (Cloud Condensation Nuclei) activity of sub micrometer-sized aerosols containing nuclear fission products (CsI and CsOH) and abundant ambient inorganic aerosols ammonium sulphates ((NH)SO), ammonium chloride (NHCl), sodium nitrate (NaNO), and sodium chloride (NaCl). The presence of these atmospheric-relevant compounds internally mixed with fission product compounds has the potential to affect the capacity of ambient particulates of aerosols to absorb water and function as CCN. Once in the atmosphere, the dynamics of airborne radionuclides and subsequently their fate gets affected by dry and wet deposition processes.

Rational choices elicit stronger sense of agency in brain and behavior.

The sense of agency is the subjective feeling of control over one's own actions and the associated outcomes. Here, we asked whether and to what extent the reasons behind our choices (operationalized by value differences, expected utility, and counterfactual option sets) drive our sense of agency. We simultaneously tested these three dimensions during a novel value-based decision-making task while recording explicit (self-reported) and implicit (brain signals) measures of agency.