Systemic Inflammatory Diseases in Children with Sickle Cell Disease: A French Multicenter Observational Study on Diagnostic and Therapeutic Issues.

Background: Systemic inflammatory diseases (SIDs) have been reported in patients with sickle cell disease (SCD), but clinical data in children are scarce.

Objectives: To identify clinical and laboratory features at diagnosis of SID in children with SCD and to describe their evolution.

Methods: Data from children with SCD and SIDs were retrospectively collected in a French multicenter study from 1991 to 2018.

Postneonatal Cerebral Palsy in Europe: Prevalence and Clinical Characteristics According to Contributory Events: An SCPE Study.

Background: Postneonatal cerebral palsy (PNCP) is rare and requires large databases to be studied over time.

Objectives: To study the time trend of prevalence of PNCP overall and by cause, and to describe the clinical characteristics of children with PNCP according to cause and compared with children with pre/peri/neonatal CP (PPNCP).

Methods: The Surveillance of Cerebral Palsy in Europe (SCPE) database was used.

How does the environmental inadequacy mediate the effect of functional limitations on participation restrictions in young adults with cerebral palsy?

Background: Adults with cerebral palsy (CP) face various functional limitations and comorbidities, that prevent them from participating fully in social life. Disability models suggest that an environment not tailored to their needs could partly explain the link between functional limitations and participation restrictions. However, there is still insufficient knowledge about how the environment hinders participation.

Neurodevelopmental outcome in children between one and five years after persistent pulmonary hypertension of term and near-term newborns.

Background: Persistent pulmonary hypertension of the newborn (PPHN) is a serious condition that affects 1-2 per 1,000 newborns. Scientific data report the existence of neurological developmental abnormalities between 10 and 30%, but the description of these disorders linked with this situation of cerebral hypoxia and haemodynamic failure remains poorly documented.

Objective: The main goal of this study was to describe the prevalence of neuro-psychomotor developmental disorders in children aged between one and five years old who have been hospitalised at birth in a neonatal intensive care unit for the management of PPHN.

Impact of hydroxyurea on follicle density in patients with sickle cell disease.

The impact of hydroxyurea (HU) on the ovarian reserve of female patients with sickle cell disease (SCD) remains poorly elucidated. Only direct histological analysis of ovarian follicle density can effectively evaluate HU's effect on ovarian reserve. By analyzing digitized slides of ovarian tissue from girls and young women with SCD who underwent ovarian tissue cryopreservation (OTC) before hematological stem cell transplantation, we meticulously counted follicles and categorized them based on their growth stage.

Perspectives of cerebral palsy experts on access to health care in Europe.

Aim: To explore the perspectives of cerebral palsy (CP) experts on access to healthcare and an analysis of socioeconomic and environmental determinants impacting young individuals with CP in Europe.

Method: Cross-sectional survey designed by a convenience multi-disciplinary panel of invited experts and completed by clinicians, researchers and opinions leaders in the field of CP.

Results: Fifty-eight experts (response rate 85 %) from 39 regions in 26 European countries completed the survey.

Intermittent hypoxia increases lipid insulin resistance in healthy humans: A randomized crossover trial.

Sympathetic overactivity caused by chronic intermittent hypoxia is a hallmark of obstructive sleep apnea. A high sympathetic tone elicits increases in plasma free fatty acid and insulin. Our objective was to assess the impact of 14 nights of chronic intermittent hypoxia exposure on sympathetic activity, glucose control, lipid profile and subcutaneous fat tissue remodelling in non-obese healthy humans.

Are the class 18 myosins Myo18A and Myo18B specialist sarcomeric proteins?

Initially, the two members of class 18 myosins, Myo18A and Myo18B, appeared to exhibit highly divergent functions, complicating the assignment of class-specific functions. However, the identification of a striated muscle-specific isoform of Myo18A, Myo18Aγ, suggests that class 18 myosins may have evolved to complement the functions of conventional class 2 myosins in sarcomeres. Indeed, both genes, and , are predominantly expressed in the heart and somites, precursors of skeletal muscle, of developing mouse embryos.

Unmet environmental needs and unmet healthcare needs in a population of young adults with cerebral palsy: what the SPARCLE study tells us.

Introduction: Optimizing care for young adults with cerebral palsy is crucial for their physical and psychological well-being. The inadequacy of proximal environment may play a role in the provision of health services. The aim of this study is to explore the association between unmet environmental needs in the physical, social and attitudinal domains and unmet healthcare needs in four interventions: physiotherapy, occupational therapy, speech therapy and psychological counselling.

Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease: safety, ovarian function follow-up, and results of ovarian tissue transplantation.

Purpose: To describe the experience of performing ovarian tissue cryopreservation (OTC) before hematopoietic stem cell transplantation (HSCT), among girls/women with severe sickle cell disease (SCD)(SS or S/β-thalassemia) who are, besides the usual surgical risk, at risk of SCD-related complications during the fertility preservation procedure for improving their counseling and management.

Methods: This retrospective study included 75 patients (girls/women) with SCD who have had OTC before myeloablative conditioning regimen (MAC) for HSCT. Characteristics of patients and data on OTC, ovarian status follow-up, and results of ovarian tissue transplantation (OTT) were collected in medical records.

Satisfaction with health care services in young people with cerebral palsy in the transition period: results from a European multicenter study.

Background: Young people with chronic health conditions and disabilities rely on the healthcare system to maintain their best possible health. The appropriate delivery and utilization of healthcare services are key to improve their autonomy, self-efficacy and employment outcomes. The research question of our study is directed toward investigating if poor availability and accessibility of healthcare services in general, as identified by unmet needs in healthcare, are associated with dissatisfaction with healthcare.

Left atrial strain: A memory of the severity of atrial myocardial stress in atrial fibrillation.

Background: Left atrial (LA) strain is a simple marker of LA function. The aim of the study was to evaluate the determinants of atrial cardiomyopathy in AF.

Methods: In this pilot study, we prospectively evaluated clinical, biological, metabolomic and echocardiographic parameters for 85 consecutive patients hospitalized for atrial fibrillation (AF) with restoration of sinus rhythm at 6 months.

Assessing the Adequacy of the Physical, Social, and Attitudinal Environment to the Specific Needs of Young Adults With Cerebral Palsy: The European Adult Environment Questionnaire.

Objectives: To present the development of the European Adult Environment Questionnaire (EAEQ), to assess to what extent it covers the International Classification of Functioning, Disability and Health (ICF), and to describe the adequacy of the physical, social, and attitudinal environment to the specific needs of young adults with cerebral palsy (CP).

Design: Cross-sectional.

Setting: Administrative regions in France, Germany, Italy, Portugal, and Sweden.

Differences in participation between young adults with cerebral palsy and their peers: A cross-sectional multicentre European study.

Background: Children and adolescents with disabilities are known to participate less in most areas of life than their non-disabled peers.

Objectives: (1) To estimate differences in participation between young adults with cerebral palsy (CP) and their non-disabled peers; (2) to test the mediating role of financial difficulties and student status; (3) to test the moderating role of personal factors on participation difference; and (4) to test the moderating role of impairment.

Methods: A cross-sectional study was conducted in young adults [19-28 years] with CP (n = 228) and non-disabled peers (n = 2861) in France, Germany and Sweden.

Confirmatory validation of the french version of the Duchenne Muscular Dystrophy module of the pediatric quality of life inventory (PedsQL3.0DMDfv).

Duchenne Muscular Dystrophy (DMD) is a neuromuscular disease that inevitably leads to total loss of autonomy. The new therapeutic strategies aim to both improve survival and optimise quality of life. Evaluating quality of life is nevertheless a major challenge.