-related disorders: expansion of the phenotypic spectrum and long-term efficacy of deep brain stimulation.

Heterozygous mutations in are associated with an early-onset, progressive and often complex dystonia (DYT28). Key characteristics of typical disease include focal motor features at disease presentation, evolving through a caudocranial pattern into generalized dystonia, with prominent oromandibular, laryngeal and cervical involvement. Although -related disease is emerging as one of the most common causes of early-onset genetic dystonia, much remains to be understood about the full spectrum of the disease.

TREOCAPA: prophylactic treatment of the ductus arteriosus in preterm infants by acetaminophen-statistical analysis plan for the randomized phase III group sequential trial.

Background: Persistent patency of the ductus arteriosus (PDA) has challenged neonatologists for more than 40 years. Controversies persist about the management of PDA in extremely preterm infants. PDA is associated with morbidities, but no therapeutic strategy has resulted in an improved neonatal outcome.

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.

Management of patent ductus arteriosus in preterm infants: Abundant literature, low certainty of evidence for clinicians.

Management of patent ductus arteriosus (PDA) in preterm newborns has been debated extensively for more than six decades. Some authors affirm that PDA is only an innocent mirror of infant immaturity, without specific health consequences. They also underline the futility, even danger, of treating it.

Management of neonates exposed prenatally to opioids: Impact of a developmental care program implementation.

Background: Traditional approaches to management of neonates prenatally exposed to opioids are based on the use of pharmacotherapy (PT), adjusted to evolution of infant symptoms. Newer approaches focused on non-pharmacological care (NPC) are emerging, but there is little evidence on the active policies to implement to ensure their widespread practice. The primary objective of the study was to assess whether the implementation of a developmental care program in our neonatal medicine department had an impact on the management of these infants during hospitalization, notably exposure to NPC.

Mortality and neurodevelopmental outcomes at 2 years' corrected age of very preterm infants with necrotising enterocolitis or spontaneous intestinal perforation: The EPIPAGE-2 cohort study.

Purpose: The primary objective was to evaluate the impact of necrotising enterocolitis (NEC) and spontaneous intestinal perforation (SIP) on mortality and neurodevelopmental outcomes at 2 years' corrected age (CA) in infants born before 32 weeks' gestation (WG).

Methods: We studied neurodevelopment at 2 years' CA of infants with NEC or SIP who were born before 32 WG from the EPIPAGE-2 cohort study. The primary outcome was death or the presence of moderate-to-severe motor or sensory disability defined by moderate-to-severe cerebral palsy or hearing or visual disability.

Screening preterm-born infants for autistic traits may help to identify social communication difficulties at five years of age.

Aim: This study compared neurodevelopmental screening questionnaires completed when preterm-born children reached 2 years of corrected age with social communication skills at 5.5 years of age.

Methods: Eligible subjects were born in 2011 at 24-34 weeks of gestation, participated in a French population-based epidemiological study and were free of motor and sensory impairment at 2 years of corrected age.

Neurodevelopment at age 5.5 years according to Ages & Stages Questionnaire at 2 years' corrected age in children born preterm: the EPIPAGE-2 cohort study.

Objective: To report neurodevelopment at age 5.5 years according to developmental delay screening with the Ages & Stages Questionnaire (ASQ) in late infancy in preterm-born children.

Design: Population-based cohort study, EPIPAGE-2.

Efficacy of intravenous clonazepam for paediatric convulsive status epilepticus.

Aim: To compare the efficacy of intravenous clonazepam (CLZ) for the initial management of convulsive status epilepticus (CSE) in children as a function of the first-line in-hospital dose used.

Method: This monocentric retrospective study included children who received a first dose of CLZ for CSE at Montpellier University Hospital, France, between January 2016 and June 2019. Data from medical records (clinical, treatment, course) were collected and compared as a function of the first CLZ dose used.

ROX (Respiratory rate-OXygenation) index to predict early response to high-flow nasal cannula therapy in infants with viral bronchiolitis.

Introduction: High-flow nasal cannula (HFNC) is commonly used as first step respiratory support in infants with moderate-to-severe acute viral bronchiolitis (AVB). This device, however, fails to effectively manage respiratory distress in about a third of patients, and data are limited on determinants of patient response. The respiratory rate-oxygenation (ROX) index is a relevant tool to predict the risk for HFNC failure in adult patients with lower respiratory tract infections.

Prophylactic Intravenous Acetaminophen in Extremely Premature Infants: Minimum Effective Dose Research by Bayesian Approach.

Background: Patent ductus arteriosus (PDA) in preterm infants is associated with increased morbidities and mortality. Prophylactic treatment with cyclooxygenase inhibitors, as indomethacin or ibuprofen, failed to demonstrate significant clinical benefits. Acetaminophen may represent an alternative treatment option.

Music Therapy During Basic Daily Care in Critically Ill Children: A Randomized Crossover Clinical Trial.

Objective: To assess whether music therapy (MT) is effective to reduce pain during daily personal hygiene care (DPHC), a procedure performed in all patients in a pediatric intensive care unit.

Methods: Fifty critically ill children were enrolled in a crossover controlled clinical trial with random ordering of the intervention, that is, passive MT, and standard conditions, and blind assessment of pain on film recordings. The primary outcome was variation of the Face Legs Activity Cry Consolability (FLACC) score (range, 0-10) comparing before and during DPHC.

Cognitive Training for Visuospatial Processing in Children Aged 5½ to 6 Years Born Very Preterm With Working Memory Dysfunction: A Randomized Clinical Trial.

Importance: Compared with term-born peers, children born very preterm generally perform poorly in executive functions, particularly in working memory and inhibition. By taking advantage of neuroplasticity, computerized cognitive training of working memory in those children could improve visuospatial processing by boosting visual inhibition via working memory.

Objective: To evaluate the long-term effect of cognitive working memory training on visuospatial processing in children aged 5½ to 6 years born very preterm who have working memory impairment.

Pulmonary hypertension among preterm infants born at 22 through 32 weeks gestation in France: Prevalence, survival, morbidity and management in the EPIPAGE-2 cohort study.

Objective: To determine the prevalence, short-term prognosis and pharmacologic management of pulmonary hypertension (PH) among very preterm infants born before 32 weeks gestation (WG).

Study Design: In the EPIPAGE-2 French national prospective population-based cohort of preterm infants born in 2011, those presenting with PH were identified and prevalence was estimated using multiple imputation. The primary outcome was survival without severe morbidity at discharge and was compared between infants with or without PH after adjusting for confounders, using generalized estimating equations models.

Think out of the box: association of left congenital diaphragmatic hernia and abnormal origin of the right pulmonary artery : A train can hide another.

Background: We report the occurrence of a severe pulmonary hypertension (PH) in a neonate affected by a left congenital diaphragmatic hernia (CDH). PH in this patient was associated with an abnormal origin of the right pulmonary artery from the right brachiocephalic artery. This malformation, sometimes named hemitruncus arteriosus, has to the best of our knowledge never been reported in association with a CDH.

Neurodevelopment and healthcare utilisation at age 5-6 years in bronchopulmonary dysplasia: an EPIPAGE-2 cohort study.

Objective: We aimed to study neurodevelopmental outcomes and healthcare utilisation at age 5-6 years in very preterm children with bronchopulmonary dysplasia (BPD).

Design: Prospective and national population-based study.

Setting: All the neonatal units in 25 French regions (21 of the 22 metropolitan regions and 4 overseas regions).

Intraputaminal Gene Delivery in Two Patients with Aromatic L-Amino Acid Decarboxylase Deficiency.

Background: Aromatic l-amino acid decarboxylase deficiency (AADCD) is a rare, early-onset, dyskinetic encephalopathy mostly reflecting a defective synthesis of brain dopamine and serotonin. Intracerebral gene delivery (GD) provided a significant improvement among AADCD patients (mean age, ≤6 years).

Objective: We describe the clinical, biological, and imaging evolution of two AADCD patients ages >10 years after GD.

Risk factors for early occurrence of malnutrition in infants with severe congenital heart disease.

To identify the risk factors of early occurrence of malnutrition in infants with severe congenital heart disease (CHD) during their first year of life. Retrospective longitudinal multicenter study carried out from January 2014 to December 2020 in two tertiary care CHD centers. Four CHD hemodynamic groups were identified.

Birth during on-call period: Impact of care organization on mortality and morbidity of very premature neonates.

Objectives: The evidence that risks of morbidity and mortality are higher when very premature newborns are born during the on-call period is inconsistent. This study aimed to assess the impact of this situation among other determinants of outcomes, particularly newborn characteristics and care organization.

Methods: Observational study including all infants born < 30 weeks' gestation in a French tertiary perinatal center between 2007 and 2020.

In-hospital Outcomes and Early Hemodynamic Management According to Echocardiography Use in Hypotensive Preterm Infants: A National Propensity-Matched Cohort Study.

Background: Hypotension is a common condition during the first postnatal days of very preterm infants and has been associated with an increased risk of adverse outcomes but its management remains controversial. There is a consensus to promote the use of neonatologist-performed echocardiography (NPE) in hypotensive very preterm infants, although no clinical trial ever assessed this practice.

Methods: We conducted a retrospective analysis of prospectively collected data from the French national EPIPAGE-2 cohort to evaluate the association of NPE with survival, severe morbidity, and therapeutic management in very preterm infants with early hypotension.

Neurodevelopment at 5 Years of Age According to Early Screening for Patent Ductus Arteriosus in Extremely Preterm Infants.

This study compares neurodevelopment at 5 years of age in children who were extremely preterm infants and who underwent early systematic echocardiographic screening for patent ductus arteriosus (PDA) vs those who did not undergo screening.