The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study.

Background: Elexacaftor-tezacaftor-ivacaftor has been approved in Europe for people with cystic fibrosis with at least one F508del CFTR variant. Additionally, it is approved by the US Food and Drug Administration (FDA) for people with cystic fibrosis with at least one of 177 rare variants. The aims of this study were to describe the clinical response to elexacaftor-tezacaftor-ivacaftor for people with cystic fibrosis without a F508del CFTR variant in France and to determine CFTR variant responsiveness to elexacaftor-tezacaftor-ivacaftor based on the observed clinical response.

Race as social determinant of growth and body composition among infants born very preterm.

Objective: Racism leads to disparities in health outcomes. Our objective was to determine if black race was independently associated with differences in fat accretion at discharge in a large cohort of very preterm infants (32 weeks of gestation or less).

Methods: De-identified demographic, anthropometric and body composition data were collected from seven neonatal units around the United States.

Human milk, breastfeeding, and early neurodevelopmental outcomes for infants with critical CHD.

Human milk improves neurodevelopment for preterm infants, but relationships between human milk and neurodevelopment for infants with critical CHD are unknown. We aimed to (1) explore associations between human milk/direct breastfeeding and neurodevelopment at 1-year and 2-year follow-up and (2) describe patterns of human milk (maternal, donor) and commercial formula during hospitalisation in the first year of life.This retrospective cohort study included infants who underwent surgery for CHD < 6 months old.

Sexual dysfunction in cystic fibrosis.

Background: Sexual dysfunction (erectile dysfunction in males, sexual dissatisfaction, sexual interest/arousal disorders, and dyspareunia in females) has not been the subject of indepth research in people with cystic fibrosis (CF). This study aimed to determine the prevalence of sexual dysfunction in adults with CF, factors associated with sexual dysfunction, and the impact of sexual dysfunction on quality of life.

Method: We conducted a multicentre study in adults with cystic fibrosis followed in specialist centres in Western France.

Multisystemic Effects of Elexacaftor-Tezacaftor-Ivacaftor in Adults with Cystic Fibrosis and Advanced Lung Disease.

Limited data exist on the safety and effectiveness of elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) and advanced lung disease. To evaluate the effects of ETI in an unselected population of pwCF and advanced lung disease. A prospective observational study, including all adults aged 18 years and older with percentage predicted forced expiratory volume in 1 second (ppFEV) ⩽ 40 who initiated ETI from December 2019 to June 2021 in France, was conducted.

Insulin-like growth factor-1 and insulin-like growth factor binding protein-3 as early predictors of growth, body composition, and neurodevelopment in preterm infants.

Objective: To investigate the relationship between insulin-like growth factor 1 (IGF-1), insulin-like growth factor binding protein 3 (IGFBP-3) and long-term growth, body composition, and neurodevelopment in preterm infants.

Study Design: Prospective data were collected from ≤32 weeks gestational age infant cohort (N = 50). IGF-1 and IGFBP-3 concentrations were measured at 1 week (early) and 35 weeks (late) post-menstrual age (PMA).

Physical activity promotion in French cystic fibrosis centers: capitalizing on experience.

Background: Physical activity (PA) provides physical and psychosocial benefits for people with cystic fibrosis (pwCF). However, practice levels remain below recommendations and strategies for promoting PA in specialist centers need to be better identified. The socio-ecological model of health emphasizes the central role of policies and environment in influencing individuals' health behaviors.

Body composition after implementation of an enhanced parenteral nutrition protocol in the neonatal intensive care unit: a randomised pilot trial.

Background: Very low birthweight (VLBW) infants are at risk for growth failure and poor neurodevelopment. Optimised parenteral nutrition may help promote optimal growth and development, but concerns that provision of enhanced nutrition may contribute to increased early adiposity and later metabolic disease remain.

Aim: To determine associations between provision of an early enhanced parenteral nutrition protocol or standard parenteral nutrition protocol and growth and body composition for VLBW preterm infants in the neonatal intensive care unit.

An exploratory study of clinical factors associated with IGF-1 and IGFBP-3 in preterm infants.

Background: Despite advances in parenteral nutrition, postnatal growth failure in very low birthweight (VLBW) preterm infants is common and associated with chronic health problems. Insulin-like growth factor 1 (IGF-1) is positively associated with improved infant growth, but factors which promote IGF-1 levels in this population have not been clearly identified. The objective of this study was to explore early factors that influence IGF-1 in VLBW preterm infants.

Prevalence and severity of functional urinary and anorectal disorders and their impact on quality of life in cystic fibrosis.

Background: In cystic fibrosis (CF), coughing is associated with a risk of pelvic floor dysfunction. However, data on the prevalence of symptoms (stress urinary incontinence, bladder overactivity, dysuria, and faecal incontinence) are lacking in males and females with CF. The impact of incontinence on adherence to respiratory care has not been studied.

Feeding characteristics of healthy infants without reported feeding impairments throughout the first month of life.

Objective: Elucidate characteristics of feeding performance in healthy infants without reported feeding problems throughout the first month of life.

Study Design: Feeding was monitored in 61 healthy infants by caregiver report for 48 h a week from birth to 4 weeks old. Outcomes included feeding modality, how much they consumed, how long the feed lasted, and how many coughing episodes the infant exhibited.

Establishing Normative Values for Healthy Term Infant Feeding Performance: Neonatal Eating Assessment Tool-Mixed, Oral Feeding Scale, and Early Feeding Skills Assessment.

Purpose: Infants with perceived feeding problems are frequently referred for assessment of their feeding abilities. However, little is known regarding how healthy nondysphagic infants perform on commonly used assessments, making determination of impairment difficult. The aim of this investigation was to elucidate the characteristics of healthy term infant feeding performance using three commonly employed clinical assessments: Neonatal Eating Assessment Tool-Mixed (NeoEat-Mixed), Oral Feeding Scale, and Early Feeding Skills (EFS).

Identification of clinical factors associated with timing and duration of spontaneous regression of retinopathy of prematurity not requiring treatment.

Objective: Identify clinical factors that delay or prolong spontaneous regression of retinopathy of prematurity (ROP).

Study Design: Secondary analysis of three prospective studies with 76 infants with ROP not requiring treatment, born ≤30 weeks postmenstrual age (PMA) and ≤1500 grams. Outcomes were PMA at greatest severity of ROP (PMA MSROP), at which regression began, at time of complete vascularization (PMA CV), and regression duration.

Dilemmas in parenteral glucose delivery and approach to glucose monitoring and interpretation in the neonate.

Glucose control continues to be challenging for intensivists, in particular in high-risk neonates. Many factors play a role in glucose regulation including intrinsic and extrinsic factors. Optimal targets for euglycemia are debatable with uncertain short and long-term effects.

Enhanced Parenteral Nutrition Is Feasible and Safe in Very Low Birth Weight Preterm Infants: A Randomized Trial.

Objective: The objective of this study was to determine the feasibility and safety of enhanced early (PN) (early initiation of intralipids and faster advancement of glucose infusion rate) during the first week of life for very low birth weight (VLBW) preterm infants.

Methods: 90 VLBW preterm infants (<32 weeks gestational age at birth) admitted to the University of Minnesota Masonic Children's Hospital between August 2017 and June 2019 were included. Enrolled infants were stratified by gestational age-groups and randomized to either the enhanced nutrition protocol (intervention group) or the standard PN protocol (standard group).

The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del variant.

Background: The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (CF) carrying at least one F508del variant. The United States Food and Drug Administration (FDA) also approved ETI for people with CF carrying one of 177 rare variants.

Methods: An observational study was conducted to evaluate the effectiveness of ETI in people with CF with advanced lung disease who were not eligible for ETI in Europe.

Preterm infant body composition, working memory, and temperament.

Altered body composition in preterm infants is associated with risks to cognitive development, but the effect specific to prefrontal cortex (PFC) development is unknown. We were interested in the impact of fat mass (FM) and fat free mass (FFM) gains out to 4 months corrected gestational age (CGA) on PFC development, as indexed by working memory and temperament. This is a prospective observational pilot study recruiting 100 preterm (<33 weeks gestation), appropriate for gestational age, and very low birth weight infants, of which 49 infants met inclusion criteria.

Randomized Trial of Early Enhanced Parenteral Nutrition and Later Neurodevelopment in Preterm Infants.

Retrospective studies indicate that the parenteral provision of calories, proteins, and lipids in the first week of life is associated with improved later neurodevelopment. We aimed to determine whether infants randomized to an enhanced parenteral nutrition protocol had improved developmental outcomes at 4, 12, or 24 months corrected age (CA). In total, 90 preterm infants (<32 weeks gestational age and <1500 g) were randomized to receive enhanced parenteral nutrition (PN) or standard PN during the first week of life.

Body composition measurement for the preterm neonate: using a clinical utility framework to translate research tools into clinical care.

Body composition analysis to distinguish between fat mass and fat-free mass is an established research approach to assess nutritional status. Within neonatal medicine, preterm infant body composition is linked with later health outcomes including neurodevelopment and cardiometabolic health. Mounting evidence establishing fat-free mass as an indicator of nutritional status, coupled with the availability of testing approaches that are feasible to use in preterm infants, have enhanced interest in measuring body composition in the neonatal intensive care unit (NICU) setting.

The Impact of Human Milk on Outcomes for Infants with Congenital Heart Disease: A Systematic Review.

Infants with congenital heart disease (CHD) are at risk for feeding-related morbidity and mortality, with growth failure and oral feeding problems associated with poor outcomes. The benefits of human milk (HM) for preterm infants have been well documented, but evidence on HM for infants with CHD has recently begun to emerge. Our primary aim was to examine the impact of HM feeding on outcomes for infants with CHD.

Sustained effectiveness of elexacaftor-tezacaftor-ivacaftor in lung transplant candidates with cystic fibrosis.

Background: Elexacaftor-tezacaftor-ivacaftor induces rapid clinical improvement in patients with cystic fibrosis (CF) and advanced pulmonary disease, often leading to suspend the indication for lung transplantation. Yet no long-term data is available in lung transplant candidates.

Methods: Lung transplant candidates (defined as being waitlisted for lung transplantation or considered for listing within 3 months) who have initiated elexacaftor-tezacaftor-ivacaftor were identified in the French cohort of patients with CF and advanced pulmonary disease.