Early glucose abnormalities revealed by continuous glucose monitoring associate with lung function decline in cystic fibrosis: A five-year prospective study.

Background: Cystic fibrosis related diabetes (CFRD) is commonly associated with declining lung function and nutritional status. We aimed to evaluate the pulmonary impact of early glucose abnormalities by using 2-h standard oral glucose tolerance testing (OGTT) and continuous glucose monitoring (CGM) in people with cystic fibrosis (PwCF).

Methods: PwCF aged ≥10 years old without known CFRD were included in a five-year prospective multicentre study.

Screening strategies for glucose tolerance abnormalities and diabetes in people with cystic fibrosis.

The increase in life expectancy of patients with cystic fibrosis has come with new comorbidities, particularly diabetes. The gradual development of glucose tolerance abnormalities means that 30 to 40% of adults will be diabetic. Cystic fibrosis-related diabetes is a major challenge in the care of these patients because it is a morbidity and mortality factor at all stages of the disease.

Nutritional status at age 1 year in patients born with esophageal atresia: A population-based, prospective cohort study.

Objective: Despite recent progress in caring for patients born with esophageal atresia (EA), undernutrition and stunting remain common. Our study objective was to assess nutritional status in the first year after birth with EA and to identify factors associated with growth failure.

Study Design: We conducted a population-based study of all infants born in France with EA between 2010 and 2016.

Clinical practice versus guidelines for the screening of cystic fibrosis-related diabetes: A French survey from the 47 centers.

This study aimed to analyze clinical practices concerning cystic fibrosis-related diabetes (CFRD) screening in France. A web-based questionnaire was distributed between December 1, 2020 and January 31, 2021 among 47 cystic fibrosis centers including pediatric, adult, and mixed units. In accordance with guidelines, 92.

Comparison of Continuous Glucose Monitoring in Cystic Fibrosis Patients With or Without Pancreatic Exocrine Insufficiency.

This study aimed to compare continuous glucose monitoring (CGM) in cystic fibrosis (CF) according to pancreatic exocrine status.CGM and oral glucose tolerance testing (OGTT) were realized annually over five years in people with CF (pwCF) aged≥10 years without cystic fibrosis-related diabetes (CFRD). CGM parameters in patients with normal glucose tolerance (NGT), impaired glucose tolerance (IGT), and newly diagnosed CFRD were compared according to presence of pancreatic sufficiency (PS) or insufficiency (PI).

Using chest computed tomography and unsupervised machine learning for predicting and evaluating response to lumacaftor-ivacaftor in people with cystic fibrosis.

Objectives: Lumacaftor-ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator known to improve clinical status in people with cystic fibrosis (CF). The aim of this study was to assess lung structural changes after 1 year of lumacaftor-ivacaftor treatment and to use unsupervised machine learning to identify morphological phenotypes of lung disease that are associated with response to lumacaftor-ivacaftor.

Methods: Adolescents and adults with CF from a French multicentre real-world prospective observational study evaluating the first year of treatment with lumacaftor-ivacaftor were included if they had pre-therapeutic and follow-up chest computed tomography (CT) scans available.

Chest physiotherapy enhances detection of in nonexpectorating children with cystic fibrosis.

Lung damage in cystic fibrosis (CF) is strongly associated with lower airway infections. Early treatment of is recommended. Pathogen detection requires sampling of lower airway secretions, which remains a challenge in nonexpectorating patients.

Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function.

Background: Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA) in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and percent predicted forced expiratory volume in 1 s (ppFEV) between 40 and 90. Marketing authorizations have been granted for patients at all levels of ppFEV.

Methods: To evaluate the safety and effectiveness of LUMA-IVA over the first year of treatment in patients with ppFEV<40 or ppFEV≥90 in comparison with those with ppFEV [40-90[.

Effect of one-year lumacaftor-ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients.

Objectives: To investigate the effects of 1-year lumacaftor-ivacaftor treatment on abnormalities in glucose tolerance (AGT) in Phe508del homozygous cystic fibrosis (CF) patients.

Methods: Untreated CF patients with glucose intolerance or newly diagnosed diabetes were included in a prospective, observational study. After 1-year lumacaftor-ivacaftor treatment, AGT were evaluated by using oral glucose tolerance test.

Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis.

Lumacaftor-ivacaftor is a CFTR (cystic fibrosis transmembrane conductance regulator) modulator combination recently approved for patients with cystic fibrosis (CF) homozygous for the Phe508del mutation. To evaluate the safety and effectiveness of lumacaftor-ivacaftor in adolescents (≥12 yr) and adults (≥18 yr) in a real-life postapproval setting. The study was conducted in the 47 CF reference centers in France.

[From pediatric care to adult medicine: Transition of sickle cell patients, a French monocentric study].

Sickle cell disease, a hemoglobin disorder with autosomal recessive transmission, is one of the most common genetic diseases screened in France. Thanks to early management, 95% of sickle cell patients reach adulthood and require transition from pediatric care to adult care. Through a retrospective study of records from serious sickle cell patients over 17 years old, followed in the hematology-oncology pediatric unit of Reims University Hospital Center in France, we analyzed transition conditions, compared pediatric and adult management, and proposed a plan for transition care.

[An indolent and fluctuating subcutaneous mass of the skull in a 5-year-old patient: Diagnostic approach and difficulties].

A subcutaneous mass of the skull in children can have many different causes (infectious, tumoral, and inflammatory). We report on the case of a 5-year-old patient with a subcutaneous mass of the skull evolving over several months. The first pathological analysis concluded in Kimura disease.

[Pancreatic infringement exocrine and endocrine in cystic fibrosis].

The exocrine pancreatic insufficiency affects more than 80% of cystic fibrosis (CF) infants. Pancreatic insufficiency is diagnosed by low levels of fecal elastase. An optimal caloric intake, a pancreatic enzyme treatment are the keys to maintain a good nutritional status.

Rhinovirus-associated pulmonary exacerbations show a lack of FEV1 improvement in children with cystic fibrosis.

Background: Respiratory viral infections lead to bronchial inflammation in patients with cystic fibrosis, especially during pulmonary exacerbations. The aim of this study was to determine the impact of viral-associated pulmonary exacerbations in children with cystic fibrosis and failure to improve forced expiratory volume in 1 s (FEV1 ) after an appropriate treatment.

Methods: We lead a pilot study from January 2009 until March 2013.

Causes of death in French cystic fibrosis patients: The need for improvement in transplantation referral strategies!

Background: Little data exist on causes of death in cystic fibrosis (CF) patients in the era of lung transplantation.

Methods: Deaths in CF patients in France (2007-2010) were identified using the French CF Registry and causes of deaths were determined based on medical files by a mortality adjudication committee.

Results: Of 256 deaths, half occurred after lung transplantation and were related to early or late complications of transplantation, whereas half occurred in patients who did not receive lung transplantation and were primarily related to respiratory failure or massive hemoptysis.

[National consensus regarding azithromycin use in cystic fibrosis].

Aim: To propose a formalized consensus agreement regarding the prescription of azithromycin in cystic fibrosis (CF).

Material And Methods: Application of the Delphi method in 5 thematic fields: indications, contra-indications, dosage, precautions for use and treatment follow-up.

Results: Thirty identified French CF centers participated in the process on 49 (61%), which comprised 3 rounds.

Management of pancreatic, gastrointestinal and liver complications in adult cystic fibrosis.

Introduction: The gastrointestinal tract is a major source of morbidity in adults with cystic fibrosis (CF), with a wide range of complications, some of them being specific to the underlying disease.

State Of Knowledge: Abnormal CFTR function, with reduced bicarbonate and other ion transport levels through the apical surface of epithelial cells, affects the intestinal tract including the pancreas and the liver. Similarly to what is observed in the respiratory tract, gastrointestinal CFTR dysfunction leads to mucus accumulation, dysmotility, small bowel bacterial overgrowth and inflammation with alteration of innate immune responses, all of which being likely to be interrelated.

Inhibition of the NOTCH pathway using γ-secretase inhibitor RO4929097 has limited antitumor activity in established glial tumors.

Notch signaling is altered in many cancers. Our previous findings in primary pediatric ependymoma support a role for NOTCH in glial oncogenesis. The present study evaluates the γ-secretase inhibitor RO4929097 in glial tumor models.

Transient remission of childhood acute lymphoblastic and myeloid leukemia without any cytostatic treatment: 2 case reports and a review of literature.

Transient remissions (TRs) of acute leukemia without any antileukemic treatment are extremely rare events. We report 2 TRs of acute lymphoblastic leukemia and acute myeloid leukemia in a 2-year-old boy and a 12-year-old girl, respectively, both associated with red blood cells and platelets transfusions and infection. These 2 factors are frequently present in previously reported cases and could induce a stimulation of the immune system although the underlying mechanisms of TRs are still unknown.

Cystic fibrosis airway epithelium remodelling: involvement of inflammation.

Chronic inflammation is a hallmark of cystic fibrosis (CF) lung disease and airway epithelium damage and remodelling are important components of lung pathology progression in CF. Whether this remodelling is secondary to deleterious infectious and inflammatory mediators, or to alterations of CF human airway epithelial (HAE) cells, such as their hyper inflammatory phenotype or their basic cystic fibrosis transmembrane conductance regulator (CFTR) default, remains debated. In this study, we evaluated the involvement of alterations of CF HAE cells in airway epithelium remodelling.

Social cognition in ADHD: irony understanding and recursive theory of mind.

The main goal of the present study was to characterise the social cognition abilities of French children with ADHD, in terms of their understanding of people's recursive mental states and their irony comprehension. We hypothesised that these children have difficulty understanding second-order false beliefs and ironic remarks, owing to the executive dysfunction that is characteristic of ADHD. We therefore conducted an experiment in which children with ADHD and typically developing matched controls performed second-order false-belief and executive function tasks.

How to minimize toxic exposure to pyridine during continuous infusion of ceftazidime in patients with cystic fibrosis?

Ceftazidime is particularly efficient against Pseudomonas aeruginosa in cystic fibrosis patients. Thus, the spontaneous production of pyridine, which is a toxic product, raises some concern. Our aim was to examine the kinetics of degradation of ceftazidime in portable infusion pumps either at 4°C, 22°C, or 33°C and to propose some recommendations in order to reduce the pyridine exposure.

[A retrospective study to evaluate a protocol aimed at reducing the number of unnecessary voiding cystourethrographies performed after a first episode of febrile urinary tract infection].

Aim: Whether or not voiding cystourethrography (VCUG) should be performed after a first episode of urinary tract infection (UTI) remains a matter of debate. The role of VCUG is primarily to diagnose high-grade vesicoureteral reflux (≥grade III) (VUR) and hence prevent the development of renal scars and poor long-term outcome. We designed a protocol designed to reduce the indications for performing unnecessary VCUGs after a first episode of febrile UTI.

Enteroviruses as major cause of microbiologically unexplained acute respiratory tract infections in hospitalized pediatric patients.

Objective: To assess the etiological role and the clinical characteristics of HRV and HEV infections in pediatric patients hospitalized for acute respiratory tract infections (ARTIs).

Methods: RT-qPCR assays and molecular sequencing methods were used to identify HRV and HEV strains in nasopharyngeal aspirates of 309 hospitalized pediatric patients with microbiologically unexplained ARTIs and in 210 hospitalized pediatric patients without respiratory symptoms from September 2009 to June 2010 in France.

Results: Among the 309 ARTI cases, 15 HEV and 172 HRV strains were identified whereas only 1 HEV and 37 HRV strains were observed in control patients (187 vs.