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The number of pregnancies in women with cystic fibrosis (CF) has significantly increased in recent years, leading to a corresponding rise in the number of healthy infants exposed to cystic fibrosis transmembrane conductance regulator modulator (CFTRm) such as elexacaftor-tezacaftor-ivacaftor (ETI) or Kaftrio/Kalydeco® (K/K) triple therapy. Currently, data on the immediate outcomes for these children is reassuring; however, some cases of abnormal liver tests and cataracts have been reported in a few newborns indirectly exposed to ETI in utero or postnatally. Long-term neurodevelopment remains a concern that requires further investigation. A working group from the Société Française de la Mucoviscidose has developed recommendations for monitoring these children during the first two years and beyond. Given the increasing number of infants born to mothers taking CFTR modulators, as well as questions regarding their immediate care during the maternity stay and the feasibility of breastfeeding, it is crucial for pediatricians to be aware of these recommendations, which are based on a comprehensive review of the literature.
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http://dx.doi.org/10.1016/j.arcped.2025.03.005 | DOI Listing |
JAMA Pediatr
September 2025
Department of Epidemiology and Biostatistics, University of South Carolina, Columbia.
J Antimicrob Chemother
September 2025
Department of Pharmaceutical Sciences, University of Michigan College of Pharmacy, Ann Arbor, MI 48109, USA.
Background: Synergy between antibiotic pairs is typically discovered using chequerboard assays that assume uniform, static drug exposure; however, such conditions rarely apply in vivo. Dynamic and heterogeneous tissue environments create spatial and temporal mismatches in drug exposure that can uncouple synergistic interactions, leading to unexpected treatment failure.
Objective: This study aims to develop a physiologically relevant in vitro model that integrates infection-site microenvironments and drug-specific pharmacokinetics.
Front Pediatr
August 2025
Division of Pediatric Pulmonology, Department of Pediatrics, Oregon Health & Science University, Portland, OR, United States.
Background: Modulator therapy restores CFTR function and has led to health benefits for persons with cystic fibrosis (CF) (PwCF), including lower rates of pulmonary exacerbations. It is unknown if modulators affect lung function trajectories after inpatient treatment of pulmonary exacerbations (PEx).
Methods: We conducted a retrospective review of hospital encounters for PEx for subjects 6-25 years old with mild to moderate lung disease admitted to a large tertiary care center from 2014 to 2021 to capture hospitalizations of PwCF before and after starting modulators.
Front Pharmacol
August 2025
Department of Pharmacy, Shanghai Pulmonary Hospital, Tongji University School of Medicine, Shanghai, China.
Dipeptidyl peptidase 1 (DPP1) inhibitors constitute a major advance in respiratory disease therapeutics. Through selective blockade of neutrophil serine protease (NSP) activation, these agents establish novel treatment paradigms for inflammatory respiratory conditions characterized by neutrophil-driven pathology. This comprehensive review examines the development status, clinical efficacy, and safety profile of DPP1 inhibitors in neutrophil-driven diseases, particularly non-cystic fibrosis bronchiectasis (NCFBE) and chronic obstructive pulmonary disease (COPD).
View Article and Find Full Text PDFAim: Positive expiratory pressure devices are frequently used for airway clearance in children with cystic fibrosis and tracheobronchomalacia. This study aimed to establish if electrical impedance tomography is a feasible measure to titrate pressures in non-sedated children.
Method: Ten children with cystic fibrosis and tracheobronchomalacia performed airway clearance using positive pressure devices whilst monitored with electrical impedance tomography.