Preliminary proposals for the follow-up of infants born to mothers with cystic fibrosis treated with CFTR modulators during the first two years of life

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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Publication Details

Journal
Archives de Pédiatrie
Published
2025-06-13
DOI
https://doi.org/10.1016/j.arcped.2025.03.005
Citations
2
Primary Topic
Cystic Fibrosis Research Advances
Type
article
Field-Weighted Citation Impact
3.04
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article

Preliminary proposals for the follow-up of infants born to mothers with cystic fibrosis treated with CFTR modulators during the first two years of life

Camille Audousset, Sophie Gautier, Isabelle Sermet‐Gaudelus, Philippe Reix et al.
2 citations
Archives de Pédiatrie
Cystic Fibrosis Research Advances
3.04
article

Preliminary proposals for the follow-up of infants born to mothers with cystic fibrosis treated with CFTR modulators during the first two years of life

Camille Audousset, Sophie Gautier, Isabelle Sermet‐Gaudelus, Philippe Reix, Emmanuelle Girodon
article en
2 citations

Abstract

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.

Archives de PédiatrieVol. 32(5)
Centre National de la Recherche Scientifique (FR), Inserm (FR), Université Paris Cité (FR), Centre Hospitalier Universitaire de Lille (FR), Hôpital Femme Mère Enfant (FR), Hôpital Cochin (FR), Vaincre la Mucoviscidose (FR), Assistance Publique – Hôpitaux de Paris (FR), Center for Infection and Immunity of Lille (FR)
Good health and well-being
Openalex Percentile: Top 9%
Cystic Fibrosis Research Advances
3.04
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