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 - 11/07/25
, Camille Audousset b, Emmanuelle Girodon c, Isabelle Sermet Gaudelus d, Sophie Gautier eHighlights |
• | Increasing number of infants born to mothers under CFTR modulators. |
• | Pharmacological data indicate fetuses exposed to significant drug levels in utero. |
• | No increased risk of malformation reported in France or other countries. |
• | Abnormal liver tests and cataracts have been reported and required early monitoring. |
• | CFTR genetic testing must be checked newborn screened babies because of possible falsy low immunoreactive trypsin levels. |
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.
Le texte complet de cet article est disponible en PDF.Keywords : Infant, Cystic fibrosis, Pregnancy, Elexacaftor, Ivacaftor, Tezacaftor
Plan
Vol 32 - N° 5
P. 344-350 - juillet 2025 Retour au numéro
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