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<p>Over the past 50 years, therapeutic advances have significantly improved the management of cystic fibrosis (CF). Recently, cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been widely regarded as a therapeutic revolution, and their use is associated with a marked increase in pregnancies among affected women. However, pregnant women were excluded from clinical trials, limiting what is known about maternal and fetal safety. Maternal and fetal outcome tend to be poorer when the disease is severe, but there is limited data on such pregnancies exposed to CFTR modulators. While available data suggest a low risk of birth defects, information on neurological development is lacking and concerns remain regarding lung abnormalities in animal studies, as well as a few cases of congenital cataracts reported in humans. For fetuses affected by CF, the transplacental transfer of CFTR modulators could potentially prevent severe complications, such as meconium ileus, opening promising therapeutic avenues.</p>

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A therapeutic revolution: CFTR modulators in cystic fibrosis and their impacts on pregnant women and the fetus

  • 期刊论文

  • 发表日期：

    2026年2月23日

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  • Marie Denef,
  • Marie Mawet,
  • Jessica Pirson,
  • Justine Tack,
  • Laure Noël,
  • Frédéric Chantraine

摘要

Over the past 50 years, therapeutic advances have significantly improved the management of cystic fibrosis (CF). Recently, cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been widely regarded as a therapeutic revolution, and their use is associated with a marked increase in pregnancies among affected women. However, pregnant women were excluded from clinical trials, limiting what is known about maternal and fetal safety. Maternal and fetal outcome tend to be poorer when the disease is severe, but there is limited data on such pregnancies exposed to CFTR modulators. While available data suggest a low risk of birth defects, information on neurological development is lacking and concerns remain regarding lung abnormalities in animal studies, as well as a few cases of congenital cataracts reported in humans. For fetuses affected by CF, the transplacental transfer of CFTR modulators could potentially prevent severe complications, such as meconium ileus, opening promising therapeutic avenues.