Offering prenatal CFTR modulators to prevent cystic fibrosis-related organ damage in utero.

This case series from one center describes eight pregnancies at risk for cystic fibrosis in which prenatal CFTR modulator therapy (elexacaftor/tezacaftor/ivacaftor) was offered, with only two women ultimately receiving it. In both treated cases, fetal bowel obstruction findings improved or resolved and the infants were born without meconium ileus, though the small sample and lack of a control group limit any conclusions about efficacy. The authors conclude that decisions about prenatal modulator therapy are complicated by variable genotype-phenotype relationships, inconsistent insurance coverage, and uncertain risks and benefits.