Prenatal CFTR modulator therapy and fetal meconium ileus in cystic fibrosis: a systematic review and individual patient data meta-analysis.

Authors

Vincenzo G Menditto, Giovanni Pomponio, Nicole Caporelli, Marica Bordicchia, Alessandra Boni, Marco Bruno Luigi Rocchi, Giacomo Menditto, Benedetta Fabrizzi

Published in

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. Aug 22, 2026. Epub Aug 22, 2026.

Abstract

Fetal meconium ileus (MI) is a clinically relevant manifestation of cystic fibrosis (CF). We conducted a systematic review with meta-analysis to evaluate the efficacy of prenatal CFTRm therapy, administered to carrier mothers or mothers with CF, for the prevention or treatment of fetal MI. The primary outcome was the occurrence of MI requiring surgical intervention.
A systematic search was conducted in PubMed/MEDLINE, Embase, SCOPUS and the Cochrane Library. In-vitro and animal studies were excluded. Reporting quality and the risk of bias of the cohort studies was assessed using the NOS Scale and the CARE checklist, as appropriate, and the ROBINS-I V2, respectively. An individual patient data meta-analysis and meta-regression were performed.
We included 18 studies. Among 42 fetuses antenatally diagnosed with MI and exposed to CFTRm, 12% (95% CI, 6%-25%) required surgery after birth. Comparison with a historical cohort suggested a possible 59.5% absolute reduction in the risk of surgery (95% CI, 44%-69%). None of the nine asymptomatic fetuses exposed to treatment developed MI. On meta-regression, the diagnosis of MI in second trimester was associated with lower odds of surgery (OR 0.05, 0.01-0.50; p = 0.011), whereas maternal treatment duration shorter than 5 weeks was associated with increased odds (OR 15.15, 1.41-162.73; p = 0.025).
Prenatal exposure to CFTRm appears to be effective in treating or preventing MI in fetuses with CF. However, the evidence is limited to case reports or small observational studies and prospective controlled studies are needed.

PMID:
42632793
Bibliographic data and abstract were imported from PubMed on 23 Aug 2026.

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