# Antenatal CFTR Modulators to Treat a Healthy Pregnant Woman with a Fetus Affected by Cystic Fibrosis Complicated by Meconium Ileus and Intestinal Volvulus: From a Suspicion of the Disease to a Targeted Treatment in Utero: Case Report and Narrative Review

## TL;DR

This paper discusses the use of CFTR modulators during pregnancy to treat a fetus with cystic fibrosis and related complications, based on a case report and literature review.

## Contribution

The paper presents a new case report and highlights key elements for successful in utero treatment of fetal cystic fibrosis with CFTR modulators.

## Key findings

- The case represents the fifth unsuccessful attempt of in utero CFTR modulator treatment for fetal cystic fibrosis.
- The paper identifies potential key factors necessary for successful treatment outcomes.
- The safety and effectiveness of CFTR modulators during pregnancy remain areas requiring further study.

## Abstract

The introduction of cystic fibrosis transmembrane conductance regulator modulators (CFTRms) in the treatment of cystic fibrosis (CF) has significantly improved both the life expectancy and quality of life for patients affected by the disease. While this new therapy shows promising results in CF patients, data about the use of CFTRms during pregnancy in women who are carriers with fetuses affected by CF are limited. We present a new case report and literature review of fetal CF treated by the maternal assumption of CFTRms to understand the safety and the effectiveness of the treatment in resolving fetal and neonatal CF clinical manifestations (meconium ileus, postnatal exocrine pancreatic insufficiency, and postnatal intestinal surgery). Our report represents the fifth unsuccessful case of fetal CF treated in utero by CFTRms and highlights the potential key elements necessary for a successful treatment.