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Published on: February 11, 2017
Treatment with CFTR Modulators for Cystic Fibrosis: What a Pediatric Gastroenterologist Needs to Know
David Gonzalez Jimenez1, Ruth García Romero2, Alejandro Rodríguez Martínez3
1Cystic Fibrosis Unit, Hospital Universitario Central de Asturias, 33011 Oviedo, Spain.
CFTR modulators improve gastrointestinal and nutritional health in cystic fibrosis patients. Pediatric gastroenterologists must monitor digestive and bone health outcomes with these new therapies.
Area of Science:
- Pediatric Gastroenterology
- Genetics
- Pharmacology
Background:
- Cystic fibrosis (CF) is a genetic disorder impacting multiple organ systems.
- CFTR modulators, particularly elexacaftor/tezacaftor/ivacaftor (ETI), are transforming CF care.
- Emerging GI and nutritional issues require attention in pediatric CF patients on modulators.
Purpose of the Study:
- To review the impact of CFTR modulators on pediatric GI, nutritional, bone, and hepatobiliary health.
- To assess emerging challenges and benefits of modulator therapy in children with CF.
- To inform pediatric gastroenterologists on monitoring and management strategies.
Main Methods:
- Conducted a narrative review of the literature.
- Searched PubMed, EMBASE, and Scopus databases.
- Included 68 relevant articles in English or Spanish after full-text review.
Main Results:
- CFTR modulators show potential for improved GI function, nutrient absorption, and weight gain.
- ETI therapy in children is linked to increased lean mass and vitamin levels.
- Variable liver and bone density outcomes necessitate careful monitoring.
Conclusions:
- CFTR modulators offer systemic benefits for CF, but long-term pediatric GI and skeletal effects require further study.
- Pediatric gastroenterologists are crucial for monitoring outcomes and guiding personalized care.
- Ongoing research is needed to fully understand the systemic impact of CFTR modulators in children.
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