1 - Pediatrics Department,, Hospital Dona Estefânia, Centro Hospitalar Universitário Lisboa Central, Lisbon, Portugal,
2 - Gastrenterology And Heptology Unit, Hospital Dona Estefânia, Centro Hospitalar Universitário Lisboa Central, Lisbon, Portugal,
3 - Hospital Dona Estefânia, Centro Hospitalar Universitário Lisboa Central, Lisbon, Portugal,
4 - Pediatric Pneumology Unit, Hospital Dona Estefânia, Centro Hospitalar Universitário Lisboa Central, Lisbon, Portugal
- Poster, 56th Annual Meeting of the European Society for Pediatric Gastroenterology, Hepatology and Nutricion (ESPGHAN), Milão, 15-18 de maio 2024
RESUMO:
Objectives and Study: Cystic fibrosis (CF) is a genetic disorder stemming from alterations in the CFTR protein. Pharmacological intervention in CFTR protein synthesis and function has demonstrated effectiveness and safety. This study seeks to characterize the gastrointestinal (GI) and hepatic effects within the first year of modulator therapy in CF patients.
Methods: In a retrospective analysis of clinical, laboratory, and imaging data over the initial 12 months of modulator therapy, we performed descriptive and comparative statistical analyses on patients using Ivacaftor (IVA), Lumacaftor+Ivacaftor (LUMA-IVA), and Elexacaftor+Tezacaftor+Ivacaftor (ETI) with SPSS®.
Results: Out of 27 CF patients, 16 were eligible, and 13 were on modulator therapy: LUMA-IVA 43.7%, ETI 50%, and IVA 6.3%. The median age at treatment onset was 10.1 years. Clinically, GI side effects were observed at 3 months, specifically diarrhea in 12.5% and abdominal pain in 6.3%. One patient underwent surgery due to distal intestinal obstruction syndrome. Another patient with pre-existing liver disease experienced worsening fibrosis in elastography. Two patients with normal elastography at the beginning showed values compatible with mild fibrosis after one year. After 12 months, a two-fold elevation of AST and ALT levels was observed in 27% and 9% of patients, significantly higher than pre-therapy levels (p<0.01). ETI use was associated with higher bilirubin levels at 3, 6, 9, and 12 months (p<0.05) and lower platelet counts at 3, 9, and 12 months (p<0.05) compared to other modulator therapies. No significant variations were recorded between different therapies regarding other laboratory and ultrasound findings or clinical outcomes. Therapy was not discontinued in any patient.
Conclusions: In this cohort, the most common GI effects were diarrhea and abdominal pain, with no therapy discontinuation. The ETI combination was associated with higher bilirubin values and lower platelet counts, emphasizing the need for clinical consideration.