Reported Adverse Events in a Multicenter Cohort of Patients Ages 6-18 Years with Cystic Fibrosis and at Least One F508del Allele Receiving Elexacaftor/Tezacaftor/Ivacaftor - 22/10/24
, Cristina Fevola, PhD 1, Santiago Presti, MD 2, Alice Castaldo, MD 1, 3, 4, Valeria Daccò, MD 5, Laura Claut, MD 5, Angela Sepe, MD 4, Fabio Majo, MD 6, Rosaria Casciaro, MD 7, Irene Esposito, MD 8, Pamela Vitullo, MD 9, Marta Salvi, MD 10, Patrizia Troiani, MD 11, Francesca Ficili, MD 12, Giuseppe Fabio Parisi, MD 2, Stefano Pantano, MD 13, Stefano Costa, MD 14, Giuseppina Leonetti, MD 15, Nicola Palladino, MD 16, Giovanni Taccetti, MD 1, Paolo Bonomi, BSc 17, Donatello Salvatore, MD 18Abstract |
Objective |
The objective of this study was to describe reported adverse events (AEs) associated with elexacaftor/tezacaftor/ivacaftor (ETI) in a pediatric sample with cystic fibrosis (CF) aged 6-18 years, with at least one F508del variant, followed at multiple Italian CF centers.
Study design |
This was a retrospective, multicenter, observational study. All children receiving ETI therapy from October 2019 to December 2023 were included. We assessed the prevalence and type of any reported potential drug-related AEs, regardless of discontinuation necessity. Persistent AEs were defined as those continuing at the end of the observation period.
Results |
Among 608 patients on ETI, 109 (17.9%) reported at least 1 AE. The majority (n = 85, 77.9%) were temporary, with a median duration of 11 days (range 1-441 days). Only 7 (1.1%) patients permanently discontinued treatment, suggesting good overall safety of ETI. The most common AEs leading to discontinuation were transaminase elevations (temporary 14.1%, persistent 25.9%) and urticaria (temporary 41.2%, persistent 7.4%). Creatinine phosphokinase elevation was uncommon. No significant differences in AEs were observed based on sex, age groups (6-11 vs 12-18 years), or genotype. Pre-existing CF-related liver disease was associated with an increased risk of transaminase elevations. We identified significant variability in the percentage of reported AEs (ANOVA P value .026).
Conclusions |
This real-world study highlights significant variability in reported AEs. Our findings suggest that ETI is a safe and well-tolerated therapy in children and adolescents with CF. However, further long-term safety and effectiveness investigations are warranted.
Le texte complet de cet article est disponible en PDF.Keywords : Trikafta, Kaftrio, children, benign intracranial hypertension, rash, transaminases, management
Abbreviations : aCFLD, AE, ALT, AST, CF, CFHBI, CFTR, CPK, ELX, ETI, IVA, TEZ, ULN
Plan
Vol 274
Article 114176- novembre 2024 Retour au numéro
Article précédent
- National Trends in Inpatient Hospital Outcomes of Children with Osteogenesis Imperfecta and the Importance of Extraskeletal Manifestations: A Kids’ Inpatient Database Study
- Colby Nielsen, R. Reichenbach, Dallin Merrell, Chase Irwin, Reggie C. Hamdy, Mohan V. Belthur
- Molecular Basis and Diagnostic Approach to Isolated and Syndromic Lateralized Overgrowth in Childhood
- Simone Bellucca, Diana Carli, Andrea Gazzin, Stefania Massuras, Simona Cardaropoli, Maria Luca, Paola Coppo, Mirko Caprioglio, Roberta La Selva, Marilidia Piglionica, Piera Bontempo, Gemma D'Elia, Rosanna Bagnulo, Giovanni Battista Ferrero, Nicoletta Resta, Alessandro Mussa
Bienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Déjà abonné à cette revue ?