Efficacy and safety of using Elexacaftor/Tezacaftor/Ivacaftor in the treatment of children with cystic fibrosis: real-world evidence from Brazil
ETI demonstrated effectiveness in improving weight gain, reducing pulmonary exacerbations, and significantly lowering sweat chloride concentration. The treatment was well-tolerated, with a favorable safety profile. These findings align with existing literature, supporting ETI's role as a transformative therapy in pediatric CF management.
Authors
- Nelson Augusto Rosário Filho (ORCID: https://orcid.org/0000-0002-8550-8051)
- Guilherme da Silva Martins (ORCID: https://orcid.org/0000-0002-8287-8460)
- Luiz Vicente Ribeiro Ferreira da Silva Filho (ORCID: https://orcid.org/0000-0001-8431-1997)
- Herberto José Chong‐Neto (ORCID: https://orcid.org/0000-0002-7960-3925)
- Débora Carla Chong‐Silva (ORCID: https://orcid.org/0000-0002-7385-4598)
- Carlos Antônio Riedi (ORCID: https://orcid.org/0000-0002-6044-3327)
- Carlos Roberto Lebarbenchon Massignan
- Gabriela Spessatto
- Juliana Gonçalves Primon
- Thalita Gonçalves Picciani
- Maitê Milagres Saab
- Aline Didoni Fajardo
- Roberta Corrêa da Cunha
- Bruno Hernandes David João
- Carolina Rambo
Institutions
- Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (BR)
- Universidade Federal do Paraná (BR)
Publication Details
- Journal
- Jornal de Pediatria
- Published
- 2025-06-24
- DOI
- https://doi.org/10.1016/j.jped.2025.05.007
- Citations
- 3
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 4.56