Effectiveness of treatment with triple therapy of CFTR modulating drugs in patients with cystic fibrosis with f508del mutation and/or minimal CFTR gene function: systematic review and meta-analysis

Identifier:  TFG:7689

Handle: https://hdl.handle.net/20.500.11797/TFG7689

Authors:  Herrerías Dávila, Paula

Abstract:
Cystic fibrosis is an autosomal recessive genetic disease characterized by persistent lung infections, pancreatic insufficiency and high levels of chloride in sweat, caused by mutations in the CFTR protein, the most common being F508del. Treatments include antibiotics, physical therapy, and CFTR modulators. This study evaluated the effectiveness of triple therapy with CFTR modulators in patients ≥ 6 years of age with F508del and/or minimal function mutations. 8 clinical trials with 1243 participants were analyzed, showing significant improvements in lung function (ppFEV1), sweat chloride and the CFQ-R questionnaire. Triple therapy proved to be superior.