Neuropsychological effects of therapy with CFTR modulators in patients with cystic fibrosis

Abstract

Objective: To evaluate the main hypotheses regarding neuropsychological adverse events reported by patients undergoing treatment with CFTR modulators in cystic fibrosis. Methods: An integrative literature review was conducted in six stages, following the PICO protocol for formulating the research question. The search was performed on the Virtual Health Library (BVS) and Google Scholar platforms using descriptors combined with Boolean operators. Strict inclusion and exclusion criteria were applied. The study selection and screening process was illustrated using the PRISMA flowchart. Results: Seven articles were included and synthesized based on the lead author, level of evidence, and main findings, according to the Oxford Centre for Evidence-based Medicine. Neuropsychological side effects associated with CFTRm treatment were reported. Pathophysiological hypotheses include collateral action on the 5-HT2c receptor, potentiation of the BKCa channel in neuronal cells, CYP3A4 metabolism, and inflammatory responses involving glial cells. Final considerations: Despite adverse events, the benefits outweigh the risks. Dose adjustments should be considered, although they are not formally described by the pharmaceutical company. Further investigations are essential to better guide clinical practice and assess these impacts more comprehensively and over the long term.