INTRODUÇÃO: Cystic fibrosis (CF) affects several organs, but the most severe consequences are observed in the lung. Reduced lung function in CF patients is associated with increased morbidity and mortality. Despite significant progress in the development of CFTR–specific treatments for CF lung disease, it appears reasonable to search for alternative drug targets in CF.
OBJETIVO: We propose to evaluate the safety and efficacy of oral Benzbromarone as adjuvant therapy in CF patients with refractory reduced lung function.
METODOLOGIA: A prospective, open-label pilot study of oral benzbromarone (100 mg/day) administered once/day for 90 days. Patients were followed in the reference center in southern Brazil. The primary outcome was safety measured by reported adverse events (AEs). Secondary outcomes were forced expiratory volume in 1 s (FEV1) and pulmonary exacerbations (PE). Local institutional review boards approved the protocol, and written informed consent was obtained from all patients.
RESULTADOS: Ten subjects were recruited. Benzbromarone was safe, and no drug-related AEs were observed during the study. Eight patients concluded the study, the median of relative change FEV1% values tended to increase during treatment, with an 8% increase from baseline at the final visit. The non-parametric test did not reveal a significant difference in the change described (p = 0.06). Only 1/8 of patients experienced at least one PE during the study.
CONCLUSÃO: Oral Benzbromarone is safe and potentially led to an increase in FEV1%. This is the first study evaluating oral Benzbromarone safety and efficacy in patients with CF. Further evaluation in more extensive trials should be performed to elucidate whether oral Benzbromarone can serve as a potential adjunctive therapy for CF.
