Benzbromarone as adjuvant therapy for cystic fibrosis lung disease: a pilot clinical trial

• Published in: Jornal brasileiro de pneumologia Vol. 50; no. 3; p. e20230292
• Main Authors: Friedrich, Frederico, Petry, Lucas Montiel, Garcia, Laura de Castro E, Pieta, Marina Puerari, Meneses, Amanda da Silva, Bittencourt, Luana Braga, Xavier, Luiza Fernandes, Antunes, Marcos Otávio Brum, Grun, Lucas Kich, Lumertz, Magali, Kunzelmann, Karl, Pinto, Leonardo Araujo
• Format: Journal Article
• Language: English
• Published: Brazil Sociedade Brasileira de Pneumologia e Tisiologia 2024
• Subjects: Adolescent; Adult; Benzbromarone - administration & dosage; Benzbromarone - therapeutic use; Chemotherapy, Adjuvant; Cystic Fibrosis - drug therapy; Cystic Fibrosis - physiopathology; Cystic fibrosis/therapy; Female; Forced Expiratory Volume - drug effects; Humans; Male; Mucociliary clearance; Original; Pilot Projects; Prospective Studies; RESPIRATORY SYSTEM; respiratory tract diseases; Statistics, Nonparametric; Time Factors; Treatment Outcome; Uricosuric Agents - therapeutic use; Young Adult; Mucociliary clearance; Cystic fibrosis/therapy; respiratory tract diseases
• ISSN: 1806-3756; 1806-3713; 1806-3756
• DOI: 10.36416/1806-3756/e20230292

Cystic fibrosis (CF) affects multiple organs, the most severe consequences being observed in the lungs. Despite significant progress in developing CF transmembrane conductance regulator-specific treatments for CF lung disease, exploring alternative CF-targeted medications seems reasonable. We sought to evaluate the potential beneficial effects of oral benzbromarone as an adjuvant therapy in CF patients with reduced lung function. This was a prospective open-label pilot study of oral benzbromarone (100 mg/day) administered once daily for 90 days. Patients were followed at a tertiary referral center in southern Brazil. Safety was assessed by the number of reported adverse events. Secondary objectives included percent predicted FEV1 (FEV1%) and pulmonary exacerbations. Ten patients were enrolled. Benzbromarone was found to be safe, with no serious drug-related adverse events. Eight patients completed the study; the median relative change in FEV1% tended to increase during the treatment, showing an 8% increase from baseline at the final visit. However, a nonparametric test showed that the change was not significant (p = 0.06). Of a total of ten patients, only one experienced at least one pulmonary exacerbation during the study. Oral benzbromarone appears to be safe, and improved FEV1% has been observed in patients with CF. Further assessment in larger trials is warranted to elucidate whether oral benzbromarone can be a potential adjuvant therapy for CF.