Cystic fibrosis is an autosomal recessive genetic disease. It affects multiple organs, but the most serious consequences are in the lungs. Around 90% of patients succumb to the progression of this lung disease.
Experimental studies have demonstrated the role of the TMEM16A antibody in the production and secretion of mucus.
Benzbromarone, a uricosuric agent used to treat chronic gout, has been shown to inhibit TMEM16A activity in animal models, suggesting potential therapeutic benefits by reducing mucus production and the severity of this disease.
The study was a prospective, open-label pilot trial involving ten cystic fibrosis patients followed up at a tertiary referral centre in southern Brazil. These patients received benzbromarone 100 mg/day orally for 90 days. Inclusion criteria included a positive sweat chloride test (>60 mEq/L), two identified pathogenic variants in the CFTR gene, and a predicted FEV1% of less than 90%. Exclusion criteria included pregnancy, initiation of new therapy, use of CFTR modulators, and pulmonary exacerbations in the four weeks prior to the study.
The primary endpoint was benzbromarone safety, assessed by pulmonary exacerbations, vital signs, physical examinations, haematological and coagulation markers, and drug-related adverse events.
Spirometry tests (FEV1%) were performed at screening, at baseline (day 1), and during follow-up (at 30, 60 and 90 days). Changes in FEV1% were assessed by comparing the values obtained at Day 1 with those obtained at the final visit.
This pilot study was the first to evaluate the use of oral benzbromarone as an adjuvant treatment for cystic fibrosis. Benzbromarone was found to be safe and well tolerated.
The results indicated an increase in FEV1%, suggesting a potential therapeutic benefit.
These results are consistent with preclinical studies showing that inhibition of TMEM16A could reduce mucus production and bronchial hyperresponsiveness. However, larger-scale studies are required to confirm these results and determine the efficacy of benzbromarone as adjuvant therapy.
Oral administration of benzbromarone appears to be a safe therapeutic option with a trend towards improved lung function in cystic fibrosis patients.
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