Efficacy and safety of febuxostat in Japanese paediatric patients with hyperuricaemia including gout: Phase 2, single arm, open-label, multicentre studies

Objectives Urate-lowering efficacy and safety of febuxostat was evaluated in paediatric patients with hyperuricaemia including gout. Methods A Phase 2 study of febuxostat in paediatric patients aged 6-18 years with hyperuricaemia including gout was conducted. We evaluated the proportion of patients achieving serum uric acid (sUA) level <= 6.0 mg/dl at Week 26, and long-term safety and efficacy at Week 52. We also considered efficacy stratified by renal function. Results Thirty patients (10 at <40 kg and 20 at >= 40 kg) were enrolled. Twenty-four were males, 29 had asymptomatic hyperuricaemia, and 1 had gout. Age was 8 to 18 years. Of these, 63.3% (95% confidence interval 43.9-80.1%) achieved a sUA level of <= 6.0 mg/dl at Week 26. sUA level (mean +/- standard deviation) was 5.55 +/- 0.87 mg/dl, reduced from 9.01 +/- 1.23 mg/dl at baseline. Febuxostat efficacy appeared similar for mild to moderate renal dysfunction and with normal renal function. There were no major safety issues. Conclusions In paediatric patients with hyperuricaemia including gout, febuxostat showed long-term, well-controlled urate-lowering efficacy with no major safety issues. Findings suggest that no dose adjustment is required for paediatric patients with mild to moderate renal dysfunction.