PURPOSE: Febuxostat, a nonpurine selective inhibitor of xanthine oxidase has elucidated an effective urate-lowering in adults. However, data on febuxostat utility in children is limited. The present study summarizes the current knowledge on the efficacy and safety of febuxostat in children with hyperuricemia. METHODS: We searched PubMed, Google Scholar, Embase, and Web of Science from inception to September 2024 for studies assessing febuxostat or comparing febuxostat with allopurinol in children and adolescents with hyperuricemia with diverse etiologies. Randomized controlled trials, prospective observational, and systematic reviews, meta-analyses, and retrospective studies were included. FINDINGS: Search results illuminated three studies assessing febuxostat in children with hyperuricemia of different etiologies
including tumor lysis syndrome (TLS), asymptomatic hyperuricemia, and gout. An open-label observational study and one-retrospective study assessed febuxostat efficacy for asymptomatic hyperuricemia and gout, respectively. There was one retrospective study that compared febuxostat to allopurinol for TLS prevention. IMPLICATIONS: Febuxostat is a promising medication that is effective in attaining the desired outcomes in children with hyperuricemia. However, existing evidence does not permit any conclusion regarding the comparative efficacy and safety of febuxostat with allopurinol in children. Future randomized clinical trials evaluating its effectiveness and safety are needed.