Burosumab improved clinical and biochemical outcomes in patients with tumor-induced osteomalacia.

This hybrid study, comprising a local clinical survey and a systematic review, assessed the efficacy of burosumab in patients affected by tumor-induced osteomalacia (TIO). The investigation included 10 patients from the local survey and 49 cases from the systematic review, sourced from Federico II University and literature databases including Medline and the Cochrane Library. The intervention involved burosumab administration, compared against no treatment.

Regarding primary outcomes, clinical and biochemical improvement was observed in 2 of 2 patients receiving burosumab. In the comparator group, death was observed in 2 of 8 patients not receiving treatment. Secondary outcomes included safety, tolerability, normalization of phosphate serum levels, and reduction of clinical symptoms. The study did not report specific adverse events, serious adverse events, discontinuations, or statistical measures such as p-values or confidence intervals.

Safety and tolerability were described as well tolerated, though specific adverse event data were not reported. The study lacked reported follow-up duration and formal causality assessments. Key limitations include the small sample size of the local survey and the absence of statistical reporting for the primary efficacy outcome.

The practice relevance suggests burosumab may be administered in cases of tumor identification while awaiting surgery or when surgical inoperability exists to manage hormonal FGF23 effects. Clinicians should interpret these results with caution due to the observational nature of the data and the lack of rigorous statistical analysis.