Frontiers in endocrinology · 2026
Burosumab an emerging therapy in TIO: a local clinical survey and a systematic review with individual patient data analysis.
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Vergatti Anita, Abate Veronica, Zarrella Aquilino F, Fieramosca Natalia, De Filippo Gianpaolo, Merlotti Daniela, Gennari Luigi, Menale Ciro, D'Elia Lanfranco, Rendina Domenico
Аннотация
INTRODUCTION: Burosumab, a recombinant antibody against fibroblast growth factor 23 (FGF23), is a new therapeutical option for Tumor induced osteomalacia (TIO). To estimate the clinical need and to evaluate the efficacy and safety of burosumab, we conducted a hybrid study composed by a clinical survey and a systematic review.
METHODS: All patients referring for TIO to the Federico II University were enrolled in our clinical survey. A comprehensive literature search on a Medline, Google Scholar, Google Books, and the Cochrane Library was conducted for the Systematic Review. Data were extracted on 12/31/2025.
RESULTS: For the local survey, we collected 10 patients affected by TIO [M: F = 5 (50.0%): 5 (50.0%); mean age at symptom onset 57.6 ± 14.3 years]. Two (20.0%) patients received burosumab, obtaining clinical and biochemical improvement. Two who didn't receive the treatment died. For the systematic review, we collected 42 studies and 49 cases [M: F = 19 (44.2%): 24 (55.8%); mean age at onset 36.8 ± 21.3 years; mean age at diagnosis 44.5 ± 22.9 years]. Burosumab reduced clinical symptoms and ameliorates biochemical investigations by normalizing phosphate serum levels. It is also safe and well tolerated.
CONCLUSION: Burosumab represents a safe and efficient treatment in TIO patients, to be administered in case of tumor identification while waiting for surgery, and in case of surgical inoperability to manage hormonal FGF23 effect.
METHODS: All patients referring for TIO to the Federico II University were enrolled in our clinical survey. A comprehensive literature search on a Medline, Google Scholar, Google Books, and the Cochrane Library was conducted for the Systematic Review. Data were extracted on 12/31/2025.
RESULTS: For the local survey, we collected 10 patients affected by TIO [M: F = 5 (50.0%): 5 (50.0%); mean age at symptom onset 57.6 ± 14.3 years]. Two (20.0%) patients received burosumab, obtaining clinical and biochemical improvement. Two who didn't receive the treatment died. For the systematic review, we collected 42 studies and 49 cases [M: F = 19 (44.2%): 24 (55.8%); mean age at onset 36.8 ± 21.3 years; mean age at diagnosis 44.5 ± 22.9 years]. Burosumab reduced clinical symptoms and ameliorates biochemical investigations by normalizing phosphate serum levels. It is also safe and well tolerated.
CONCLUSION: Burosumab represents a safe and efficient treatment in TIO patients, to be administered in case of tumor identification while waiting for surgery, and in case of surgical inoperability to manage hormonal FGF23 effect.