Burosumab in Chinese adults with X-linked hypophosphatemia: a phase 4 study.

ABSTRACT: X-linked hypophosphatemia (XLH) is a rare genetic disorder, for which burosumab treatment may be beneficial. This study evaluated the efficacy, pharmacokinetics, and safety of burosumab in Chinese adults with XLH. In this open-label, multicenter, postmarketing phase 4 study, 18 Chinese adult outpatients with XLH received conventional oral phosphate (30 mg/kg/d in 5 doses) and calcitriol (0.50 μg/d in 2 doses) during a 14-wk run-in period. Patients then received subcutaneous burosumab 1.0 mg/kg every 4 wk for 48 wk (12 doses). The main outcome measures were changes from baseline in mean serum phosphate level at the end of burosumab treatment, along with patient-reported outcomes. Sixteen (88.9%) and four (22.2%) patients achieved mean serum phosphate levels >2.5 mg/dL at the mid-point and t

SUBMITTER: Liu W 

PROVIDER: S-EPMC12796815 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature