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 the end of the dose cycle, respectively. The mean (SD) serum phosphate level increased from 1.6 (0.25) mg/dL at baseline to 2.4 (0.28) mg/dL at the end of the dose cycle. The mean (SD) change from baseline in serum phosphate level was 50.1% (14.11%) following burosumab treatment. The tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR) increased from 1.3 (0.32) mg/dL at week 0 to 2.0 (0.42) mg/dL at week 48. Over 48 wk, patients reported that their worst pain, severity, interference, and stiffness were relieved and Measurement Information System quality of life assessment scores increased. Motor function assessed by the 6-min walking test improved…
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Evidence Hub reads this as a clinical trial, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence.
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