Efficacy and Safety of Burosumab in X-linked Hypophosphatemia
Sen Wang, Xiaoqin Wang, Miao He, Yuanfen Li, Minghui Xiao, Houxun Ma- Biochemistry (medical)
- Clinical Biochemistry
- Endocrinology
- Biochemistry
- Endocrinology, Diabetes and Metabolism
Abstract
Context
Burosumab is approved for the treatment of X-linked hypophosphatemia (XLH).
Objective
To assess the efficacy and safety of burosumab in XLH patients, we conducted a systematic review and meta-analysis.
Methods
We searched PubMed, the Cochrane Library, Embase, ClinicalTrials.gov, and Web of Science for studies on the use of burosumab in patients with XLH. Meta-analysis of randomized controlled trials (RCTs) and single-arm trials (SATs) was done to explore burosumab treatment on the efficacy and safety of XLH.
Results
Of the 8 eligible articles, 5 were from RCTs and 3 were from SATs. Compared with the control group in RCTs, serum phosphorus level was significantly increased in the burosumab group (0.52 mg/dL, 95% CI 0.24-0.80 mg/dL). A meta-analysis of the burosumab arms in all trials revealed significant increase in serum phosphorus levels (0.78 mg/dL, 95% CI 0.61-0.96 mg/dL), TmP/GFR (0.86 mg/dL, 95% CI 0.60-1.12 mg/dL), and 1,25-dihydroxyvitamin D level (13.23 pg/mL, 95% CI 4.82-21.64 pg/mL) as well. Changes in secondary events also validated the effects of burosumab treatment. Compared with the control group, in RCTs, the safety profile of burosumab is not much different from the control group. Data of the single-arm combined group demonstrated the incidence of any treatment emergency adverse event (TEAE) and the related TEAE rate were high, but the severity of most adverse events is mild to moderate, and the rate of serious TEAE is low.
Conclusion
This study suggests that burosumab can be an option for patients with XLH and did not significantly increase the incidence of adverse events.