Efficacy and Safety of Burosumab in X-linked Hypophosphatemia

作者全名："Wang, Sen; Wang, Xiaoqin; He, Miao; Li, Yuanfen; Xiao, Minghui; Ma, Houxun"

作者地址："[Wang, Sen; Wang, Xiaoqin; Li, Yuanfen; Xiao, Minghui; Ma, Houxun] Chongqing Med Univ, Dept Geriatr, Affiliated Hosp 1, Chongqing 400016, Peoples R China; [He, Miao] Shanxi Med Univ, Dept Neurol, Clin Med Coll 9, Taiyuan 030000, Peoples R China"

通信作者："Ma, HX (通讯作者)，Chongqing Med Univ, Dept Geriatr, Affiliated Hosp 1, 1 Youyi St, Chongqing 400016, Peoples R China."

来源：JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

ESI学科分类：CLINICAL MEDICINE

WOS号：WOS:001187054800020

JCR分区：Q1

影响因子：5.8

年份：2023

卷号：109

期号：1

开始页：293

结束页：302

文献类型：Review

关键词：burosumab; X-linked hypophosphatemia; monoclonal antibody; fibroblast growth factor 23; serum phosphorus; adverse event; meta-analysis

摘要："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."

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