PDF(Pubmed)
Abstract：
Burosumab, a monoclonal antibody directed against the fibroblast growth factor 23 (FGF23), has been approved for the treatment of X-linked hypophosphatemia (XLH). We conducted a systematic review to compare the efficacy and safety of burosumab versus conventional therapy (phosphorus and calcitriol) on XLH treatment. After a comprehensive literature search on MEDLINE/PubMed and Embase, we found nine studies for inclusion in the analysis. Risk of bias was assessed, and a random-effects model was used to determine the effect size. Clinical, biochemical, and radiological parameters of disease severity before and after treatment were analyzed and expressed in standardized mean difference (SMD). Burosumab resulted in normalization of phosphate homeostasis with an increase in renal tubular phosphate reabsorption and significant resolution of skeletal lesions (change in Thacher\'s total rickets severity score SMD: -1.46, 95% confidence interval [CI]: -1.76 to -1.17, p < 0.001, improvement in deformities, and decline in serum alkaline phosphatase levels [SMD: 130.68, 95% CI: 125.26-136.1, p < 0.001)]. Conventional therapy led to similar improvements in all these parameters but to a lower degree. In adults, burosumab normalized phosphorus levels (SMD: 1.23, 95% CI: 0.98-1.47, p < 0.001) with resultant clinical improvement. Burosumab treatment was well tolerated, with only mild treatment-related adverse effects. The present review indicates a potential role for burosumab in improving rickets, deformities, and growth in children with XLH. Given its superior efficacy and safety profile, burosumab could be an effective therapeutic option in children. We suggest further studies comparing burosumab versus conventional therapy in children and adults with XLH.
摘要：
Burosumab,一种针对成纤维细胞生长因子23（FGF23）的单克隆抗体，已被批准用于X连锁低磷酸盐血症（XLH）的治疗。我们进行了系统评价，以比较burosumab与常规治疗（磷和骨化三醇）对XLH治疗的疗效和安全性。经过对MEDLINE/PubMed和Embase的全面文献检索，我们发现9项研究纳入分析.评估了偏见的风险，并使用随机效应模型来确定效应大小。临床,生物化学，对治疗前后疾病严重程度的放射学参数进行分析，并以标准化均差（SMD）表示。Burosumab导致磷酸盐体内平衡正常化，肾小管磷酸盐重吸收增加，骨骼病变显著消退（Thacher病总严重程度评分变化SMD：-1.46，95％置信区间[CI]：-1.76至-1.17，p<0.001，畸形改善，血清碱性磷酸酶水平下降[SMD:130.68,95%CI:125.26-136.1,p<0.001]。常规治疗导致所有这些参数的类似改善，但程度较低。在成年人中,burosumab标准化的磷水平（SMD：1.23，95％CI：0.98-1.47，p<0.001），最终的临床改善。Burosumab治疗耐受性良好，只有轻微的治疗相关的不良反应。本综述表明burosumab在改善病方面的潜在作用，畸形,和XLH儿童的成长。鉴于其优越的疗效和安全性，burosumab可能是儿童的有效治疗选择。我们建议进一步研究比较burosumab与XLH儿童和成人的常规治疗。
