Department of Endocrinology, Seth G.S. Medical College & KEM Hospital, Mumbai, Maharashtra, India.
Department of Child Health, Christian Medical College, Vellore, Tamil Nadu, India.
Arch Endocrinol Metab. 2024 May 17;68:e230242. doi: 10.20945/2359-4292-2023-0242.
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, < 0.001, improvement in deformities, and decline in serum alkaline phosphatase levels [SMD: 130.68, 95% CI: 125.26-136.1, < 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, < 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.
布罗索尤单抗是一种针对成纤维细胞生长因子 23（FGF23）的单克隆抗体，已被批准用于治疗 X 连锁低磷血症（XLH）。我们进行了一项系统评价，比较了布罗索尤单抗与传统疗法（磷和骨化三醇）在 XLH 治疗中的疗效和安全性。在对 MEDLINE/PubMed 和 Embase 进行全面文献检索后，我们找到了 9 项研究纳入分析。评估了偏倚风险，并使用随机效应模型确定了效应大小。分析了治疗前后疾病严重程度的临床、生化和影像学参数，并以标准化均数差（SMD）表示。布罗索尤单抗可使磷酸盐内稳态正常化，增加肾小管磷酸盐重吸收，并显著缓解骨骼病变（Thacher 总佝偻病严重程度评分变化 SMD：-1.46，95%置信区间 [CI]：-1.76 至-1.17，<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，<0.001]）。传统疗法在所有这些参数上也导致了类似的改善，但程度较低。在成人中，布罗索尤单抗可使磷水平正常化（SMD：1.23，95%CI：0.98-1.47，<0.001），从而改善临床症状。布罗索尤单抗治疗耐受性良好，仅有轻度治疗相关不良反应。本综述表明布罗索尤单抗在改善 XLH 儿童佝偻病、畸形和生长方面具有潜在作用。鉴于其优越的疗效和安全性，布罗索尤单抗可能成为儿童的有效治疗选择。我们建议进一步研究比较布罗索尤单抗与 XLH 儿童和成人的传统疗法。
