Lyseng-Williamson Katherine A
Springer, Private Bag 65901, Mairangi Bay, 0754 Auckland, New Zealand.
Drugs Ther Perspect. 2018;34(11):497-506. doi: 10.1007/s40267-018-0560-9. Epub 2018 Oct 8.
Burosumab (Crysvita), a fully human IgG1 monoclonal antibody directed at fibroblast growth factor 23 (FGF23), is indicated for the treatment of X-linked hypophosphatemia (XLH), a condition associated with excessive FGF23 production. It directly addresses the excessive FGF23 activity in patients with XLH by binding to FGF23, and inhibiting its signaling. This leads to increased gastrointestinal phosphate absorption and renal phosphate reabsorption, thereby improving serum phosphate levels, and, ultimately, bone mineralization and the risk of bone disease. In clinical trials, subcutaneous burosumab increased serum phosphorus levels in pediatric and adult patients with XLH, as well as significantly improving the severity of rickets in children, and improving pain, stiffness, physical functioning, and fracture/pseudofracture healing in adults. Burosumab is well tolerated by children and adults with XLH, with most treatment-emergent adverse events being of mild to moderate severity.
布罗索尤单抗（Crysvita）是一种针对成纤维细胞生长因子23（FGF23）的全人IgG1单克隆抗体，适用于治疗X连锁低磷血症（XLH），这是一种与FGF23产生过多相关的病症。它通过与FGF23结合并抑制其信号传导，直接解决XLH患者中FGF23活性过高的问题。这导致胃肠道磷吸收增加和肾脏磷重吸收增加，从而提高血清磷水平，并最终改善骨矿化和骨病风险。在临床试验中，皮下注射布罗索尤单抗可提高XLH儿科和成人患者的血清磷水平，显著改善儿童佝偻病的严重程度，并改善成人的疼痛、僵硬、身体功能以及骨折/假性骨折愈合情况。XLH儿童和成人对布罗索尤单抗耐受性良好，大多数治疗中出现的不良事件为轻度至中度严重程度。
