asfotase alfa用于罕见病低碱性磷酸酶血症的文献综述

A review of the application of asfotase alfa in the rare disease hypophosphatasia

低碱性磷酸酶血症(hypophosphatasia, HPP)是一种罕见的以骨骼和(或)牙齿矿化障碍,伴有血清碱性磷酸酶活性降低为特征的遗传性内分泌系统疾病。asfotase alfa(AA)作为重组骨靶向人非特异性碱性磷酸酶获得美国FDA孤儿药认证,并于2015年上市用于HPP的治疗。本文就AA的作用机制、药动学特点、有效性、安全性及用法用量进行综述,为该药物用于HPP患者提供参考和证据支持。

Hypophosphatasia(HPP)is a rare hereditary endocrine system disease characterized by bone and/or tooth mineralization disorders accompanied by decreased serum alkaline phosphatase activity.Asfotase alfa(AA)received orphan drug approval by FDA as a recombinant bone-targeted human nonspecific alkaline phosphatase and was marketed in 2015 for the treatment of HPP.This paper reviews the mechanism of action,pharmacokinetic characteristics,efficacy,safety,usage and dosage of AA to provide reference and evidence support for the use of AA in the patients with HPP.