New progress in treatment of hypophosphatasia
10.3760/cma.j.cn101070-20190917-00882
- VernacularTitle:低磷酸酶血症治疗新进展
- Author:
Yun ZHAO
;
Min LIU
- From:
Chinese Journal of Applied Clinical Pediatrics
2021;36(2):151-154
- CountryChina
- Language:Chinese
-
Abstract:
Hypophosphatasia (HPP) is caused by loss-of-function mutation(s) of the gene that encodes the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). Its manifestations are variable, including impaired skeletal mineralization, altered calcium and phosphate metabolism, recurrent fractures, pain, impaired mobility, premature tooth loss, developmental delay, and seizures.There are different attempts for HPP.Asfotase alfa (Strensiq?), is a bone-targeted recombinant TNSALP which has shown significant improvements in morbidity and mortality in patients with perinatal and infantile hypophosphatasia, and it can improve growth, mobility function and quality of life.This enzyme replacement therapy has generally been well-tolerated, with most adverse reactions being mild-to-moderate in nature.This article reviews recent advances in the treatment of the disease.
