New advance in hypophosphatemic rickets
10.3760/cma.j.issn.2095-428X.2019.17.006
- VernacularTitle: 低磷性佝偻病研究新进展
- Author:
Guixia DING
1
Author Information
1. Department of Nephrology, Children′s Hospital of Nanjing Medical University, Nanjing 210008, China
- Publication Type:Journal Article
- Keywords:
Rickets;
Hypophosphatemia;
Fibroblast growth factor 23;
Osteomalacia;
1, 25(OH)2D
- From:
Chinese Journal of Applied Clinical Pediatrics
2019;34(17):1304-1308
- CountryChina
- Language:Chinese
-
Abstract:
Hypophosphatemic rickets(HR) is a bone mineralization disorder caused by phosphate wasting, including hypophosphatemia, bone abnormalities and short stature.X-linked hypophosphatemia(XLH) is the most common inherited disease related to phosphate metabolism, which might result in elevated levels of fibroblast growth factor 23 (FGF23). FGF23 plays an important role in the disease mechanism, so a human anti-FGF23 antibody is developed as a potential treatment for XLH.In many clinical trials, subcutaneous Burosumab increased serum phosphorus levels in pediatric and adult patients with XLH.With the development of phase 1-3 clinical trials of Burosumab in children and adults with XLH, the efficacy and safety of Burosumab is proven to be superior to that of phosphate and calcitriol used in traditional therapy.This review aims to investigate the physiopathology and treatment of HR and to enhance the recognition of HR.