作者
Erik A Imel, Francis H Glorieux, Michael P Whyte, Craig F Munns, Leanne M Ward, Ola Nilsson, Jill H Simmons, Raja Padidela, Noriyuki Namba, Hae Il Cheong, Pisit Pitukcheewanont, Etienne Sochett, Wolfgang Högler, Koji Muroya, Hiroyuki Tanaka, Gary S Gottesman, Andrew Biggin, Farzana Perwad, Meng Mao, Chao-Yin Chen, Alison Skrinar, Javier San Martin, Anthony A Portale
发表日期
2019/6/15
期刊
The Lancet
卷号
393
期号
10189
页码范围
2416-2427
出版商
Elsevier
简介
Background
X-linked hypophosphataemia in children is characterised by elevated serum concentrations of fibroblast growth factor 23 (FGF23), hypophosphataemia, rickets, lower extremity bowing, and growth impairment. We compared the efficacy and safety of continuing conventional therapy, consisting of oral phosphate and active vitamin D, versus switching to burosumab, a fully human monoclonal antibody against FGF23, in paediatric X-linked hypophosphataemia.
Methods
In this randomised, active-controlled, open-label, phase 3 trial at 16 clinical sites, we enrolled children with X-linked hypophosphataemia aged 1–12 years. Key eligibility criteria were a total Thacher rickets severity score of at least 2·0, fasting serum phosphorus lower than 0·97 mmol/L (3·0 mg/dL), confirmed PHEX (phosphate-regulating endopeptidase homolog, X-linked) mutation or variant of unknown significance in the patient or a family …
学术搜索中的文章
EA Imel, FH Glorieux, MP Whyte, CF Munns, LM Ward… - The Lancet, 2019
