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目的评价重组人类生长激素rhGH治疗儿童X-连锁低血磷性佝偻病(XLH)的临床疗效与安全性。方法检查Cochrane囊性纤维化及遗传性疾病工作组建立的先天性代谢缺陷数据库。手检相关骨和矿物质代谢的杂志和会议摘要。纳入所有单独采用重组人类生长激素或联合传统疗法与安慰剂或单用传统疗法治疗儿童XLH作比较的随机对照试验或半随机对照试验。2个评价员独立选择纳入的试验、评价试验的方法学质量及提取数据。结果只有1个研究符合该系统评价的纳入标准,且样本量小。rhGH治疗可能改善XLH患儿身高增长,并可因暂时减少尿磷的排出而提高血磷水平。结论尚没有足够的证据显示rhGH是否对患儿的身高增长、矿物质代谢、内分泌、肾功能、骨矿密度和上下部量比例有影响。
Objective To evaluate the clinical efficacy and safety of recombinant human growth hormone rhGH in the treatment of children with X-linked hypophosphatemic rickets (XLH). METHODS: The inborn metabolic defect database established by the Cochrane Cystic Fibrosis and Hereditary Disease Working Group was examined. Hand-check summary of magazines and conferences related to bone and mineral metabolism. All randomized controlled trials or quasi-randomized controlled trials comparing all XLH patients treated with recombinant human growth hormone alone or in combination with conventional therapy with placebo or with conventional therapy alone were included. Two reviewers independently selected included trials to evaluate the methodological quality of the trial and data extraction. Results Only one study met the inclusion criteria for this systematic review with a small sample size. Treatment with rhGH may improve height growth in children with XLH and may increase serum phosphate levels by temporarily reducing urinary phosphate excretion. Conclusions There is not yet enough evidence to show whether rhGH affects the children’s height growth, mineral metabolism, endocrine, renal function, bone mineral density and the proportion of upper and lower parts.