中国实用儿科杂志

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改善矮小儿童成年身高的药物研究进展

  

  1. 1.浙江大学医学院附属第一医院儿科,浙江  杭州  310003; 2.浙江大学医学院附属儿童医院,浙江  杭州  310058
  • 出版日期:2020-06-06 发布日期:2020-07-03

Advances in the study of drugs to improve growth of children with short stature

  1. *Department of Pediatrics,the First Affiliated Hospital,College of Medicine,Zhejiang University,Hangzhou  310003,China
  • Online:2020-06-06 Published:2020-07-03

摘要: 儿童的身高问题越来越被社会、家长及广大儿科医生关注,该文重点介绍改善矮小儿童成年身高的药物研究进展。生长和最终身高与遗传、宫内发育、出生后的营养、环境和内分泌激素等均有关。重组人生长激素、长效人重组生长激素对已批准的适应证改善儿童成年终身高的疗效是确定的,目前主要进行长效人重组生长激素品种的研发上市以及扩大适应证的临床试验。重组人类胰岛素生长因子-1(IGF-1)是治疗IGF-1缺乏的主要药物,使用中要注意低血糖和肿瘤的风险。促性腺激素释放激素类似物(GnRHa)、第三代非甾体类芳香化酶抑制剂等可有效延缓骨骺融合,联合长效人重组生长激素(rhGH)治疗对改善终身高有一定益处,但其疗效与病因、年龄、遗传身高、治疗疗程等因素有关,临床应综合考虑药物远期安全性、疗效以及经济等因素,谨慎评估、选择。修饰后的重组C型利钠肽已完成Ⅲ期临床试验,对改善软骨发育不全儿童的身高有一定获益。期望能继续开发出安全、有效、优质、稳定的新药用于改善儿童矮身材,促进矮小儿童的身心健康。

关键词: 矮小, 生长激素, 类胰岛素生长因子-1, 芳香化酶抑制剂, 软骨发育不全

Abstract: Children’s height has been more and more concerned about by the society,parents and pediatricians. We discussed the research progress of drugs to improve growth of pediatric patients with short stature. Growth and final height are related to heredity,intrauterine development,postnatal nutrition,environment and endocrine hormones. New data confirm the known-efficacy of recombinant human growth hormone(rhGH) in growth hormone deficiency(GHD) and idiopathic short stature,and long-acting recombinant human growth hormone(LA-rhGH) in GHD. In future,the research and development of LA-rhGH and its clinical trials to expand the indications will be mainly carried out. Recombinant human insulin growth factor-1(rhIGF-1) is the main drug in the treatment of IGF-1 deficiency,and hypoglycemia and neoplasm should be monitored. GnRHa and the third generation of nonsteroidal aromatase inhibitors(AIs)can effectively delay epiphyseal fusion,and when combined with rhGH treatment it is beneficial to improve height. However,its efficacy is related to the etiology,age,heredity,treatment course and other factors. Long-term safety,efficacy and economy of the GnRHa or AIs should be considered comprehensively to make careful evaluation and selection. The modified recombinant C-type natriuretic peptide has completed the phase Ⅲ clinical trial, which has certain benefits to improve the height of children with achondroplasia. Some safe,effective,high-quality and stable new drugs were expected to be developed in order to enhance growth and promote their physical and mental health.

Key words: short stature, growth hormone, IGF-1, aromatase inhibitors, achondroplasia