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儿童生长激素治疗的适应证。

Indications for growth hormone therapy in children.

机构信息

Department of Endocrinology, Birmingham Children's Hospital, Steelhouse Lane, Birmingham B4 6NH, UK.

出版信息

Arch Dis Child. 2012 Jan;97(1):63-8. doi: 10.1136/adc.2010.186205. Epub 2011 May 3.

DOI:10.1136/adc.2010.186205
PMID:21540481
Abstract

Growth hormone (GH) therapy has now been available for over 5 decades, with all GH now biosynthetically produced, and administered by daily injection. Paediatric GH is currently licensed in six different conditions: growth hormone deficiency (GHD), Turner syndrome (TS), small for gestational age (SGA), Prader-Willi-syndrome (PWS), chronic renal insufficiency (CRI), and short stature due to SHOX deficiency; all of these have been ratified by the most recent (2010) NICE review. Whilst the primary purpose of paediatric GH therapy in most indications is to improve short and long-term growth, in others (eg. PWS) it has a role in improvement of body composition. Recent UK national audits indicate approximately 4700 children receiving GH therapy, with approximately 760 new starts a year, with most prescription still via historical growth centres. There are currently 7 different manufacturers of GH, and while most UK units currently offer free patient choice for GH device, with preliminary evidence indicating that this may improve adherence with therapy, the 30% price difference may limit choice in the future.

摘要

生长激素(GH)治疗现已应用超过 50 年,目前所有的 GH 均为生物合成,并通过每日注射给药。儿科 GH 目前在六种不同的情况下获得许可:生长激素缺乏症(GHD)、特纳综合征(TS)、小于胎龄儿(SGA)、普拉德-威利综合征(PWS)、慢性肾功能不全(CRI)和由于 SHOX 缺乏导致的身材矮小;所有这些均已被最近(2010 年)NICE 审查认可。虽然儿科 GH 治疗在大多数适应证中的主要目的是改善短期和长期生长,但在其他情况下(例如 PWS),它在改善身体成分方面也有作用。最近的英国全国性审计显示,约有 4700 名儿童接受 GH 治疗,每年约有 760 名新患者开始治疗,大多数处方仍通过历史上的生长中心开出。目前有 7 家不同的 GH 制造商,虽然大多数英国单位目前提供免费的 GH 设备供患者选择,但初步证据表明这可能会提高治疗的依从性,但 30%的价格差异可能会限制未来的选择。

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