Intervention Review

Recombinant growth hormone for idiopathic short stature in children and adolescents

  1. Jackie Bryant*,
  2. Louise Baxter,
  3. Carolyn B Cave,
  4. Ruairidh Milne

Editorial Group: Cochrane Metabolic and Endocrine Disorders Group

Published Online: 18 JUL 2007

Assessed as up-to-date: 30 DEC 2005

DOI: 10.1002/14651858.CD004440.pub2


How to Cite

Bryant J, Baxter L, Cave CB, Milne R. Recombinant growth hormone for idiopathic short stature in children and adolescents. Cochrane Database of Systematic Reviews 2007, Issue 3. Art. No.: CD004440. DOI: 10.1002/14651858.CD004440.pub2.

Author Information

  1. Southampton University, Wessex Institute for Health Research and Development, Southampton, Hants, UK

*Jackie Bryant, Wessex Institute for Health Research and Development, Southampton University, Mailpoint 728, Biomedical Sciences Building, Bassett Crescent East, Southampton, Hants, SO16 7PX, UK. jsb1@soton.ac.uk. j.s.bryant@soton.ac.uk.

Publication History

  1. Publication Status: Edited (no change to conclusions)
  2. Published Online: 18 JUL 2007

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Abstract

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

Background

Idiopathic short stature (ISS) refers to children who are very short compared with their peers for unknown or hereditary reasons. Recombinant human growth hormone (GH) has been used to increase growth and final height in children with ISS.

Objectives

To assess the effects of recombinant human GH on short-term growth and final height in children with ISS.

Search methods

Studies were obtained from computerised searches of MEDLINE, EMBASE, The Cochrane Library, Science Citation Index, BIOSIS and Current Controlled Trials. Article reference lists were assessed for trials and experts and pharmaceutical companies were contacted.

Selection criteria

Randomised controlled trials were included if they were carried out in children with ISS with normal GH secretion. GH had to be administered for a minimum of six months and be compared with placebo or no treatment. A growth or height outcome measure had to be assessed.

Data collection and analysis

Two reviewers assessed studies for inclusion criteria and for methodological quality. Data were extracted by one reviewer and checked by a second. The primary outcome was final height and secondary outcomes included short term growth, health related quality of life and adverse effects. To estimate summary treatment effects, data were pooled, when appropriate using a random effects model.

Main results

Ten RCTs were included. One trial reported near final height in girls and found that girls treated with GH were 7.5 cm taller than untreated controls (GH group, 155.3 cm ± 6.4; control, 147.8 cm ± 2.6; P = 0.003); another trial which reported adult height standard deviation score found that children treated with GH were 3.7 cm taller than children in a placebo-treated group (95% confidence intervals 0.03 to 1.10; P < 0.04). The other trials reported short term outcomes. Results suggest that short-term height gains can range from none to approximately 0.7 SD over one year. One study reported health related quality of life and showed no significant improvement in GH treated children compared with those in the control group, whilst another found no significant evidence that GH treatment impacts psychological adaptation or self-perception in children with ISS. No serious adverse effects of treatment were reported.

Authors' conclusions

GH therapy can increase short-term growth and improve (near) final height. Increases in height are such that treated individuals remain relatively short when compared with peers of normal stature. Large, multicentre RCTs are required which should focus on final height and address quality of life and cost issues.

 

Plain language summary

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

Recombinant growth hormone for idiopathic short stature in children and adolescents

There is some evidence that recombinant human growth hormone improves short term growth and (near) final adult height in children with idiopathic short stature.

Idiopathic short stature is the term used when children are very short compared with others of their age for unknown or hereditary reasons. They do not have a disease. Recombinant human growth hormone has been used to try to overcome growth failure in these children. It must be injected under the skin six to seven times a week until adult height is reached. Existing evidence suggests that growth hormone can increase short term growth and improve final or near final adult height.
Ten studies included altogether 741 children and lasted between six months and 6.2 years. Results showed that individuals treated with growth hormone remain relatively short when compared with peers of normal stature. Girls treated with growth hormone were 7.5 cm taller than untreated controls (growth hormone treated group 155.3 cm and control group 147.8 cm); another trial found that children treated with growth hormone were 3.7 cm taller than children in a placebo-treated group. No serious adverse effects were reported in the included studies. Although serious adverse effects (there has been concern that growth hormone would induce new tumours or increase the likelihood of tumour relapse) may be rare, their possibility must also be taken into consideration.

 

摘要

  1. Top of page
  2. Abstract
  3. Plain language summary
  4. 摘要

背景

合成生長激素對於兒童與青少年之特發性矮小的作用

特發性矮小係指孩童之身高,因不明或遺傳之因素,而與同年齡者相較明顯較矮者。合成人類生長激素已被用於增進特發性矮小孩童之生長和最終身高。

目標

評估合成人類生長激素對於患有特發性矮小的孩童之短期生長及最終身高的影響。

搜尋策略

我們以電腦搜尋MEDLINE、EMBASE、The Cochrane Library、Science Citation Index、BIOSIS and Current Controlled Trials等資料庫內的相關研究。我們亦分析了參考文獻的列表,尋找相關的試驗、專家及藥廠並與之聯繫。

選擇標準

我們選用在生長激素正常分泌之特發性矮小孩童身上所進行的隨機對照試驗(randomised controlled trials)。生長激素需使用至少6個月,且必須與使用安慰劑或未受治療的對照組比較。同時,亦須進行對生長或身高結果的分析。

資料收集與分析

有2位回顧者來分析研究的篩選條件及方法學上的品質。其中1位回顧者將數據自各研究中截取出來,再由另1位負責檢查。其主要結果為最後的身高,而次要結果則包括了短期內的生長、與健康相關的生活品質和副作用。為了估計總合治療效果,各數據被適當的用隨機效應模型(random effect model)集合起來。

主要結論

共有10個隨機對照試驗被收錄。其中1個試驗報告了女孩的近似最終身高,並發現了以生長激素治療的女孩,身高比未接受治療的對照組高了7.5公分(生長激素組,155.3公分�.4;對照組,147.8公分�.6;P = 0.003);另1個報告成人身高標準差的試驗則發現:以生長激素治療的孩童,身高比接受安慰劑治療組高了3.7公分(95%信賴區間由0.03到1.10;P < 0.04)。另1個試驗報告短期的結果,則顯示出1年內身高的增加可以從沒有到大約0.7個標準差。1個以與健康相關的生活品質為主題的研究則指出:與對照組相較,以生長激素治療的孩童並未獲得明顯改善;同時,另1個研究則發現:生長激素治療對於特發性矮小孩童的心理適應或自我認知並未有顯著的影響。報告並未指出有與治療相關的嚴重副作用。

作者結論

生長激素治療能增進短期的生長及(近似)最終身高。經過治療的患者,其身高雖然增加,但與標準身材的同輩相較仍較為矮小。目前仍須大型且多中心的隨機對照試驗,來對最終身高、生活品質及費用等項目做進一步分析。

翻譯人

本摘要由臺灣大學附設醫院林志弘翻譯。

此翻譯計畫由臺灣國家衛生研究院(National Health Research Institutes, Taiwan)統籌。

總結

有一些證據指出,在特發性矮小的孩童身上使用合成人類生長激素,可增進短期的生長及(近似)最終成人身高。特發性矮小係因指不明或遺傳之因素,使孩童之身高與同齡者相較特別矮小者。其本身並非疾病所造成。合成人類生長激素已被嘗試用於克服這些孩童的生長衰竭。他們每週必須接受六到七次的皮下注射,直至達到成人身高為止。現有證據指出,生長激素可增進短期生長及最終或近似最終之成人身高。在收錄的10個研究當中,共包含了741位孩童,而試驗期間則從6個月到6.2年。結果顯示,經生長激素治療者,與正常身材的同儕相較,其身高仍相對較矮。經生長激素治療的女孩,則較未治療的對照組高出7.5公分(生長激素治療組:155.3公分;對照組:147.8公分)。另一個試驗則指出:經生長激素治療的孩童,其身高較接受安慰劑治療的對照組高出了3.7公分。在收錄的研究當中,並未發現嚴重的副作用。雖然嚴重副作用(如生長激素會增加腫瘤的產生或復發機會)可能很罕見,但仍必須考量其可能性。