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Recombinant growth hormone for children and adolescents with Turner syndrome

  • Review
  • Intervention

Authors


Abstract

Background

Turner syndrome (TS) affects about one in 1500 to 2500 live-born females. One of the most prevalent and salient features of the syndrome is extremely short stature. Untreated women are approximately 20 to 21 cm shorter than normal women within their respective populations. Recombinant human growth hormone (hGH) has been used to increase growth and final height in girls who have Turner syndrome.

Objectives

To assess the effects of recombinant growth hormone in children and adolescents with TS.

Search methods

MEDLINE, EMBASE, The Cochrane Library, LILACS, BIOSIS, Science Citation Index and reference lists were used to identify relevant trials.

Selection criteria

Randomised controlled trials were included if they were carried out in children with TS before achieving final height. Growth hormone had to be administered for a minimum of six months and compared with a placebo or no treatment control condition.

Data collection and analysis

Two reviewers assessed studies for inclusion criteria and for methodological quality. The primary outcomes were final height and growth. Secondary outcomes included bone age, quality of life, cognitive performance, and adverse effects.

Main results

Four RCTs that included 365 participants after one year of treatment were included. Only one trial reported final height in 61 treated women to be 148 cm and 141 cm in 43 untreated women (mean difference (MD) seven cm, 95% CI 6 to 8). Short-term growth velocity was greater in treated than untreated girls after one year (two trials, MD three cm per year, 95% CI 2 to 4) and after two years (one trial, MD two cm per year, 95% CI 1 to 2.3). Skeletal maturity was not accelerated by treatment with recombinant growth hormone (hGH). Adverse effects were minimally reported.

Authors' conclusions

Recombinant human growth hormone (hGH) doses between 0.3 to 0.375 mg/kg/wk increase short-term growth in girls with Turner syndrome by approximately three (two) cm in the first (second) year of treatment. Treatment in one trial increased final height by approximately six cm over an untreated control group. Despite this increase, the final height of treated women was still outside the normal range. Additional trials of the effects of hGH carried out with control groups until final height is achieved would allow better informed decisions about whether the benefits of hGH treatment outweigh the requirement of treatment over several years at considerable cost.

摘要

背景

基因重組生長素在透納氏症(Turner syndrome)的小孩及青少年之應用

透納氏症約佔活產女嬰的1500到2500分之一。這個症候群其中一個最盛行及顯著的特徵是非常矮小。沒有治療的婦女比相對人口的正常婦女矮約20到21公分。基因重組生長素已經被應用在促進透納氏症女孩之生長及最終之身高。

目標

評估基因重組生長素在透納氏症的小孩及青少年之效果。

搜尋策略

從MEDLINE, EMBASE, 考科藍圖書館, LILACS, BIOSIS, Science Citation Index 及參考文獻找出相關的試驗。

選擇標準

在尚未達最終身高的透納氏症小孩所進行的隨機對照試驗被納入分析。研究組接受了至少六個月的生長素注射治療,然後與使用安慰劑或未接受治療的對照組做比較。

資料收集與分析

兩位審查者評估研究被納入之條件及方法學之品質。初級結果是最終的身高及生長情況。次級結果包括骨齡、生活品質、認知表現及副作用。

主要結論

四個隨機對照試驗被納入分析,共包括365位治療一年的受試者。只有一個試驗有報告最終身高,其中61位接受治療的受試者最終身高為148公分,而43位沒有接受治療者為141公分(平均差7公分,95%信賴區間為6到8公分)。治療組比未治療組之短期生長速率較快(經過一年治療的兩個試驗,平均差為每年3公分,95%信賴區間為2到4公分,經過兩年治療的一個試驗,平均差為每年2公分,95%信賴區間為1到2.3公分)。生長素治療沒有促進骨骼成熟度。副作用很少。

作者結論

基因重組生長素(劑量為每周每公斤體重0.3到0.375毫克)對透納氏症女孩的短期生長影響,在治療的第一年及第二年分別增加3公分和2公分。在一個試驗中,治療組比未治療組之最終身高多了6公分。雖然如此,治療組女性的最終身高仍低於正常範圍。我們需要額外的試驗,觀察以生長激素治療效果與對照組比較,才能協助告知、評估與決定以生長激素治療的好處是否多於昂貴且多年的治療時間。

翻譯人

本摘要由臺灣大學附設醫院張然舜翻譯。

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

總結

有些證據顯示:基因重組生長素可改善透納氏症女性短期生長速度和最終身高。透納氏症是一種影響女性生殖發育和外觀的遺傳疾病。透納氏症的女性比正常女性矮很多(約21公分或8英吋)。為了克服生長緩慢的問題,基因重組生長素被用來治療。使用方式為一週數次皮下注射,直到達到最終身高。文獻回顧顯示,基因重組生長素確實可增加透納氏症女性短期生長速度及成年之身高(大約5公分或2英吋)。雖然如此,經過基因重組生長素治療的透納氏症女性的最終身高仍低於正常女性。61位接受治療的女性平均身高為148公分;而未接受治療的43位女性平均身高為141公分。

Plain language summary

Recombinant growth hormone for children and adolescents with Turner syndrome

Turner syndrome (TS) is a genetic disorder affecting the sexual development and appearance of girls and women. Women with TS are much shorter than other women (by about 21 cm or eight inches). To try to overcome slow growth, recombinant growth hormone (hGH) has been given. The hormone is injected under the skin several times a week until final adult height is achieved. The review found some evidence that hGH does increase short-term growth in girls with TS and adult height (an increase of perhaps five centimeters or two inches). However, girls treated with hGH are still substantially shorter than other women as adults. Final height in 61 treated women was 148 cm and 141 cm in 43 untreated women.

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