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特发性身材矮小儿童诊断与治疗的共识声明:生长激素研究学会、劳森·威尔金斯儿科内分泌学会及欧洲儿科内分泌学会研讨会纪要

Consensus statement on the diagnosis and treatment of children with idiopathic short stature: a summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop.

作者信息

Cohen P, Rogol A D, Deal C L, Saenger P, Reiter E O, Ross J L, Chernausek S D, Savage M O, Wit J M

机构信息

Department of Endocrinology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine at UCLA, 10833 Le Conte Avenue MDCC 22-315, Los Angeles, California 90095-1752, USA.

出版信息

J Clin Endocrinol Metab. 2008 Nov;93(11):4210-7. doi: 10.1210/jc.2008-0509. Epub 2008 Sep 9.

DOI:10.1210/jc.2008-0509
PMID:18782877
Abstract

OBJECTIVE

Our objective was to summarize important advances in the management of children with idiopathic short stature (ISS).

PARTICIPANTS

Participants were 32 invited leaders in the field.

EVIDENCE

Evidence was obtained by extensive literature review and from clinical experience.

CONSENSUS

Participants reviewed discussion summaries, voted, and reached a majority decision on each document section.

CONCLUSIONS

ISS is defined auxologically by a height below -2 sd score (SDS) without findings of disease as evident by a complete evaluation by a pediatric endocrinologist including stimulated GH levels. Magnetic resonance imaging is not necessary in patients with ISS. ISS may be a risk factor for psychosocial problems, but true psychopathology is rare. In the United States and seven other countries, the regulatory authorities approved GH treatment (at doses up to 53 microg/kg.d) for children shorter than -2.25 SDS, whereas in other countries, lower cutoffs are proposed. Aromatase inhibition increases predicted adult height in males with ISS, but adult-height data are not available. Psychological counseling is worthwhile to consider instead of or as an adjunct to hormone treatment. The predicted height may be inaccurate and is not an absolute criterion for GH treatment decisions. The shorter the child, the more consideration should be given to GH. Successful first-year response to GH treatment includes an increase in height SDS of more than 0.3-0.5. The mean increase in adult height in children with ISS attributable to GH therapy (average duration of 4-7 yr) is 3.5-7.5 cm. Responses are highly variable. IGF-I levels may be helpful in assessing compliance and GH sensitivity; levels that are consistently elevated (>2.5 SDS) should prompt consideration of GH dose reduction. GH therapy for children with ISS has a similar safety profile to other GH indications.

摘要

目的

我们的目的是总结特发性身材矮小(ISS)儿童管理方面的重要进展。

参与者

参与者为该领域32位受邀的领军人物。

证据

通过广泛的文献综述和临床经验获取证据。

共识

参与者审查讨论总结、进行投票,并就每份文件的各个部分达成多数决定。

结论

通过体格检查,ISS被定义为身高低于 -2标准差评分(SDS),且经儿科内分泌学家进行全面评估(包括刺激后的生长激素水平)未发现疾病迹象。对于ISS患者,无需进行磁共振成像检查。ISS可能是心理社会问题的一个风险因素,但真正的精神病理学情况罕见。在美国和其他七个国家,监管机构批准对身高低于 -2.25 SDS的儿童使用生长激素治疗(剂量高达53微克/千克·天),而在其他国家,则提出了更低的临界值。芳香化酶抑制可增加ISS男性的预测成年身高,但尚无成年身高数据。值得考虑将心理咨询作为激素治疗的替代方法或辅助手段。预测身高可能不准确,并非生长激素治疗决策的绝对标准。孩子越矮,就越应考虑使用生长激素。生长激素治疗第一年的成功反应包括身高SDS增加超过0.3 - 0.5。生长激素治疗(平均疗程4 - 7年)使ISS儿童的成年身高平均增加3.5 - 7.5厘米。反应差异很大。胰岛素样生长因子-I(IGF-I)水平可能有助于评估依从性和生长激素敏感性;持续升高(>2.5 SDS)的水平应促使考虑降低生长激素剂量。ISS儿童的生长激素治疗安全性与其他生长激素适应证相似。

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