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身材矮小儿童生长激素使用指南。劳森·威尔金斯儿科内分泌学会药物与治疗委员会报告。

Guidelines for the use of growth hormone in children with short stature. A report by the Drug and Therapeutics Committee of the Lawson Wilkins Pediatric Endocrine Society.

出版信息

J Pediatr. 1995 Dec;127(6):857-67. doi: 10.1016/s0022-3476(95)70019-6.

Abstract

On the basis of the information currently available, the only conditions in which GH therapy appears to be safe and effective in increasing adult height are GH deficiency and, likely, Turner syndrome. Therapy with GH also increases the growth velocity of children with CRI and may increase adult height, but no long-term data are available. Encouraging short-term results have been reported in patients with a few other conditions, such as patients with glucocorticoid-induced growth failure, renal transplantation, and Prader-Willi syndrome, but the data are limited and no long-term studies have been reported; in many other conditions the data are either inconclusive or discouraging. For children in these latter groups, GH therapy should be considered investigational and undertaken only as part of ethically sound, controlled clinical trials. Knowledge concerning the conditions in which GH is safe and effective is a prerequisite to making rational decisions concerning its use. However, in deciding whether therapy is warranted in an individual child, one must consider other important factors. The age and emotional maturity of the child, the family structure and dynamics, and even financial considerations may, in some cases, outweigh the presence of a GH-responsive condition. Likewise, the child's and the family's views about "short" stature and the likely benefits of therapy must be considered. Ultimately, a decision concerning the appropriateness of GH therapy must be individualized and based on a realistic assessment of its impact on the quality of life of the child and future adult.

摘要

根据目前可得的信息,生长激素(GH)治疗在增加成人身高方面似乎安全有效的唯一情况是生长激素缺乏症,可能还有特纳综合征。生长激素治疗也能提高慢性肾功能不全(CRI)儿童的生长速度,并且可能增加成人身高,但尚无长期数据。对于其他一些病症的患者,如糖皮质激素诱导生长衰竭、肾移植和普拉德-威利综合征患者,已有令人鼓舞的短期结果报道,但数据有限且尚无长期研究报道;在许多其他病症中,数据要么不确定,要么令人沮丧。对于后一组中的儿童,生长激素治疗应被视为试验性的,并且仅应作为符合伦理道德、有对照的临床试验的一部分进行。了解生长激素安全有效的病症是就其使用做出合理决策的前提。然而,在决定某个儿童是否需要治疗时,必须考虑其他重要因素。在某些情况下,儿童的年龄和情感成熟度、家庭结构和动态,甚至经济因素可能比存在生长激素反应性病症更为重要。同样,必须考虑儿童及其家庭对“身材矮小”以及治疗可能带来的益处的看法。最终,关于生长激素治疗是否合适的决定必须因人而异,并基于对其对儿童和未来成人生活质量影响的现实评估。

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