Harris Mark, Hofman Paul L, Cutfield Wayne S
Department of Paediatrics, University of Auckland, Auckland, New Zealand.
Paediatr Drugs. 2004;6(2):93-106. doi: 10.2165/00148581-200406020-00003.
Since the advent of growth hormone (GH), the pediatric applications of GH therapy have expanded. Children with a wide variety of growth disorders have received GH treatment. The therapeutic effects and safety profile of GH in a number of pediatric conditions are reviewed, including GH deficiency (GHD), Turner syndrome, chronic renal failure, children born small for gestational age, Prader-Willi syndrome, juvenile chronic arthritis, and cystic fibrosis. GH therapy has been clearly shown to improve height velocity during childhood in a variety of pediatric conditions in which growth is compromised. There is now data that confirms GH treatment also improves final height in a number of diagnostic subgroups. Early initiation and individualization of GH treatment has the potential to normalize childhood growth in children with idiopathic GHD and enable them to achieve their genetic target height in a cost-effective manner. In children in whom GHD is not the main factor compromising growth, supra-physiological doses of GH have been shown to increase height velocity during childhood and final height. The development of predictive models for these conditions may allow further improvements in height outcome while maintaining an acceptable safety profile. Survivors of childhood malignancy, particularly those who have had craniospinal irradiation, represent a particularly challenging group. They appear to be less responsive to GH than children with idiopathic GHD and have a tendency to enter puberty at an earlier age. Both of these factors have a negative impact on their final height. Strategies that combine GH treatment with suppression of puberty using a gonadotropin releasing hormone analog may result in improved height outcomes. When children with GHD are treated with standard doses of GH there is a strong safety record. Adverse events during GH therapy are uncommon and often not drug related. Continued surveillance into adult life is crucial however, particularly in children receiving supra-physiological doses of GH or whose underlying condition increases their risk of adverse effects.
自生长激素（GH）问世以来，GH治疗在儿科的应用范围不断扩大。患有各种生长障碍的儿童都接受了GH治疗。本文综述了GH在多种儿科疾病中的治疗效果和安全性，包括生长激素缺乏症（GHD）、特纳综合征、慢性肾功能衰竭、小于胎龄儿、普拉德-威利综合征、幼年慢性关节炎和囊性纤维化。在多种生长受限的儿科疾病中，GH治疗已被明确证明可提高儿童期的身高增长速度。现在有数据证实，GH治疗在一些诊断亚组中也能提高最终身高。对于特发性GHD儿童，早期开始GH治疗并实现个体化，有可能使儿童期生长正常化，并使其以具有成本效益的方式达到其遗传目标身高。对于GHD不是影响生长的主要因素的儿童，超生理剂量的GH已被证明可增加儿童期的身高增长速度和最终身高。针对这些疾病的预测模型的开发可能会在保持可接受的安全性的同时，进一步改善身高结果。儿童恶性肿瘤幸存者，尤其是接受过颅脊柱照射的幸存者，是一个特别具有挑战性的群体。他们似乎对GH的反应不如特发性GHD儿童，并且有更早进入青春期的倾向。这两个因素都会对他们的最终身高产生负面影响。将GH治疗与使用促性腺激素释放激素类似物抑制青春期相结合的策略可能会改善身高结果。当用标准剂量的GH治疗GHD儿童时，有很强的安全记录。GH治疗期间的不良事件并不常见，且往往与药物无关。然而，持续监测至成年期至关重要，特别是对于接受超生理剂量GH或其潜在疾病增加不良反应风险的儿童。
