Growth hormone treatment in children: review of safety and efficacy.
Growth hormone treatment in children: review of safety and efficacy.
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DOI：
10.2165/00148581-200406020-00003
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发表时间：
2004-01-01
期刊：
影响因子：
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通讯作者：
Cutfield, Wayne S
中科院分区：
文献类型：
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作者：
Harris, Mark;Hofman, Paul L;Cutfield, Wayne S
自生长激素 (GH) 出现以来，GH 治疗的儿科应用不断扩大。患有多种生长障碍的儿童接受了 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 GH 缺乏症 (GHD)、特纳综合征、慢性肾功能衰竭、小于胎龄儿、普瑞德威利综合征、青少年慢性关节炎和囊性纤维化。生长激素疗法已被清楚地证明可以在儿童时期改善各种生长受到损害的儿科疾病的身高增长速度。现在有数据证实 GH 治疗还可以提高许多诊断亚组的最终身高。早期开始和个体化的 GH 治疗有可能使特发性 GHD 儿童的儿童生长正常化，并使他们能够以具有成本效益的方式实现其遗传目标身高。对于 GHD 不是影响生长的主要因素的儿童，超生理剂量的 GH 已被证明可以增加儿童时期的身高增长速度和最终身高。针对这些情况开发预测模型可以进一步改善身高结果，同时保持可接受的安全性。儿童恶性肿瘤的幸存者，特别是那些接受过颅脊髓照射的幸存者，是一个特别具有挑战性的群体。与患有特发性 GHD 的儿童相比，他们似乎对 GH 的反应较差，并且有提前进入青春期的倾向。这两个因素都会对其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。当患有 GHD 的儿童接受标准剂量的 GH 治疗时，有很强的安全记录。 GH 治疗期间的不良事件并不常见，而且通常与药物无关。然而，对成人生活的持续监测至关重要，特别是对于接受超生理剂量的 GH 或其潜在状况增加不良反应风险的儿童。
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.