Current Indications for Growth Hormone Therapy for Children and Adolescents
Current Indications for Growth Hormone Therapy for Children and Adolescents
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DOI：
10.1159/000316130
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发表时间：
2010-01-01
期刊：
影响因子：
--
通讯作者：
Rogol, Alan D.
中科院分区：
文献类型：
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作者：
Richmond, Erick;Rogol, Alan D.
自 20 世纪 60 年代以来，生长激素 (GH) 疗法一直适用于 GH 严重缺乏的儿童和青少年。由于人垂体衍生激素的供应量很少，因此不能认真考虑用于身材矮小的其他病症。这种状态在 20 世纪 80 年代中期发生了显着变化，因为与人类垂体组织来源的 hGH 相关的克雅氏病以及（几乎）无限供应的重组 22 kDa (r)hGH 的开发。后者允许所有 GH 缺乏的儿童获得治疗，并且可以设计使用 rhGH 来增加因 GH 缺乏以外的原因造成身材矮小的婴儿、儿童和青少年的成年身高的试验，以及在成人中进行的试验GH 缺乏的男性和女性批准的适应症（美国食品和药物管理局）包括。 GH 缺乏、慢性肾病、特纳综合征、小于胎龄且未能赶上正常身高百分位数、普瑞德-威利综合征、特发性身材矮小、SHOX 基因单倍体不足和努南综合征（截至 2008 年 10 月）。儿童最常见的功效结果是身高增长速度的增加，尽管rhGH可以预防一些先天性垂体功能低下婴儿的低血糖，并增加大多数儿童的瘦/脂比，特别是那些患有严重GH缺乏症或普瑞德威利综合征的儿童。影响身体成分和健康相关生活质量的成人剂量，每公斤去脂体重远低于儿童。安全性相当有利，颅内压升高的发生率虽小，但显着，脊柱侧凸、肌肉和关节不适，包括股骨头骨骺滑脱。批准rhGH治疗用于身材矮小、非生长激素缺乏的儿童，验证了生长激素敏感性的概念，这使一些身材显着矮小但刺激生长激素测试结果正常的儿童有机会从rhGH治疗中受益，并可能达到正常范围内的成年身高，并适合其父母中期目标身高（遗传潜力）。版权所有 (C) 2010 S Karger AG，巴塞尔
Growth hormone (GH) therapy has been appropriate for severely GH-deficient children and adolescents since the 1960s. Use for other conditions for which short stature was a component could not be seriously considered because of the small supply of human pituitary-derived hormone. That state changed remarkably in the mid-1980s because of Creutzfeldt-Jakob disease associated with human pituitary tissue-derived hGH and the development of a (nearly) unlimited supply of recombinant, 22 kDa (r)hGH The latter permitted all GH-deficient children to have access to treatment and one could design trials using rhGH to increase adult height in infants, children and adolescents with causes of short stature other than GH deficiency, as well as trials in adult GH-deficient men and women Approved indications (US Food and Drug Administration) include. GH deficiency, chronic kidney disease, Turner syndrome, small-for-gestational age with failure to catch up to the normal height percentiles, Prader-Willi syndrome, idiopathic short stature, SHOX gene haploinsufficiency and Noonan syndrome (current to October 2008). The most common efficacy outcome in children is an increase in height velocity, although rhGH may prevent hypoglycemia in some infants with congenital hypopituitarism and increase the lean/fat ratio in most children especially those with severe GH deficiency or Prader-Willi syndrome Doses for adults, which affect body composition and health-related quality of life, are much lower than those for children, per kilogram of lean body mass The safety profile is quite favorable with a small, but significant, incidence of raised intracranial pressure, scoliosis, muscle and Joint discomfort, including slipped capital femoral epiphysis. The approval of rhGH therapy for short, non-GH-deficient children has validated the notion of GH sensitivity, which gives the opportunity to some children with significant short stature, but with normal stimulated GH test results, to benefit from rhGH therapy and perhaps attain an adult height within the normal range and appropriate for their mid-parental target height (genetic potential). Copyright (C) 2010 S Karger AG, Basel