Gibney James, Johannsson Gudmundur
Department of Endocrinology, St Vincent's University Hospital, Dublin 4, Ireland.
Expert Opin Drug Saf. 2004 Jul;3(4):305-16. doi: 10.1517/14740338.3.4.305.
Growth hormone (GH) is a powerful metabolic hormone that regulates fuel homeostasis through its protein anabolic and lipolytic actions. The introduction of recombinant human GH has expanded the narrow indication of treating children with severe GH deficiency (GHD) to include a broader target population of children with growth retardation and short stature and adults with hypopituitarism and severe GHD. Furthermore, because children continue to receive GH replacement therapy into adult life, the duration of treatment exposure has increased and the safety of long-term GH treatment has become increasingly important. This is of particular concern given that GH-deficient children and adults may be more vulnerable to the mitogenic stimuli of GH and insulin-like growth factor-1, both because of the underlying cause of GHD and also because of previous treatment such as radiotherapy and chemotherapy. This review focuses on the safety of treating adults with severe GHD, with specific emphasis on dose regimens, carbohydrate metabolism, neoplasia, and morbidity and mortality. Available experience from long-term replacement therapy, studies using supraphysiological doses of GH in adults and lessons learned from patients with acromegaly who have high endogenous GH levels over many years, is considered.
生长激素(GH)是一种强大的代谢激素,通过其蛋白质合成代谢和脂肪分解作用来调节能量平衡。重组人生长激素的问世,将治疗严重生长激素缺乏症(GHD)儿童这一狭窄适应证,扩大到包括生长发育迟缓、身材矮小的更广泛儿童群体,以及患有垂体功能减退和严重GHD的成人。此外,由于儿童会持续接受生长激素替代治疗直至成年,治疗暴露时间增加,长期生长激素治疗的安全性变得越来越重要。鉴于生长激素缺乏的儿童和成人,可能因生长激素缺乏的潜在病因,以及既往放疗和化疗等治疗,而对生长激素和胰岛素样生长因子-1的促有丝分裂刺激更为敏感,这一点尤其令人担忧。本综述聚焦于治疗严重生长激素缺乏症成人患者的安全性,特别强调剂量方案、碳水化合物代谢、肿瘤形成以及发病率和死亡率。文中考虑了长期替代治疗的现有经验、在成人中使用超生理剂量生长激素的研究,以及从多年来内源性生长激素水平较高的肢端肥大症患者身上吸取的教训。