Pitukcheewanont Pisit, Desrosiers Paul, Steelman Joel, Rapaport Robert, Fuqua John S, Kreher Nerissa C, Hamlett Anthony
Department of Endocrinology, The Keck School of Medicine at USC and Childrens Hospital, Los Angeles, California 90026, USA.
Pediatr Endocrinol Rev. 2008 Feb;5 Suppl 2:702-7.
The GHMonitor observational registry collates data on pediatric subjects receiving Saizen (recombinant human growth hormone (GH)) therapy. From January 2003 through August 2006, 1335 subjects were enrolled in the registry, approximately two-thirds of whom are male. The most common diagnosis in the registry is idiopathic growth hormone deficiency (58%). Most subjects in GHMonitor are receiving Saizen doses using a needle-free delivery device, the cool.click (73%). Mean height standard deviation scores show an improvement from -2.1 at screening to -1.1 following 3 years of Saizen therapy. To date, adverse events have been reported in 4% of subjects. Three serious adverse events were identified to be related to Saizen by the primary investigator: cellulitis at the injection site, behavioral problems/suicidal ideation, and enlargement of a craniopharyngioma. This article provides an update on data from the registry and briefly discusses topical and controversial issues in the treatment of pediatric patients requiring GH therapy.
GHMonitor观察性注册研究收集了接受赛增(重组人生长激素(GH))治疗的儿科患者的数据。从2003年1月至2006年8月,共有1335名患者纳入该注册研究,其中约三分之二为男性。该注册研究中最常见的诊断是特发性生长激素缺乏症(58%)。GHMonitor中的大多数患者使用无针给药装置cool.click接受赛增治疗(73%)。平均身高标准差评分显示,在接受3年赛增治疗后,从筛查时的-2.1改善至-1.1。迄今为止,4%的患者报告了不良事件。主要研究者确定了3起严重不良事件与赛增有关:注射部位蜂窝织炎、行为问题/自杀意念以及颅咽管瘤增大。本文提供了该注册研究数据的更新情况,并简要讨论了需要GH治疗的儿科患者治疗中的热点和争议问题。