Pediatric Endocrinology Unit, Istanbul Faculty of Medicine, Istanbul University, Istanbul, Turkey.
Horm Res Paediatr. 2011;76 Suppl 1:42-6. doi: 10.1159/000329161. Epub 2011 Jul 21.
Growth hormone (GH) therapy successfully increases height prognosis in children with GH deficiency (GHD); however, adult height data are still limited.
This study investigated near-adult height (NAH) in patients with idiopathic GHD (i.e. those with a GH peak <10 μg/l with no organic pathology) divided into two groups: isolated GHD and multiple pituitary hormone deficiency (MPHD).
All patients were registered in the Pfizer International Growth Study Database (KIGS). Median (10th to 90th percentile) values are given and measurements were expressed as standard deviation scores (SDS). Parental-adjusted height was determined.
GH therapy was started at a median age of 9.2 (range 4.9-12) years in patients with isolated GHD (n = 1,619, 60% males) and at 7.7 (range 2.8-12.2) years in those with MPHD (n = 554, 65% males; p < 0.001) at a median dose of 0.20 mg/kg/week. Height SDS at onset of therapy was -3.1 (range -4.5 to -2.1) and -3.8 (range -5.7 to -2.3), respectively (p < 0.001). The maximum GH peak and insulin-like growth factor I SDS were significantly (p < 0.05) lower in patients with MPHD than in those with isolated GHD. Both groups showed a significant (p < 0.05) increase in height SDS at 1 year that continued until the onset of puberty. Parental-adjusted height at the start of puberty was -0.1 (range -1.6 to 1.1) in patients with isolated GHD and -0.4 (range -1.9 to 1.2) in those with MPHD. Parental-adjusted NAH SDS in patients with isolated GHD was 0.0 (range -1.5 to 1.2) and slightly, but significantly, higher than NAH (-0.3, range -2.1 to 1.2; p < 0.001) in patients with MPHD. In patients with isolated GHD, total change in height SDS while receiving GH therapy was 1.6 (range 0.5-3.2), and the change in height SDS at puberty was 0.1 (range -0.7 to 1). The respective values were 2.6 (range 0.9-4.6) and 0.2 (range -1 to 1.3) in patients with MPHD. Parental-adjusted NAH was slightly lower in girls than in boys with isolated GHD, but no gender difference was observed in patients with MPHD. Multivariate analysis in patients with GHD and MPHD showed that higher birth weight, taller parents, greater height at onset, first-year responsiveness, and predicted height velocity were the most important predictors of NAH.
89% of patients with isolated GHD and 81% of those with MPHD reached an NAH within their genetic potential while receiving GH therapy. Most of the height gain occurred during prepubertal years.
生长激素（GH）治疗可成功提高生长激素缺乏症（GHD）患儿的身高预测值；然而，目前成人身高数据仍有限。
本研究调查了特发性 GHD（即 GH 峰值 <10μg/l 且无器质性病变）患者的近成人身高（NAH），并将患者分为孤立性 GHD 组和多种垂体激素缺乏症（MPHD）组。
所有患者均登记在辉瑞国际生长研究数据库（KIGS）中。给出中位数（10 至 90 百分位数）值，并用标准差评分（SDS）表示测量值。采用父母身高调整后的身高。
孤立性 GHD 患者（n=1619，男性占 60%）开始 GH 治疗的中位年龄为 9.2 岁（范围 4.9-12 岁），MPHD 患者（n=554，男性占 65%）为 7.7 岁（范围 2.8-12.2 岁），中位剂量为 0.20mg/kg/周。治疗开始时的身高 SDS 分别为-3.1（范围-4.5 至-2.1）和-3.8（范围-5.7 至-2.3）（p<0.001）。MPHD 患者的最大 GH 峰值和胰岛素样生长因子 I SDS 明显低于孤立性 GHD 患者（p<0.05）。两组患者在接受治疗 1 年后身高 SDS 均显著增加（p<0.05），这种增加一直持续到青春期开始。青春期开始时，孤立性 GHD 患者的父母身高调整后的 NAH SDS 为-0.1（范围-1.6 至 1.1），MPHD 患者为-0.4（范围-1.9 至 1.2）。孤立性 GHD 患者的父母身高调整后的 NAH SDS 为 0.0（范围-1.5 至 1.2），略高于 MPHD 患者的 NAH（-0.3，范围-2.1 至 1.2；p<0.001）。孤立性 GHD 患者接受 GH 治疗期间的身高 SDS 总变化为 1.6（范围 0.5-3.2），青春期身高 SDS 变化为 0.1（范围-0.7 至 1）。MPHD 患者的相应值分别为 2.6（范围 0.9-4.6）和 0.2（范围-1 至 1.3）。孤立性 GHD 女孩的父母身高调整后的 NAH 略低于男孩，但 MPHD 患者中未观察到性别差异。GHD 和 MPHD 患者的多变量分析显示，出生体重较高、父母较高、发病时身高较高、第一年反应性和预测的生长速度是 NAH 的最重要预测因素。
89%的孤立性 GHD 患者和 81%的 MPHD 患者在接受 GH 治疗时达到了遗传潜力范围内的 NAH。大多数身高增长发生在青春期前。
