Introduction Growth hormone (GH) therapy improves height outcomes in short children born small for gestational age (SGA); however, real-world data on long-term GH exposure are few. Methods We report results from an observational study (NCT01578135) including children born SGA, treated with GH at 126 sites in France and followed-up for >5 years until achieving final adult height (FAH) or until study termination. Primary endpoints were the proportion of patients with normal (>−2) height standard deviation score (SDS) at last visit and with normal FAH SDS. Post hoc analyses were performed by multivariate logistic regression analysis with stepwise elimination to identify factors associated with GH dose modulation and normal height SDS achievement. Results Of 1408 registered patients, a representative sample (n = 291) was selected for long-term follow-up. At last visit, 193/291 (66.3%) children achieved normal height SDS and 72/291 (24.7%) reached FAH. FAH SDS was >–2 for chronological age in 48 (66.7%) children and >–2 for adult age in 40 (55.6%) children. In the post hoc analyses, height SDS at last visit was a significant determinant of whether GH dose had been modulated. Factors significantly associated with reaching normal height SDS were baseline height SDS (taller, better), age at treatment start (younger, better), treatment duration excluding discontinuation periods (longer, better) and absence of a chronic disease. Most (70%) adverse events were non-serious, with 39% considered possibly/probably related to GH treatment. Conclusions GH therapy was fairly effective in most short children born SGA. No new safety concerns were identified.


中文翻译：

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生长激素剂量调节和小于胎龄儿的最终身高：法国真实数据

简介 生长激素 (GH) 疗法可改善出生小于胎龄 (SGA) 的矮个儿的身高结果；然而，关于长期接触 GH 的真实数据很少。方法 我们报告了一项观察性研究 (NCT01578135) 的结果，其中包括出生 SGA 的儿童，在法国 126 个地点接受 GH 治疗，并随访 > 5 年，直至达到最终成年身高 (FAH) 或直至研究终止。主要终点是上次就诊时身高标准差评分 (SDS) 正常 (>−2) 和 FAH SDS 正常的患者比例。通过逐步消除的多元逻辑回归分析进行事后分析，以确定与 GH 剂量调节和正常身高 SDS 成就相关的因素。结果 在 1408 名登记患者中，选择了具有代表性的样本（n = 291）进行长期随访。在上次访问时，193/291 (66.3%) 儿童达到正常身高 SDS，72/291 (24.7%) 儿童达到 FAH。48 名 (66.7%) 儿童的实际年龄 FAH SDS >–2，40 名 (55.6%) 儿童的成年年龄 FAH SDS >–2。在事后分析中，最后一次访视时的身高 SDS 是 GH 剂量是否已调整的重要决定因素。与达到正常身高 SDS 显着相关的因素包括基线身高 SDS（越高，越好）、治疗开始时的年龄（越年轻、越好）、治疗持续时间（不包括停药期）（更长、越好）和没有慢性疾病。大多数（70%）不良事件并不严重，其中 39% 被认为可能/很可能与 GH 治疗有关。结论 GH 治疗对于大多数矮小的出生 SGA 儿童相当有效。没有发现新的安全问题。