特发性身材矮小儿童的最终成人身高：一项关于青春期开始时单独使用生长激素治疗的多中心研究。
Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty.
机构信息
Department of Endocrine and Genetics and Metabolism, Beijing Children's Hospital, Capital Medical University, National Centre for Children's Health, No. 56 Nanlishi Road, Xicheng District, Beijing, 100045, China.
Department of Endocrinology, Fuzhou Children's Hospital of Fujian Medical University Teaching Hospital, No.145, 817 Middle Road, Gulou District, Fuzhou, Fuzhou, 350005, Fujian Province, China.
出版信息
BMC Pediatr. 2020 Mar 28;20(1):138. doi: 10.1186/s12887-020-02034-8.
BACKGROUND
To evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study.
METHODS
A real-world observation was carried out. Children with ISS in seven hospitals in China were enrolled. The height gains standard deviation score and the height gain over the target height were evaluated.
RESULTS
There were 344 ISS patients (217 boys and 127 girls). The baseline average age of boys and girls was 12.7 and 11.7 years, with bone age of 11.7 and 10.1 years, respectively. The baseline height SDS of boys and girls was - 3.07 and - 2.74, and the FAH SDS was - 1.91 and - 1.38, respectively. Compared with the baseline height SDS, the FAH SDS was significantly increased in both boys and girls (both P = 0.0000). The FAH SDS was the highest (gain by 1.54 SD) in the ≥2y treatment course group. Two hundred eighteen patients (218/344, 63.4%) had a FAH SDS > - 2 SD. Among these patients, girls in the 1-2y treatment course group and ≥ 2y group had a FAH SDS higher than TH SDS. Even in the control group, a spontaneous catch-up growth of 1.16 SD was observed. A multivariate linear regression model was used to analyze the results, with FAH SDS as the dependent variable. It was found that the treatment course and baseline height SDS in the boys' model were statistically significant (P < 0.05), whereas the baseline height SDS and baseline bone age significantly affected the girls' FAH SDS (P < 0.05).
CONCLUSIONS
Both girls and boys of ISS improved FAH by GH therapy even if treatments begin over 10 years old and majority of them reached TH. Some peri-puberty ISS will have a spontaneous height gain. We recommend the course of GH treatment more than 2 years for girls, and longer courses for boys.
背景
在一项多中心研究中，评估 GH 改善 ISS 儿童 FAH 的疗效。
方法
进行真实世界观察。纳入中国 7 家医院的 ISS 患儿。评估身高增益标准差评分和身高增益超过目标身高。
结果
ISS 患儿 344 例（男 217 例，女 127 例）。男、女童基线平均年龄分别为 12.7 岁和 11.7 岁，骨龄分别为 11.7 岁和 10.1 岁。男、女童基线身高 SDS 分别为-3.07 和-2.74，FAH SDS 分别为-1.91 和-1.38。与基线身高 SDS 相比，男、女童 FAH SDS 均显著增加（均 P=0.0000）。治疗疗程≥2 年组 FAH SDS 最高（增加 1.54 个标准差）。218 例（218/344，63.4%）患儿 FAH SDS＞-2 SDS。其中，1-2 年疗程组和≥2 年疗程组女童 FAH SDS 高于 TH SDS。即使在对照组中，也观察到了 1.16 个标准差的自发追赶生长。采用多元线性回归模型分析结果，以 FAH SDS 为因变量。发现男孩模型中治疗疗程和基线身高 SDS 有统计学意义（P＜0.05），而基线身高 SDS 和基线骨龄显著影响女孩的 FAH SDS（P＜0.05）。
结论
即使治疗开始年龄超过 10 岁且大多数患儿达到 TH，ISS 男童和女童的 FAH 均通过 GH 治疗得到改善。一些青春期前的 ISS 患儿会有自发的身高增长。我们建议女童 GH 治疗疗程超过 2 年，而男孩则需要更长的疗程。
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