Factors affecting growth hormone treatment in short stature children born small for gestational age in China: a single-centre, real-world study.

IF 3 3区医学 Q2 ENDOCRINOLOGY & METABOLISM Endocrine Pub Date : 2024-12-01 Epub Date: 2024-08-29 DOI:10.1007/s12020-024-04009-6

Li Xi, Ruoqian Cheng, Yingkai He, Xiaojing Li, Jinwen Ni, Jing Wu, Zhenran Xu, Feihong Luo

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Abstract

Purpose: The study aimed to evaluate the factors influencing recombinant human growth hormone (rhGH) treatment in Chinese children with short stature born small for gestational age (SGA).

Methods: A single-centre, real-world retrospective study was conducted in short stature children born SGA in China. Outcomes were observed at 6, 12, 18, 24, 30, and 36 months. Outcome measures included height standard deviation score (HTSDS), height, growth velocity (GV), and change of HTSDS (ΔHTSDS). The study used the generalized estimating equation (GEE) to identify potential influencing factors, such as rhGH treatment duration, age at rhGH initiation, sex, 11p15 hypomethylation, GH secretion, and birth weight. A subgroup analysis was conducted to investigate the impact of 11p15 hypomethylation related to SGA or impaired GH secretion.

Results: Of all 101 SGA patients included in the screening, 41 were eligible for inclusion in the study. The mean age at rhGH initiation was 5.6 ± 2.4 years. The results of the GEE analysis showed a significant association between time after rhGH initiation and HTSDS, height, GV, and ΔHTSDS. GV increased after treatment, with the highest increase observed in the first six months. Additionally, the study found negative correlations between 11p15 hypomethylation and GV, as well as between birth weight and both GV and ΔHTSDS. The study found a positive correlation between impairment in GH secretion and both GV and ΔHTSDS. No statistically significant difference was observed in the comparison of GV or ΔHTSDS between the initiation age of GH treatment and 11p15 hypomethylation. After 24 and 30 months of rhGH treatment, patients with impaired GH secretion had significantly higher ΔHTSDS scores.

Conclusions: In short stature Chinese children born SGA, those without SGA-related 11p15 hypomethylation or with impaired GH secretion showed better response to rhGH treatment. These findings highlight the importance of pre-treatment evaluation, including genetic and endocrine assessments.

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影响中国胎龄不足矮身材儿童生长激素治疗的因素：一项单中心真实世界研究。

目的：本研究旨在评估影响重组人生长激素（rhGH）治疗中国小于胎龄（SGA）身材矮小儿童的因素：方法：对中国SGA出生的矮身材儿童进行了一项单中心、真实世界的回顾性研究。结果分别在 6、12、18、24、30 和 36 个月时进行观察。结果测量指标包括身高标准差评分（HTSDS）、身高、生长速度（GV）和身高标准差评分变化（ΔHTSDS）。研究采用广义估计方程（GEE）确定了潜在的影响因素，如rhGH治疗持续时间、开始使用rhGH的年龄、性别、11p15低甲基化、GH分泌和出生体重。研究人员还进行了一项亚组分析，以探讨与SGA或GH分泌受损有关的11p15低甲基化的影响：在筛查出的101名SGA患者中，有41名符合研究条件。开始接受rhGH治疗的平均年龄为（5.6 ± 2.4）岁。GEE分析结果显示，开始使用rhGH后的时间与HTSDS、身高、GV和ΔHTSDS之间存在显著关联。GV在治疗后有所增加，前六个月的增幅最大。此外，研究还发现 11p15 低甲基化与 GV 之间以及出生体重与 GV 和 ΔHTSDS 之间存在负相关。研究发现，GH 分泌障碍与 GV 和 ΔHTSDS 之间存在正相关。在GH治疗开始年龄与11p15低甲基化之间的GV或ΔHTSDS比较中，未观察到有统计学意义的差异。经过24个月和30个月的rhGH治疗后，GH分泌受损的患者的ΔHTSDS评分明显更高：在SGA出生的身材矮小的中国儿童中，没有SGA相关的11p15低甲基化或GH分泌受损的儿童对rhGH治疗的反应更好。这些发现强调了治疗前评估（包括遗传和内分泌评估）的重要性。

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来源期刊

Endocrine ENDOCRINOLOGY & METABOLISM-

CiteScore

6.50

自引率

5.40%

发文量

295

审稿时长

1.5 months

期刊介绍： Well-established as a major journal in today’s rapidly advancing experimental and clinical research areas, Endocrine publishes original articles devoted to basic (including molecular, cellular and physiological studies), translational and clinical research in all the different fields of endocrinology and metabolism. Articles will be accepted based on peer-reviews, priority, and editorial decision. Invited reviews, mini-reviews and viewpoints on relevant pathophysiological and clinical topics, as well as Editorials on articles appearing in the Journal, are published. Unsolicited Editorials will be evaluated by the editorial team. Outcomes of scientific meetings, as well as guidelines and position statements, may be submitted. The Journal also considers special feature articles in the field of endocrine genetics and epigenetics, as well as articles devoted to novel methods and techniques in endocrinology. Endocrine covers controversial, clinical endocrine issues. Meta-analyses on endocrine and metabolic topics are also accepted. Descriptions of single clinical cases and/or small patients studies are not published unless of exceptional interest. However, reports of novel imaging studies and endocrine side effects in single patients may be considered. Research letters and letters to the editor related or unrelated to recently published articles can be submitted. Endocrine covers leading topics in endocrinology such as neuroendocrinology, pituitary and hypothalamic peptides, thyroid physiological and clinical aspects, bone and mineral metabolism and osteoporosis, obesity, lipid and energy metabolism and food intake control, insulin, Type 1 and Type 2 diabetes, hormones of male and female reproduction, adrenal diseases pediatric and geriatric endocrinology, endocrine hypertension and endocrine oncology.