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Factors affecting growth hormone treatment in short stature children born small for gestational age in China: a
Li Xi1, Ruoqian Cheng1, Yingkai He2
1Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Children's Hospital of Fudan University, Shanghai, China.
Insights
Recombinant human growth hormone (rhGH) treatment improves growth in Chinese children born small for gestational age (SGA). Those without 11p15 hypomethylation or with impaired GH secretion respond best to rhGH therapy.
Area of Science:
- Pediatric Endocrinology
- Genetics
- Growth Disorders
Background:
- Short stature in children born small for gestational age (SGA) is a clinical challenge.
- Recombinant human growth hormone (rhGH) is a treatment option for SGA-induced short stature.
- Identifying factors influencing rhGH treatment response is crucial for optimizing outcomes.
Purpose of the Study:
- To evaluate factors affecting rhGH treatment efficacy in Chinese children with short stature born SGA.
- To identify predictors of growth response to rhGH in this specific population.
Main Methods:
- Retrospective, single-center study of 41 Chinese children born SGA with short stature.
- Generalized estimating equation (GEE) analysis to assess influencing factors.
- Outcome measures included height standard deviation score (HTSDS), growth velocity (GV), and change in HTSDS (ΔHTSDS).
Main Results:
- rhGH treatment significantly improved HTSDS, height, GV, and ΔHTSDS over 36 months.
- Negative correlations observed between 11p15 hypomethylation and birth weight with GV and ΔHTSDS.
- Positive correlation found between impaired GH secretion and improved GV and ΔHTSDS.
Conclusions:
- Children born SGA without 11p15 hypomethylation or with impaired GH secretion exhibit a better response to rhGH.
- Pre-treatment genetic and endocrine evaluations are essential for predicting rhGH treatment success in SGA-related short stature.
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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