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Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during
Di Wu1, Rui-Min Chen2, Shao-Ke Chen3
1Department 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.
Insights
Growth hormone (GH) therapy effectively improves final adult height (FAH) in children with idiopathic short stature (ISS). Longer treatment durations, especially over two years, yield better results for both boys and girls with ISS.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Skeletal Development
Background:
- Idiopathic Short Stature (ISS) affects children's growth, necessitating effective treatment strategies.
- Evaluating growth hormone (GH) efficacy is crucial for optimizing treatment outcomes in ISS patients.
Purpose of the Study:
- To assess the effectiveness of GH therapy in improving final adult height (FAH) in children with ISS.
- To identify factors influencing treatment success in a multicenter cohort.
Main Methods:
- A real-world, multicenter observational study involving children with ISS from seven Chinese hospitals.
- Evaluated height gain standard deviation score (SDS) and height gain over target height (TH).
- Utilized multivariate linear regression to analyze factors affecting FAH SDS.
Main Results:
- GH therapy significantly improved FAH SDS in both boys and girls with ISS (P < 0.00001).
- Treatment courses exceeding two years showed the highest FAH SDS gain (1.54 SD).
- Baseline height SDS and treatment duration were significant predictors for boys, while baseline height SDS and bone age were significant for girls.
Conclusions:
- GH therapy is effective in improving FAH for both male and female ISS patients, even when initiated after age 10.
- A treatment duration of over two years is recommended for girls, and extended courses for boys to maximize height gains.
- While some peri-pubertal ISS patients experience spontaneous growth, GH therapy offers a significant improvement in achieving target height.
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.
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