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CC BY-NC-ND 4.0 · Horm Metab Res 2023; 55(09): 599-609
DOI: 10.1055/a-2136-8654
Original Article: Endocrine Care

Long-Term Efficacy and Safety of Recombinant Human Growth Hormone in Children Born Small for Gestational Age

Wei Wu
1   Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
,
Chunxiu Gong
2   Endocrine and Genetics and Metabolism, Beijing Children’s Hospital, Beijing, China
,
Yuchuan Li
2   Endocrine and Genetics and Metabolism, Beijing Children’s Hospital, Beijing, China
,
Yuhua Hu
3   Pediatrics, Jiangsu Provincial People’s Hospital, Jiangsu, China
,
Haihong Gong
3   Pediatrics, Jiangsu Provincial People’s Hospital, Jiangsu, China
,
Junfen Fu
4   Endocrinology, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China
,
Ke Huang
4   Endocrinology, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China
,
Pin Li
5   Medical Genetics and Endocrinology, Children’s Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai Children’s Hospital, Shanghai, China
,
Xiaoping Luo
1   Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
› Author Affiliations Funding Information GeneScience Pharmaceuticals
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Abstract

There is a lack of long-term data on the benefit of growth hormone (GH) treatment in Chinese children born small for gestational age (SGA). This study was conducted to assess the long-term efficacy and safety of GH treatment in children born SGA. One hundred and twenty prepubertal SGA children who did not achieve catch-up growth with height remained less than –2 standard deviations (SD) below gender-specific height were enrolled in this two-year, randomized, dose-comparative study followed by an extension study of up to 10 years. Daily subcutaneous injections of 0.23 mg/kg/week [low-dose (LD) group] or 0.46 mg/kg/week [high-dose (HD) group] somatropin were given for 104 weeks. Dosing in the extension study was≤0.46 mg/kg/week. The main outcome measures were change in height SD score (ΔHT-SDS), height velocity, insulin-like growth factor (IGF)-1, and IGF-1/IGF binding protein-3 (IGFBP-3) molar ratio. ΔHT-SDS at week 104 was 0.91±0.53 and 1.52±0.64 in the LD and HD groups (intergroup p<0.0001), respectively, and continued in an upward trend throughout the extension study, remaining above+2 for those who received treatment for a total of 7 years or more. At week 104, significant improvements were observed in height velocity, IGF-1 SDS, and IGF-1/IGFBP-3 molar ratio. Adult HT-SDS was –0.81±1.68 for boys and –0.82±1.05 for girls (p=0.9837). Glucose metabolism and thyroid function were within the normal reference range throughout treatment. Long-term recombinant human GH treatment was tolerable and effective at improving height in children born SGA.

Keywords

growth disorder - HT-SDS - height velocity - gestational age

Supplementary Material



Publication History

Received: 17 January 2023

Accepted after revision: 13 July 2023

Article published online:
07 September 2023

© 2023. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial-License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/).

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