Pediatric Endocrine Division, Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia.
Pediatric Division, Department of Clinical Science, College of Medicine, Princess Nourah bint Abdulrahman University, Riyadh, Saudi Arabia.
J Pediatr Endocrinol Metab. 2022 Oct 7;35(11):1357-1368. doi: 10.1515/jpem-2022-0379. Print 2022 Nov 25.
We aimed to report our 10-year experience of treating short children born small for gestational age (SGA) by comparing the long-term growth, metabolic safety, and cost-effectiveness of recombinant human growth hormone (rhGH) therapy in short children born SGA with those in rhGH-treated children with growth hormone deficiency (GHD) and Turner syndrome.
We performed a 10-year retrospective cohort study at King Saud University Medical City. We included children aged 3-16 years who received rhGH for GHD, SGA, or Turner syndrome for >1 year.
A total of 166 children received rhGH therapy for GHD, 58 for SGA, and 16 for Turner syndrome. During the last study visit, the average height change was 21 cm for GHD children and 14 cm for children born SGA (p-value <0.001). The height SDS change was 0.84 for GHD children and 0.55 for SGA children (p-value=0.004). The average cost-effectiveness ratios for treating GHD and SGA children were USD 1,717.22 and USD 1,157.19 per centimeter gained, respectively. Moreover, the mean incremental cost-effectiveness ratio for GHD vs. SGA patients was USD 2,820.39 per centimeter gained. Dysglycemia developed in 70 patients: 43 (36.44%), 22 (40.74%), and 5 (13%) in the GHD, SGA, and Turner syndrome groups, respectively.
rhGH is effective in height improvement of short children. However, pursuing rhGH treatment for children born SGA requires a shared decision-making approach to balance the modest benefit of final adult height gain with the long-term metabolic effects, considering the acceptable costs on the Saudi healthcare system.
通过比较生长激素缺乏症（GHD）和特纳综合征患儿与胎儿生长受限（SGA）患儿接受重组人生长激素（rhGH）治疗的长期生长、代谢安全性和成本效益，报告我们治疗 SGA 矮小儿童的 10 年经验。
我们在沙特国王大学医疗城进行了一项为期 10 年的回顾性队列研究。纳入年龄 3-16 岁、接受 rhGH 治疗 GHD、SGA 或特纳综合征>1 年的儿童。
共有 166 名 GHD 儿童、58 名 SGA 儿童和 16 名特纳综合征儿童接受 rhGH 治疗。在最后一次研究访问时，GHD 儿童的平均身高变化为 21cm，SGA 儿童为 14cm（p 值<0.001）。GHD 儿童身高 SDS 变化为 0.84，SGA 儿童为 0.55（p 值=0.004）。治疗 GHD 和 SGA 儿童的平均成本效益比分别为每增加 1cm 身高需花费 1717.22 美元和 1157.19 美元。此外，GHD 与 SGA 患者的平均增量成本效益比为每增加 1cm 身高需花费 2820.39 美元。70 名患者出现血糖异常：GHD、SGA 和特纳综合征组分别为 43 名（36.44%）、22 名（40.74%）和 5 名（13%）。
rhGH 可有效改善矮小儿童的身高。然而，对于 SGA 矮小儿童，需要采用共同决策的方法来平衡最终成年身高增加的适度益处与长期代谢影响，同时考虑沙特医疗保健系统的可接受成本。
