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DOI：10.12201/bmr.202511.00012

The Efficacy and Safety of Different Growth Hormone Treatment Regimens in Children with Idiopathic Short Stature

• 摘要：【摘要】目的 探讨不同生长激素治疗方案在特发性矮身材（idiopathic short stature，ISS）患儿中的疗效、安全性及依从性。方法 回顾性纳入于海宁市人民医院就诊的67例ISS患儿，根据治疗方案分为长效组（16例）、长转短组（14例）和短效组（37例）。长效组全程使用聚乙二醇重组人生长激素（polyethylene glycol recombinant human growth hormone，PEG-rhGH），长换短组初期使用PEG-rhGH后改用短效重组人生长激素（recombinant human growth hormone，rhGH），短效组全程使用rhGH。比较各组治疗前及治疗3、6、9个月后的生长指标及安全性指标。结果 三组在各时间点的身高标准差积分（height standard deviation score，Ht SDS）、Ht SDS增量（ΔHt SDS）、糖代谢、肝肾功能、甲状腺功能指标均无统计学差异（P均＞0.05）。长转短组治疗3、6、9个月时的胰岛素样生长因子-1（insulin-like growth factor-1，IGF-1）水平均显著高于其他两组（P均＜0.05）。长效组、长转短组、短效组患儿的漏针率分别为0%、7.1%、34.3%，短效组漏针次数显著高于长效组（Z＝4.47，P＜0.001）。各组均未见明显不良反应。结论 三种生长激素治疗方案在ISS患儿中的疗效和安全性相当。长转短组依从性较好，漏针率较短效组降低约27%，但剂型转换后需密切监测IGF-1水平。

  关键词： 聚乙二醇重组人生长激素; 特发性矮身材; 疗效; 安全性；依从性; 

   

  Abstract: Abstract Objective To evaluate the efficacy, safety, and adherence of different growth hormone treatment regimens in children with idiopathic short stature (ISS). Method A total of 67 children diagnosed with ISS at Haining People’s Hospital were retrospectively analyzed and categorized into three groups according to treatment regimen: the long-acting group (n = 16), the switch group (n = 14), and the short-acting group (n = 37). The long-acting group received polyethylene glycol recombinant human growth hormone (PEG-rhGH) throughout treatment, the switch group initially received PEG-rhGH and was later switched to recombinant human growth hormone (rhGH), and the short-acting group received rhGH for the entire treatment period. Growth and safety indicators were compared before treatment and at 3, 6, and 9 months of therapy. Result No significant differences were observed among the three groups in height standard deviation score (Ht SDS), change in Ht SDS (ΔHt SDS), glucose metabolism, liver and kidney function, or thyroid function at any time point (all P > 0.05). Serum insulin-like growth factor-1 (IGF-1) levels in the switch group were significantly higher than those in the other two groups at 3, 6, and 9 months (all P < 0.05). The missed-injection rates were 0%, 7.1%, and 34.3% in the long-acting, switch, and short-acting group, respectively, with the short-acting group showing a significantly higher rate than the long-acting group (Z＝4.47, P＜0.001). No serious adverse events were observed in any group. Conclusion The three growth hormone regimens demonstrated comparable efficacy and safety in children with ISS. The switch group showed better treatment adherence, reducing the missed-injection rate by approximately 27% compared with the short-acting group. However, serum IGF-1 levels should be closely monitored after switching formulations.

  Key words: polyethylene glycol recombinant human growth hormone; idiopathic short stature; efficacy; safety; adherence

  提交时间：2025-11-08

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• 序号	提交日期	编号	操作
  1	2025-10-05	10.12201/bmr.202511.00012V1	下载

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