Evaluation of the efficacy， safety and cost-effectiveness of different formulations of short-acting rhGH in the treatment of patients with short stature

ZHENG Zhuoting; LIU Yilong; QIN Xiaomao; ZENG Zhen; YAN Run; LONG Enwu

doi:10.6039/j.issn.1001-0408.2025.09.16

您当前的位置：

首页 >

文章列表页 >

Evaluation of the efficacy， safety and cost-effectiveness of different formulations of short-acting rhGH in the treatment of patients with short stature

更新时间：2025-05-09

- Evaluation of the efficacy， safety and cost-effectiveness of different formulations of short-acting rhGH in the treatment of patients with short stature
- ZHONGGUO YAOFANG Vol. 36, Issue 9, Pages: 1111-1116(2025)
- 作者机构：
  
  1.西南医科大学药学院，四川泸州;646000
  2.乐山市人民医院药学部，四川乐山　614000
  3.四川省医学科学院·四川省人民医院（电子科技大学附属医院）药学部/个体化药物治疗四川省重点实验室，成都　610072
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2025.09.16
  CLC： R969;R956;R977.1
- Received：06 November 2024，
  
  Revised：15 March 2025，
  
  Accepted：2025-04-14，
  
  Published：15 May 2025
- 稿件说明：
移动端阅览
郑卓婷,刘易陇,覃小貌等.不同剂型短效rhGH治疗矮小症患者的有效性、安全性及经济性评价 ^Δ[J].中国药房,2025,36(09):1111-1116.

ZHENG Zhuoting,LIU Yilong,QIN Xiaomao,et al.Evaluation of the efficacy， safety and cost-effectiveness of different formulations of short-acting rhGH in the treatment of patients with short stature[J].ZHONGGUO YAOFANG,2025,36(09):1111-1116.
郑卓婷,刘易陇,覃小貌等.不同剂型短效rhGH治疗矮小症患者的有效性、安全性及经济性评价 ^Δ[J].中国药房,2025,36(09):1111-1116. DOI： 10.6039/j.issn.1001-0408.2025.09.16.

ZHENG Zhuoting,LIU Yilong,QIN Xiaomao,et al.Evaluation of the efficacy， safety and cost-effectiveness of different formulations of short-acting rhGH in the treatment of patients with short stature[J].ZHONGGUO YAOFANG,2025,36(09):1111-1116. DOI： 10.6039/j.issn.1001-0408.2025.09.16.

摘要

目的

比较两种不同剂型的短效重组人生长激素（rhGH）治疗矮小症患者的有效性、安全性和经济性。

方法

采用回顾性研究法，收集乐山市人民医院2016年8月－2023年6月经短效rhGH治疗的矮小症患者资料，按照患者使用的rhGH剂型分为粉剂组和水剂组，比较治疗12个月后两组患者生长相关疗效指标变化情况及不良反应发生情况；采用成本-效果分析和敏感性分析比较取得单位效果的成本；并根据患者临床诊断分为生长激素缺乏症（GHD）亚组和特发性矮小症（ISS）亚组进行亚组分析。

结果

治疗12个月后，粉剂组与水剂组患者的身高和血清胰岛素样生长因子1、胰岛素样生长因子结合蛋白3水平均较同组治疗前明显增高（

＜0.001），但两组间上述指标的变化值比较差异均无统计学意义（

＞0.05）；GHD和ISS亚组分析结果与总人群结果一致。总人群中，粉剂组患者的成本-效果比（2 582元/cm）明显优于水剂组（6 729元/cm），差异有统计学意义（

＜0.001），且该结果在GHD和ISS亚组患者和敏感性分析中表现一致。粉剂组与水剂组患者均未出现严重不良反应，两组患者各种不良反应发生率比较差异均无统计学意义（

＞0.05）。

结论

短效rhGH粉剂与水剂的有效性和安全性相当，但粉剂更具有经济学优势。

Abstract

OBJECTIVE

To compare the efficacy， safety， and cost-effectiveness of two different formulations of short-acting recombinant human growth hormone （rhGH） in the treatment of patients with short stature.

METHODS

Data from patients with short stature treated with short-acting rhGH at the Leshan People’s Hospital from August 2016 to June 2023 were collected. Patients were divided into powder formulation group and aqueous formulation group based on the rhGH formulation used. The changes in growth-related efficacy indicators and the occurrence of adverse drug reactions were compared between two groups after 12 months of treatment； cost-effectiveness analysis and sensitivity analysis were used to compare the cost per unit of effect achieved； subgroup analysis was performed by dividing the patients into growth hormone deficiency （GHD） subgroup and idiopathic short stature （ISS） subgroup based on clinical diagnosis.

RESULTS

After 12 months of treatment， the height and the levels of insulin-like growth factor-1 and insulin-like growth factor binding protein-3 in serum in aqueous formulation group and powder formulation group were significantly increased compared to before treatment （

＜0.001）， but there was no statistically significant difference in the changes of the above indicators between the two groups （

＞0.05）. The analysis results of GHD and ISS subgroups were consistent with the overall population. In the overall population， the cost-effectiveness ratio of powder formulation group （2 582 yuan/cm） was significantly better than that of aqueous formulation group （6 729 yuan/cm）， with a statistically significant difference （

＜0.001）， and the result was consistent in the GHD and ISS subgroups as well as in the sensitivity analysis. No serious adverse drug reactions occurred in either powder formulation or aqueous formulation group， and there was no statistically significant difference in the incidence of various adverse reactions between two groups （

＞0.05）.

CONCLUSIONS

Short-acting rhGH powder and aqueous formulations have equivalent efficacy and safety， but the powder formulation has greater economic advantages.

关键词

Keywords

references

姚海波，强梅 . 矮小身材儿童预防性筛查和临床治疗路径管理方案的探索 [J ] . 中国校医， 2017 ， 31 （ 2 ）： 87 - 89，91 .

杨云霞，任雅丽 . 生长激素长效制剂的研究概况 [J ] . 中国医药生物技术， 2021 ， 16 （ 5 ）： 459 - 464 .

乔建敏，班博，李鑫，等 . 中文版儿童生存质量量表在矮小症中的信效度分析 [J ] . 中国临床心理学杂志， 2017 ， 25 （ 2 ）： 276 - 279，284 .

闻钰，千茜茜，朱宁，等 . 重组人生长激素对儿童特发性矮小症的疗效及预测模型建立 [J/OL ] . 武汉大学学报（医学版）， 2024 ： 1 - 7 （ 2024-06-28 ）[ 2024-10-08 ] . https：//doi.org/10.14188/j.1671-8852.2023.0286 https://doi.org/10.14188/j.1671-8852.2023.0286 .

姜丽，赵庆兵，荚艳丽，等 . 短期重组人生长激素治疗生长激素缺乏症儿童的效果分析 [J ] . 国际医药卫生导报， 2021 ， 27 （ 8 ）： 1206 - 1209 .

中华医学会儿科学分会内分泌遗传代谢学组 . 矮身材儿童诊治指南 [J ] . 中华儿科杂志， 2008 ， 46 （ 6 ）： 428 - 430 .

王亚敏，李湛军 . 两种不同剂型重组人生长激素在体生物活性比较 [J ] . 中国生化药物杂志， 2008 ， 28 （ 6 ）： 392 - 394 .

GRIMBERG A ， DIVALL S A ， POLYCHRONAKOS C ， et al . Guidelines for growth hormone and insulin-like growth factor-1 treatment in children and adolescents：growth hormone deficiency，idiopathic short stature，and primary insulin-like growth factor-1 deficiency [J ] . Horm Res Paediatr ， 2016 ， 86 （ 6 ）： 361 - 397 .

李志鸿，沈伊娜，胡茂庆 . 两种不同剂型基因重组人生长激素治疗生长激素缺乏性矮小的临床研究 [J ] . 中国优生与遗传杂志， 2012 ， 20 （ 2 ）： 121 - 122，135 .

中华医学会儿科学分会内分泌遗传代谢学组，《中华儿科杂志》编辑委员会 . 基因重组人生长激素儿科临床规范应用的建议 [J ] . 中华儿科杂志， 2013 ， 51 （ 6 ）： 426 - 432 .

李湛军，苑方圆，梁成罡 . 重组人生长激素水针剂与冻干剂对去垂体大鼠的药效和生物活性比对研究 [J ] . 药物分析杂志， 2015 ， 35 （ 7 ）： 1140 - 1144 .

燕方龙，赵琳静，李洪森，等 . 国产与进口重组人生长激素治疗儿童原发性生长激素缺乏症的药物经济学分析 [J ] . 中国药房， 2012 ， 23 （ 30 ）： 2795 - 2798 .

方圣博，张晓荧，张欣，等 . 聚乙二醇重组人生长激素致儿童过敏性休克1例 [J ] . 药物流行病学杂志， 2017 ， 26 （ 3 ）： 219 - 220 .

MEHTA S ， OZA V ， POTASHNER R ， et al . Allergic and non-allergic skin reactions associated with growth hormone therapy：elucidation of causative agents [J ] . J Pediatr Endocrinol Metab ， 2018 ， 31 （ 1 ）： 5 - 11 .

张文慧，许峥嵘 . 不同BMI的特发性矮小症患儿应用重组人生长激素治疗的疗效及安全性分析 [J ] . 神经药理学报， 2024 ， 14 （ 3 ）： 47 - 53 .

梁欢，盛海，卫海燕，等 . 注射用重组人生长激素治疗特发性矮小症Ⅲ期临床试验研究 [J ] . 临床儿科杂志， 2023 ， 41 （ 10 ）： 685 - 691 .

王继锋，张薇，马勇 . 重组人生长激素注射液治疗生长激素缺乏症患儿的临床研究 [J ] . 中国临床药理学杂志， 2021 ， 37 （ 14 ）： 1864 - 1866，1877 .

SÄVENDAHL L ， POLAK M ， BACKELJAUW P ， et al . Long-term safety of growth hormone treatment in childhood：two large observational studies：NordiNet IOS and ANSWER [J ] . J Clin Endocrinol Metab ， 2021 ， 106 （ 6 ）： 1728 - 1741 .

LIEDERT B ， FORSSMANN U ， WOLNA P ， et al . Comparison of the pharmacokinetics，safety and tolerability of two concentrations of a new liquid recombinant human growth hormone formulation versus the freeze-dried formulation [J ] . BMC Clin Pharmacol ， 2010 ， 10 ： 14 .

STRAND V ， GONCALVES J ， ISAACS J D . Immunogenicity of biologic agents in rheumatology [J ] . Nat Rev Rheumatol ， 2021 ， 17 （ 2 ）： 81 - 97 .

Views

下载量

CSCD

Alert me when the article has been cited

提交

Tools

Publicity Resources

Rapid health technology assessment of efficacy， safety and cost-effectiveness of vonoprazan in the treatment of gastroesophageal reflux disease

Dupilumab for the treatment of severe asthma： a rapid health technology assessment

Rapid health technology assessment of ulinastatin in the treatment of acute pancreatitis

Tenecteplase in the treatment of acute ischemic stroke： a rapid health technology assessment

Rapid health technology assessment of the efficacy， safety and cost-effectiveness of vericiguat in the treatment of heart failure

Related Author

WANG Shuan

HUANG Can

QI Lamei

WANG Huayu

DONG Shichao

SUN Wei

WANG Ying

ZHENG Zihui

Related Institution

Dept. of Pharmaceutical Administration， Anqing Municipal Hospital

Dept. of Respiratory， the Second Hospital of Tianjin Medical University

Dept. of Pharmacy， the Second Hospital of Tianjin Medical University

Health Care Department， Security Bureau of Joint Staff Department of the Central Military Commission

Dept. of Pharmacy， Beijing Hospital/National Geriatric Center/Institute of Geriatrics， Chinese Academy of Medical Sciences/Beijing Key Laboratory of Clinical Risk and Individualized Application Evaluation of Drugs （Beijing Hospital）

Address：8th Floor, Sihuan Building, No. 129 Daping Main Street, Yuzhong District, Chongqing, China Postal code：400042
Technical support is provided by Beijing Founder Electronics co., LTD 渝ICP备15012710号公网安备50010302001817
It is recommended to read the content of this site in Chrome&IE9+. Please switch to extreme mode in browser 360.
Cookies We use cookies to help provide and enhance our service and tailor content. By continuing, you agree to the use of cookies.

⁰