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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
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| 篇名： | 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 |
| TITLE： | Evaluation of the efficacy，safety and cost-effectiveness of different formulations of short-acting rhGH in the treatment of patients with short stature |
| 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（P＜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（P＞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（2582元/cm）明显优于水剂组（6729元/cm），差异有统计学意义（P＜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（P＞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 （P＜0.001）， but there was no statistically significant difference in the changes of the above indicators between the two groups（P＞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 （P＜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 （P＞0.05）. CONCLUSIONS Short-acting rhGH powder and aqueous formulations have equivalent efficacy and safety， but the powder formulation has greater economic advantages. |
| 期刊： | 2025年第36卷第09期 |
| 作者： | 郑卓婷；刘易陇；覃小貌；曾珍；严润；龙恩武 |
| AUTHORS： | ZHENG Zhuoting，LIU Yilong，QIN Xiaomao，ZENG Zhen，YAN Run，LONG Enwu |
| 关键字： | 重组人生长激素；剂型；有效性；安全性；经济性；矮小症；生长激素缺乏症；特发性矮小症 |
| KEYWORDS： | recombinant human growth hormone； formulation； efficacy； safety； cost-effectiveness； short stature； growth |
| 阅读数： | 7 次 |
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