【摘要】 目的 比较促性腺激素释放激素类似物（GnRHa）联合重组人生长激素（rhGH）（联用方案）和GnRHa单药（单用方案）治疗中枢性性早熟（CPP）的长期经济性。方法 从全社会角度出发，基于四川大学华西第二医院开展的真实世界研究，以终身高为效果指标，以2022年我国农村及城镇居民人均可支配收入（20 133～49 283元）作为社会意愿支付（WTP）阈值，采用成本-效果分析法比较上述两种药物方案治疗CPP女孩的长期经济性。采用单因素敏感性分析和概率敏感性分析验证基础分析结果的稳健性，并通过情境分析比较不同长效制剂组合方案的经济性。结果 基础分析结果显示，与单用方案相比，采用联用方案每增加CPP女孩1 cm终身高需多花费25 193.49元；联用方案对于农村地区居民不具有经济性，但对于城镇地区居民具有经济性。单因素敏感性分析结果显示，对基础分析结果可能产生影响的不确定因素主要为rhGH价格、联用方案组患儿终身高、联用方案组rhGH治疗时间、单用方案组患儿终身高。概率敏感性分析结果显示，当WTP阈值大于26 010元/cm时，联用方案具有经济性的概率均高于单用方案。情境分析结果显示，当使用GnRHa长效制剂时，每3个月肌内注射1次的联用方案对于农村地区居民不具有经济性，但对于城镇地区居民具有经济性；当使用rhGH长效制剂时，每周皮下注射1次的联用方案对于农村地区和城镇地区居民均不具有经济性。结论 推荐家庭经济负担能力相对较强的CPP患儿在GnRHa治疗的基础上额外联用rhGH来改善终身高；应考虑治疗的获益、风险和可负担性，避免盲目追求身高增长而滥用rhGH。更多还原

【Abstract】 OBJECTIVE To compare the long-term costeffectiveness of gonadotrophin-releasing hormone analogue(GnRHa) combined with recombinant human growth hormone(rhGH)(combination therapy regimen) versus GnRHa monotherapy(monotherapy regimen) in the treatment of central precocious puberty(CPP). METHODS From the societal perspective and based on a real-world study conducted at West China Second Hospital of Sichuan University, the cost-effectiveness analysis was performed to compare the long-term cost-effectiveness of two pharmacotherapy regimens for CPP girls, with final height as outcome indexes, using per capita disposable income of rural residents and urban residents(20 133-49 283 yuan) in 2022as the social willing-to-pay(WTP) threshold. The robustness of the basic analysis result was verified by using one-way sensitivity analysis and probability sensitivity analysis, and the cost-effectiveness of different combinations of long-acting preparations was compared using scenario analysis. RESULTS The basic analysis result showed that the combination therapy regimen required an additional cost of 25 193.49 yuan for every one-centimeter improvement in the final height of girls with CPP compared with the monotherapy regimen, which was not cost-effective for residents in rural areas, but it was cost-effective for residents in urban areas. One-way sensitivity analysis showed that the uncertain factors with potential impacts on the results were, in order, the price of rhGH, the final height of pediatric patients in the combination therapy regimen group, the course of rhGH in the combination therapy regimen group, and the final height of pediatric patients in the monotherapy regimen group. Probabilistic sensitivity analysis indicated that the probability of the combination therapy regimen being cost-effective was higher than that of the monotherapy regimen when WTP was more than 26 010 yuan/cm. When GnRHa long-acting preparation was used for intramuscular injection every 3 months, the combination therapy regimen was not cost-effective for rural residents, but was cost-effective for urban residents; when rhGH long-acting preparation was injected subcutaneously once a week, the combination therapy regimen was not cost-effective for residents in both rural areas and urban areas. CONCLUSIONS The combination of GnRHa and rhGH is only recommended for CPP children with better affordability to improve final height. The benefits, risks, and affordability of treatment should be comprehensively considered before the decisions on pharmacotherapy, to avoid abuse of rhGH due to the blind pursuit of height growth.更多还原