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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 2022
as 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.
KEYWORDS central precocious puberty； gonadotropin-releasing hormone analogue； recombinant human growth hormone；
height； pharmacoeconomics； cost-effectiveness analysis
[8]
中枢性性早熟（central precocious puberty，CPP）是 家长进行充分沟通后再行决策 。目前国内仅有一项与
由于下丘脑-垂体-性腺轴功能提前启动而导致第二性征 CPP 药物治疗相关的经济学研究 ，但该研究仅比较了
[9]
呈现及内、外生殖器官快速发育的一种儿科内分泌系统 不同GnRHa品种和剂型的成本差异，尚未涉及rhGH，仍
疾病。每 10 万人中有 200～410 名女孩、10～50 名男孩 缺乏 GnRHa 联合 rhGH 用药与 GnRHa 单独用药“头对
患有 CPP，其中女孩发病率为男孩的 15～20 倍 [1―3] ；同 头”比较的药物经济学研究。因此，本研究基于一项前
时，世界各国儿童CPP的发病率呈逐年升高的趋势 [4―5] ， 期在四川大学华西第二医院（以下简称“我院”）开展的
青春期启动年龄普遍提前，平均每 10 年下降 0.24 岁 。 真实世界研究结果，比较了GnRHa联合rhGH和GnRHa
[6]
调查数据显示，性早熟的就诊人数（尤其是CPP）已位居 单药治疗CPP女孩的长期经济性，以期为合理选择更具
[7]
儿科门诊疾病的首位 。 有成本-效果优势的CPP治疗方案提供证据支持。
CPP 的标准治疗药物为促性腺激素释放激素类似 1 资料与方法
物（gonadotropin-releasing hormone analogue，GnRHa）， 1.1 研究框架
该类药物主要通过抑制下丘脑-垂体-性腺轴功能，控制 1.1.1 研究角度
性发育进程，延缓骨龄进展，改善患儿终身高。研究发 本研究的研究角度为全社会角度。
现，CPP患儿的身高增长速度在接受GnRHa治疗后呈现 1.1.2 目标人群
不同程度的下降，部分患儿甚至出现较为明显的生长减 目标人群为确诊为 CPP 的女孩。纳入标准为：（1）
[8]
速，其生长速率显著低于正常青春前期水平 。20 世纪 年龄＜18 岁的女孩；（2）8 岁前出现乳房结节或 10 岁前
90 年代国外有研究者提出，在 GnRHa 基础上加用重组 发生月经初潮，或在界定年龄后出现第二性征但Tanner
人生长激素（recombinant human growth hormone，rhGH） 分期进展时间＜6个月；（3）血清黄体生成素（luteinizing
治疗 CPP，rhGH 可通过作用于生长激素-胰岛素样生长 hormone，LH）基础值≥0.83 IU/mL，或基于免疫化学发
因子 1 轴，协同促进儿童骨骼生长板的发育并改善身 光法的GnRH激发试验呈阳性，即LH峰值≥5.0 IU/L且
高 。但是，在GnRHa治疗成本的基础上引入价格昂贵 LH 峰值/卵泡刺激素峰值≥0.6；（4）盆腔彩超见卵巢容
[8]
且用药频次更高的 rhGH，可能进一步增加患儿家庭的 积≥1 mL，内见多个直径≥4 mm 的卵泡；（5）骨龄超过
医疗负担和经济压力。我国相关专家共识提出，联合用 实际年龄。
药前应反复评估CPP对身高的影响程度、患儿及其家长 排除标准为：（1）有明确病因的继发性CPP，包括中
对身高的接受程度、药物经济学等因素，并与患儿及其 枢神经系统异常（如占位性病变、肿瘤、感染、获得性损
· 80 · China Pharmacy 2025 Vol. 36 No. 1 中国药房 2025年第36卷第1期
