Division of Epidemiology, Department of Internal Medicine, University of Utah (UT) School of Medicine, Salt Lake City, UT, USA.
Department of Population Health Sciences, University of UT, Salt Lake City, UT, USA.
J Natl Cancer Inst. 2023 Nov 8;115(11):1374-1382. doi: 10.1093/jnci/djad135.
Recently, several new treatment regimens have been approved for treating metastatic hormone-sensitive prostate cancer, building on androgen deprivation therapy alone. These include docetaxel androgen deprivation therapy, abiraterone acetate-prednisone androgen deprivation therapy, apalutamide androgen deprivation therapy, enzalutamide androgen deprivation therapy, darolutamide-docetaxel androgen deprivation therapy, and abiraterone-prednisone androgen deprivation therapy with docetaxel. There are no validated predictive biomarkers for choosing a specific regimen. The goal of this study was to conduct a health economic outcome evaluation to determine the optimal treatment from the US public sector (Veterans Affairs).
We developed a partitioned survival model in which metastatic hormone-sensitive prostate cancer patients transitioned between 3 health states (progression free, progressive disease to castrate resistance state, and death) at monthly intervals based on Weibull survival model estimated from published Kaplan-Meier curves using a Bayesian network meta-analysis of 7 clinical trials (7208 patients). The effectiveness outcome in our model was quality-adjusted life-years (QALYs). Cost input parameters included initial and subsequent treatment costs and costs for terminal care and for managing grade 3 or higher drug-related adverse events and were obtained from the Federal Supply Schedule and published literature.
Average 10-year costs ranged from $34 349 (androgen deprivation therapy) to $658 928 (darolutamide-docetaxel androgen deprivation therapy) and mean QALYs ranged from 3.25 (androgen deprivation therapy) to 4.57 (enzalutamide androgen deprivation therapy). Treatment strategies docetaxel androgen deprivation therapy, enzalutamide androgen deprivation therapy docetaxel, apalutamide androgen deprivation therapy, and darolutamide-docetaxel androgen deprivation therapy were eliminated because of dominance (ie, they were more costly and less effective than other strategies). Of the remaining strategies, abiraterone acetate-prednisone androgen deprivation therapy was the most cost-effective strategy at a willingness-to-pay threshold of $100 000/QALY (incremental cost-effectiveness ratios = $21 247/QALY).
Our simulation model found abiraterone acetate-prednisone androgen deprivation therapy to be an optimal first-line treatment for metastatic hormone-sensitive prostate cancer from a public (Veterans Affairs) payer perspective.
最近，已有几种新的治疗方案被批准用于治疗转移性去势敏感性前列腺癌，这些方案都是在雄激素剥夺疗法的基础上进一步制定的。这些方案包括多西他赛联合雄激素剥夺疗法、醋酸阿比特龙联合泼尼松龙雄激素剥夺疗法、阿帕鲁胺联合雄激素剥夺疗法、恩扎卢胺联合雄激素剥夺疗法、达罗他胺联合多西他赛联合雄激素剥夺疗法以及醋酸阿比特龙联合泼尼松龙联合多西他赛雄激素剥夺疗法。目前还没有经过验证的预测生物标志物来选择特定的方案。本研究的目的是进行卫生经济学结果评估，以确定从美国公共部门（退伍军人事务部）的角度来看最佳的治疗方案。
我们开发了一个分区生存模型，根据发表的 Kaplan-Meier 曲线的贝叶斯网络荟萃分析，使用威布尔生存模型对 7 项临床试验（7208 例患者）进行估计，转移性去势敏感性前列腺癌患者每月在 3 种健康状态（无进展、进展性疾病至去势抵抗状态和死亡）之间转换。我们模型中的疗效结果是质量调整生命年（QALYs）。成本投入参数包括初始和后续治疗成本、终末期护理成本以及管理 3 级或更高级别药物相关不良事件的成本，这些成本来自联邦供应计划和已发表的文献。
平均 10 年的成本范围从 34349 美元（雄激素剥夺疗法）到 658928 美元（达罗他胺联合多西他赛雄激素剥夺疗法），平均 QALYs 范围从 3.25（雄激素剥夺疗法）到 4.57（恩扎卢胺联合雄激素剥夺疗法）。多西他赛联合雄激素剥夺疗法、多西他赛联合恩扎卢胺雄激素剥夺疗法、阿帕鲁胺联合雄激素剥夺疗法和达罗他胺联合多西他赛雄激素剥夺疗法因具有优势（即，它们的成本更高，效果更差）而被排除在外。在剩余的治疗策略中，醋酸阿比特龙联合泼尼松龙雄激素剥夺疗法在 100000 美元/QALY 的支付意愿阈值下是最具成本效益的策略（增量成本效益比为 21247 美元/QALY）。
我们的模拟模型发现，从公共（退伍军人事务部）支付者的角度来看，醋酸阿比特龙联合泼尼松龙雄激素剥夺疗法是转移性去势敏感性前列腺癌的一种最佳一线治疗方法。
