Joyce Daniel D, Wymer Kevin M, Graves John A, Boorjian Stephen A, Gore John L, Khaki Ali Raza, Raldow Ann C, Williams Stephen B, Smith Angela B, Sharma Vidit
Department of Urology, Vanderbilt University Medical Center, Nashville, Tennessee.
Department of Urology, Mayo Clinic, Rochester, Minnesota.
JAMA Netw Open. 2025 Jun 2;8(6):e2517056. doi: 10.1001/jamanetworkopen.2025.17056.
Trimodal therapy (TMT) is included as an alternative to radical cystectomy (RC) for definitive management of muscle-invasive bladder cancer (MIBC) in current clinical guidelines. Moreover, a 2023 retrospective analysis reported similar oncologic outcomes between these treatments among patients deemed fit for RC. Data regarding the comparative value of these treatments are lacking.
To evaluate the comparative cost-effectiveness of TMT and RC for treatment of MIBC.
DESIGN, SETTING, AND PARTICIPANTS: This economic evaluation compared cost-effectiveness of treatments using a health transition state microsimulation model of patients with bladder cancer treated between 2005 and 2017 with 5- and 10-year horizons from a Medicare perspective. Model probabilities were informed by multicenter retrospective analyses published in 2023 comparing TMT with RC. The index patient was aged 71 years, with clinical stage T2-4aN0M0 MIBC, solitary tumor smaller than 7 cm, no or unilateral hydronephrosis, adequate bladder function, and lack of multifocal or extensive carcinoma in situ. Patients unfit for RC, radiation, or cisplatin-based chemotherapy were excluded.
TMT and RC.
Primary outcomes included effectiveness measured in quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICER) using a willingness-to-pay threshold of $100 000 per QALY. Sensitivity analyses were performed to assess the robustness of the model.
For the index patient, at 5 years, the average cost was $30 525 higher for TMT than RC. Average QALYs were 3.87 and 3.94 for RC and TMT, respectively. As such, TMT was not cost-effective at 5-year (ICER, $464 291 per QALY) or 10-year (ICER, $308 638 per QALY) time horizons. On 1-way sensitivity analyses, TMT would become cost-effective if (1) TMT costs were less than $17 605; or (2) TMT resulted in an 11.6% improvement in metastasis-free survival relative to RC.
In this economic evaluation study of TMT and RC for treatment of MIBC, TMT was associated with improved quality of life but was not cost-effective relative to RC at 5 and 10 years given higher treatment costs. These findings highlight the importance of developing policy initiatives that help reduce TMT costs and of providing patients with accurate expectations of long-term toxic effects to help guide preference-sensitive care.
在当前临床指南中，三联疗法（TMT）被列为根治性膀胱切除术（RC）的替代方案，用于肌肉浸润性膀胱癌（MIBC）的确定性治疗。此外，一项2023年的回顾性分析报告称，在被认为适合接受RC治疗的患者中，这两种治疗方法的肿瘤学结局相似。关于这些治疗方法的比较价值的数据尚缺。
评估TMT和RC治疗MIBC的比较成本效益。
设计、设置和参与者：这项经济评估使用了一个健康转变状态微观模拟模型，从医疗保险的角度比较了2005年至2017年接受治疗的膀胱癌患者在5年和10年时间范围内TMT和RC治疗的成本效益。模型概率基于2023年发表的多中心回顾性分析，该分析比较了TMT和RC。索引患者为71岁，临床分期为T2 - 4aN0M0的MIBC，孤立肿瘤小于7厘米，无或单侧肾盂积水，膀胱功能良好，且无多灶性或广泛性原位癌。不适合接受RC、放疗或基于顺铂化疗的患者被排除。
TMT和RC。
主要结局包括以质量调整生命年（QALYs）衡量的有效性，以及使用每QALY支付意愿阈值为100,000美元的增量成本效益比（ICER）。进行敏感性分析以评估模型的稳健性。
对于索引患者，在5年时，TMT的平均成本比RC高30,525美元。RC和TMT的平均QALYs分别为3.87和3.94。因此，在5年（ICER为每QALY 464,291美元）或10年（ICER为每QALY 308,638美元）的时间范围内，TMT不具有成本效益。在单因素敏感性分析中，如果（1）TMT成本低于17,605美元；或（2）相对于RC，TMT使无转移生存期提高11.6%，则TMT将具有成本效益。
在这项关于TMT和RC治疗MIBC的经济评估研究中，TMT与生活质量改善相关，但由于治疗成本较高，在5年和10年时相对于RC不具有成本效益。这些发现凸显了制定有助于降低TMT成本的政策举措以及为患者提供对长期毒性作用的准确预期以帮助指导偏好敏感型医疗的重要性。
