Department of Urology, Mayo Clinic, Rochester, Minnesota.
Department of Urology, David Geffen School of Medicine; Department of Health Policy and Management, Fielding School of Public Health; School of Nursing University of California, Los Angeles, California.
J Urol. 2020 Sep;204(3):442-449. doi: 10.1097/JU.0000000000001023. Epub 2020 Mar 19.
While guidelines support the use of maintenance bacillus Calmette-Guérin for patients with intermediate and high risk nonmuscle invasive bladder cancer, in an era of bacillus Calmette-Guérin shortage we explored the cost-effectiveness of maintenance bacillus Calmette-Guérin.
A Markov model compared the cost-effectiveness of maintenance bacillus Calmette-Guérin to surveillance after induction bacillus Calmette-Guérin for intermediate/high risk nonmuscle invasive bladder cancer from a U.S. Medicare perspective. Five-year oncologic outcomes, toxicity rates and utility values were extracted from the literature. Univariable and multivariable sensitivity analyses were conducted. A willingness to pay threshold of $100,000 per quality adjusted life year was considered cost-effective.
At 5 years mean costs per patient were $14,858 and $13,973 for maintenance bacillus Calmette-Guérin and surveillance, respectively, with quality adjusted life years of 4.046 for both, making surveillance the dominant strategy. On sensitivity analysis full dose and 1/3 dose maintenance bacillus Calmette-Guérin became cost-effective if the absolute reduction in 5-year progression was greater than 2.1% and greater than 0.76%, respectively. On further sensitivity analysis full dose and 1/3 dose maintenance bacillus Calmette-Guérin became cost-effective when maintenance bacillus Calmette-Guérin toxicity equaled surveillance toxicity. In multivariable sensitivity analyses using 100,000 Monte-Carlo microsimulations, full dose and 1/3 dose maintenance bacillus Calmette-Guérin was cost-effective in 17% and 39% of microsimulations, respectively.
Neither full dose nor 1/3 dose maintenance bacillus Calmette-Guérin appears cost-effective for the entire population of patients with intermediate/high risk nonmuscle invasive bladder cancer. These data support prioritizing maintenance bacillus Calmette-Guérin for the subset of patients with high risk nonmuscle invasive bladder cancer most likely to experience progression, in particular those who tolerated induction bacillus Calmette-Guérin well. Overall, our findings support the American Urological Association policy statement to allocate bacillus Calmette-Guérin for induction rather than maintenance therapy during times of bacillus Calmette-Guérin shortage.
虽然指南支持使用卡介苗维持治疗中高危非肌层浸润性膀胱癌患者,但在卡介苗短缺的时代,我们探索了卡介苗维持治疗的成本效益。
从美国医疗保险的角度出发,采用马尔可夫模型比较了诱导卡介苗治疗后中/高危非肌层浸润性膀胱癌行卡介苗维持治疗与监测的成本效益。从文献中提取了 5 年肿瘤学结果、毒性发生率和效用值。进行了单变量和多变量敏感性分析。愿意支付每质量调整生命年 100000 美元的阈值被认为是具有成本效益的。
在 5 年的时间里,每位患者的平均费用分别为维持卡介苗治疗组的 14858 美元和监测组的 13973 美元,两者的质量调整生命年均为 4.046,这使得监测成为主导策略。在敏感性分析中,如果 5 年进展的绝对降低大于 2.1%和大于 0.76%,则全剂量和 1/3 剂量的卡介苗维持治疗变得具有成本效益。在进一步的敏感性分析中,如果卡介苗维持治疗的毒性与监测的毒性相等,则全剂量和 1/3 剂量的卡介苗维持治疗变得具有成本效益。在使用 100000 次蒙特卡罗微模拟的多变量敏感性分析中,全剂量和 1/3 剂量的卡介苗维持治疗在 17%和 39%的微模拟中具有成本效益。
对于中高危非肌层浸润性膀胱癌患者的整个群体,全剂量和 1/3 剂量的卡介苗维持治疗都不具有成本效益。这些数据支持将卡介苗维持治疗优先用于高危非肌层浸润性膀胱癌患者亚组,特别是那些能很好耐受诱导卡介苗治疗的患者。总的来说,我们的研究结果支持美国泌尿外科学会的政策声明,即在卡介苗短缺时期,将卡介苗用于诱导治疗,而不是维持治疗。