Department of Urology, Mayo Clinic, Rochester, Minnesota.
Department of Urology, David Geffen School of Medicine; University of California, Los Angeles, California.
J Urol. 2021 May;205(5):1326-1335. doi: 10.1097/JU.0000000000001515. Epub 2020 Dec 21.
Patients with bacillus Calmette-Guérin-unresponsive carcinoma in situ are treated with radical cystectomy or salvage intravesical chemotherapy. Recently, pembrolizumab was approved for bacillus Calmette-Guérin-unresponsive carcinoma in situ.
We used a decision-analytic Markov model to compare pembrolizumab, salvage intravesical chemotherapy (with gemcitabine-docetaxel induction+monthly maintenance) and radical cystectomy for patients with bacillus Calmette-Guérin-unresponsive carcinoma in situ who are radical cystectomy candidates (index patient 1) or are unwilling/unable to undergo radical cystectomy (index patient 2). The model used a U.S. Medicare perspective with a 5-year time horizon. One-way and probabilistic sensitivity analyses were performed. Incremental cost-effectiveness ratios were compared using a willingness to pay threshold of $100,000/quality-adjusted life year.
For index patient 1, pembrolizumab was not cost-effective relative to radical cystectomy (incremental cost-effectiveness ratios $1,403,008/quality-adjusted life year) or salvage intravesical chemotherapy (incremental cost-effectiveness ratios $2,011,923/quality-adjusted life year). One-way sensitivity analysis revealed that pembrolizumab only became cost-effective relative to radical cystectomy with a >93% price reduction. Relative to radical cystectomy, salvage intravesical chemotherapy was cost-effective for time horizons <5 years and nearly cost-effective at 5 years (incremental cost-effectiveness ratios $118,324/quality-adjusted life year). One-way sensitivity analysis revealed that salvage intravesical chemotherapy became cost-effective relative to radical cystectomy if risk of recurrence or metastasis at 2 years was less than 55% or 5.9%, respectively. For index patient 2, pembrolizumab required >90% price reduction to be cost-effective (incremental cost-effectiveness ratios $1,073,240/quality-adjusted life year). Pembrolizumab was cost-effective in 0% of 100,000 microsimulations in probabilistic sensitivity analyses for both index patients.
At its current price, pembrolizumab is not cost-effective for bacillus Calmette-Guérin-unresponsive carcinoma in situ relative to radical cystectomy or salvage intravesical chemotherapy. Although gemcitabine-docetaxel is not cost-effective relative to radical cystectomy at 5 years, further studies may validate its cost-effectiveness if recurrence and metastasis thresholds are met.
卡介苗无反应原位癌患者接受根治性膀胱切除术或挽救性膀胱内化疗。最近，派姆单抗被批准用于卡介苗无反应原位癌。
我们使用决策分析马尔可夫模型比较了派姆单抗、挽救性膀胱内化疗（吉西他滨-多西他赛诱导+每月维持）和根治性膀胱切除术在适合根治性膀胱切除术的卡介苗无反应原位癌患者（指数患者 1）或不愿意/无法接受根治性膀胱切除术的患者（指数患者 2）中的应用。该模型采用美国医疗保险视角，时间范围为 5 年。进行了单因素和概率敏感性分析。使用愿意支付 10 万美元/质量调整生命年的阈值比较增量成本效益比。
对于指数患者 1，派姆单抗相对于根治性膀胱切除术（增量成本效益比为 1403008 美元/质量调整生命年）或挽救性膀胱内化疗（增量成本效益比为 2011923 美元/质量调整生命年）并不具有成本效益。单因素敏感性分析表明，只有当派姆单抗价格降低超过 93%时，它才相对于根治性膀胱切除术具有成本效益。与根治性膀胱切除术相比，挽救性膀胱内化疗在 5 年以下的时间范围内具有成本效益，在 5 年时几乎具有成本效益（增量成本效益比为 118324 美元/质量调整生命年）。单因素敏感性分析表明，如果 2 年内复发或转移的风险分别小于 55%或 5.9%，则挽救性膀胱内化疗相对于根治性膀胱切除术具有成本效益。对于指数患者 2，派姆单抗需要降低超过 90%的价格才能具有成本效益（增量成本效益比为 1073240 美元/质量调整生命年）。在对两名指数患者的 10 万次微模拟的概率敏感性分析中，派姆单抗在 0%的情况下具有成本效益。
在目前的价格下，派姆单抗相对于根治性膀胱切除术或挽救性膀胱内化疗在卡介苗无反应原位癌方面不具有成本效益。虽然吉西他滨-多西他赛在 5 年内相对于根治性膀胱切除术不具有成本效益，但如果满足复发和转移的阈值，进一步的研究可能会验证其成本效益。
