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BCG 无应答性非肌层浸润性膀胱癌不同治疗方式的成本效果分析。

Cost-effectiveness analysis of different treatment modalities in BCG-unresponsive NMIBC.

机构信息

Department of Urology, Urologic Oncology, Robot-Assisted and Specialized Urologic Surgery, University of Cologne, Cologne, Germany.

Faculty of Law, Business and Economics, University of Bayreuth, Bayreuth, Germany.

出版信息

BJU Int. 2024 Oct;134(4):582-588. doi: 10.1111/bju.16332. Epub 2024 Mar 16.

Abstract

OBJECTIVE

Radical cystectomy (RC) is the standard of care (SOC) in BCG-unresponsive NMIBC and is associated with a significant health-related quality-of-life burden. Recently, promising results have been published on Gemcitabine/Docetaxel, Pembrolizumab, and Hyperthermic Intravesical Chemotherapy (HIVEC) as salvage therapy options trying to increase the rate of bladder preservation. Here, we performed a Cost-Effectiveness-Analysis of those treatment modalities.

PATIENTS AND METHODS

We developed a Markov model from a payer's perspective drawing on clinical data of single-arm trials testing intravesical gemcitabine/docetaxel and pembrolizumab in BCG-unresponsive NMIBC, as well as clinical data from patients receiving hyperthermic intravesical chemotherapy HIVEC (n = 29) as intravesical salvage chemotherapy at our uro-oncological centre in Cologne. Costs were simulated utilising a non-commercial diagnosis-related groups grouper, utilities were derived from comparable cost-effectiveness studies. We used a Monte Carlo simulation to identify the optimal treatment, comparing the incremental cost effectiveness ratios (ICERs) at a willingness-to-pay threshold of €50 000 (euro)/quality-adjusted life year (QALY).

RESULTS

Over a horizon of 10 years, gemcitabine/docetaxel, HIVEC, and pembrolizumab were associated with costs of €48 353, €64 438, and €204 580, as well as a gain of QALYs of 6.16, 6.48, and 6.00, resulting in an ICER of €26 482, €42 567, and €184 533 respectively, in comparison to RC with total costs of €21 871 and a gain of QALYs of 5.01. Monte Carlo simulation identified HIVEC as the treatment of choice under assumption of a WTP of <€50 000.

CONCLUSION

Considering a WTP of <€50 000/QALY, gemcitabine/docetaxel and HIVEC are highly cost-effective therapeutic options in BCG-refractory NMIBC, while RC remains the cheapest option. At its current price, pembrolizumab would only be cost-effective assuming a price reduction of at least 70%.

摘要

目的

根治性膀胱切除术(RC)是卡介苗无反应性非肌层浸润性膀胱癌(NMIBC)的标准治疗方法(SOC),并与显著的健康相关生活质量负担相关。最近,在使用吉西他滨/多西他赛、派姆单抗和高热腔内化疗(HIVEC)作为挽救性治疗选择方面,已经发表了有希望的结果,试图提高膀胱保留率。在这里,我们对这些治疗方法进行了成本效益分析。

患者和方法

我们从支付者的角度出发,基于单臂试验的临床数据,开发了一个 Markov 模型,该模型测试了吉西他滨/多西他赛和派姆单抗在卡介苗无反应性 NMIBC 中的应用,以及在我们位于科隆的泌尿肿瘤中心接受高热腔内化疗 HIVEC(n=29)作为腔内挽救性化疗的患者的临床数据。利用非商业性诊断相关组分组器模拟成本,效用来自可比的成本效益研究。我们使用蒙特卡罗模拟来确定最佳治疗方法,比较在愿意支付的阈值为 50000 欧元/质量调整生命年(QALY)时的增量成本效益比(ICER)。

结果

在 10 年的时间内,吉西他滨/多西他赛、HIVEC 和派姆单抗的成本分别为 48353 欧元、64438 欧元和 204580 欧元,以及 QALY 的增加量分别为 6.16、6.48 和 6.00,因此,与总成本为 21871 欧元、QALY 增加量为 5.01 的 RC 相比,ICER 分别为 26482 欧元、42567 欧元和 184533 欧元。蒙特卡罗模拟确定,在假设支付意愿(WTP)低于 50000 欧元的情况下,HIVEC 是 BCG 耐药性 NMIBC 的首选治疗方法。

结论

考虑到低于 50000 欧元/QALY 的支付意愿,吉西他滨/多西他赛和 HIVEC 是卡介苗无反应性非肌层浸润性膀胱癌(NMIBC)的高效治疗选择,而 RC 仍然是最便宜的选择。在目前的价格下,只有假设价格降低至少 70%,派姆单抗才具有成本效益。

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