Background

The recommended treatment for patients with Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC) is radical cystectomy (RC). However, many patients refuse, or are unfit for RC. Therefore, alternative bladder-sparing treatment modalities are needed for BCG-unresponsive NMIBC. In this study we sought to assess the long-term efficacy of hyperthermic intravesical chemotherapy (HIVEC) as alternative to radical cystectomy in BCG-unresponsive non-muscle invasive bladder cancer patients.

Methods and materials

Retrospectively collected data from 56 patients with BCG-unresponsive NMIBC who received ≥5 HIVEC instillations between October 2014 and March 2020 was analyzed. All patients met the BCG-unresponsive criteria according to the current EAU guideline on NMIBC 2020. Patients were followed-up with cystoscopy and/or bladder biopsies, urine cytology and annually CT-urography. The Primary outcome was the high grade (HG) recurrence-free survival (RFS), defined as the time from the first HIVEC instillation until histologically confirmed intravesical recurrence or last follow-up. The Kaplan Meier method was used to estimate survival outcomes. Secondary outcomes were: complete response rate (CR), adverse events (AE), assessed by the Common Terminology Criteria for Adverse Events v5.0 (CTCAE) and tumor progression to muscle invasive disease or distant metastases.

Results

The median follow-up was 32.2 months (IQR 13.7–44.8). The 1- and 2-year HG-RFS was 53% (SE:6.8) and 35% (SE:6.9), respectively. The CR for patients with CIS was 70% (21/30) at 6 months. Overall, 80% of the population developed an AE, only 1 was classified as CTCAE ≥3. Limitation of this study was the small sample size.

Conclusion

HIVEC resulted in a 2-year HG-RFS of 35% for BCG-unresponsive NMIBC patients without severe side-effects and therefore HIVEC seems to be an alternative treatment option for patients who refuse or are unfit for RC.

中文翻译：

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膀胱内热灌注化疗治疗卡介苗无反应性非肌层浸润性膀胱癌的长期疗效

背景

对卡介苗(BCG) 无反应性非肌肉浸润性膀胱癌 (NMIBC)患者的推荐治疗是根治性膀胱切除术 (RC)。然而，许多患者拒绝或不适合 RC。因此，对于 BCG 无反应的 NMIBC，需要替代保留膀胱的治疗方式。在这项研究中，我们试图评估热膀胱内化疗 (HIVEC) 作为根治性膀胱切除术替代 BCG 无反应的非肌肉浸润性膀胱癌患者的长期疗效。

方法和材料

分析了 2014 年 10 月至 2020 年 3 月期间接受 ≥5 次 HIVEC 滴注的 56 名卡介苗无反应 NMIBC 患者的回顾性收集数据。根据 NMIBC 2020 现行 EAU 指南，所有患者均符合 BCG 无反应标准。对患者进行膀胱镜检查和/或膀胱活检、尿细胞学检查和每年一次的 CT 尿路造影。主要结果是高级别 (HG) 无复发生存期 (RFS)，定义为从第一次 HIVEC 滴注到组织学证实膀胱内复发或最后一次随访的时间。Kaplan Meier 方法用于估计生存结果。次要结果是：完全缓解率 (CR)、不良事件 (AE)，由不良事件通用术语标准 v5 评估。

结果

中位随访时间为 32.2 个月（IQR 13.7-44.8）。1 年和 2 年 HG-RFS 分别为 53% (SE:6.8) 和 35% (SE:6.9)。6 个月时 CIS 患者的 CR 为 70% (21/30)。总体而言，80% 的人群发生了 AE，只有 1 人被归类为 CTCAE ≥3。这项研究的局限性是样本量小。

结论

HIVEC 导致 BCG 无反应的 NMIBC 患者的 2 年 HG-RFS 为 35%，没有严重的副作用，因此 HIVEC 似乎是拒绝或不适合 RC 的患者的替代治疗选择。