Dana-Farber Cancer Institute, Boston, MA, USA.
McMaster University, Hamilton, ON, Canada.
BJU Int. 2021 Nov;128(5):607-614. doi: 10.1111/bju.15434. Epub 2021 May 18.
To evaluate outcomes of patients achieving a post-treatment pathological stage of <ypT2N0 at radical cystectomy (RC) following neoadjuvant chemotherapy (NAC) for muscle-invasive bladder cancer (MIBC) to identify an optimal definition of pathological response.
Patients from 10 international centres who underwent NAC for cT2-4aN0-1 MIBC and achieved <ypT2N0 disease at RC were included. The primary outcome was time to recurrence, either local or distant. Kaplan-Meier and Cox proportional hazards regression were used to evaluate associations between clinicopathological variables and outcomes.
A total of 625 patients were included. The median age was 66 years and 80% were male. Gemcitabine and cisplatin (GC, 56%) and methotrexate, vinblastine, doxorubicin and cisplatin (MVAC)/dose-dense (dd)MVAC (32%) were the most common NAC regimens. ypT0, pure ypTis, ypTa ±ypTis and ypT1 ± ypTis were attained in 58.1%, 20.0%, 7.6% and 14.2% of patients, respectively. The cumulative incidence of recurrence at 5 years was 9%, 16%, 29% and 30%, respectively. Pathological stage was prognostic for recurrence, with ypTa ± Tis (hazard ratio [HR] 3.20, 95% confidence interval [CI] 1.40-7.30) and ypT1 ± Tis disease (HR 4.03, 95% CI 2.13-7.63) associated with a significantly higher recurrence risk. Pure ypTis (HR 1.66, 95% CI 0.82-3.38) and the type of NAC regimen (ddMVAC: HR 1.59, 95% CI 0.55-4.56; MVAC: HR 1.18, 9%% CI 0.25-5.54; reference: GC) were not associated with recurrence.
We propose that optimal pathological response after NAC be defined as attainment of ypT0N0/ypTisN0 at RC. Patients with ypTaN0 or ypT1N0 disease (with or without Tis) at RC displayed a significantly higher risk of recurrence and may be candidates for trials investigating adjuvant therapy.
评估肌层浸润性膀胱癌（MIBC）患者接受新辅助化疗（NAC）后在根治性膀胱切除术（RC）时达到病理分期 <ypT2N0 的治疗后结局，以确定病理反应的最佳定义。
纳入了来自 10 个国际中心的、接受 NAC 治疗 cT2-4aN0-1 MIBC 并在 RC 时达到 <ypT2N0 疾病的患者。主要结局是局部或远处复发的时间。采用 Kaplan-Meier 和 Cox 比例风险回归评估临床病理变量与结局之间的关联。
共纳入 625 例患者。中位年龄为 66 岁，80%为男性。GC（56%）和 MVAC/ddMVAC（32%）是最常见的 NAC 方案。ypT0、单纯 ypTis、ypTa±ypTis 和 ypT1±ypTis 在患者中的比例分别为 58.1%、20.0%、7.6%和 14.2%。5 年时的累积复发率分别为 9%、16%、29%和 30%。病理分期与复发相关，ypTa±Tis（风险比 [HR] 3.20，95%置信区间 [CI] 1.40-7.30）和 ypT1±Tis 疾病（HR 4.03，95% CI 2.13-7.63）与更高的复发风险显著相关。单纯 ypTis（HR 1.66，95% CI 0.82-3.38）和 NAC 方案类型（ddMVAC：HR 1.59，95% CI 0.55-4.56；MVAC：HR 1.18，9%CI 0.25-5.54；参考：GC）与复发无关。
我们建议将 NAC 后最佳的病理反应定义为 RC 时达到 ypT0N0/ypTisN0。RC 时达到 ypTaN0 或 ypT1N0 疾病（伴或不伴Tis）的患者复发风险显著增加，可能是辅助治疗试验的候选者。
