PURPOSE To evaluate the efficacy and safety of UGN-102 chemoablation for the primary treatment of patients with recurrent low-grade intermediate-risk nonmuscle-invasive bladder cancer. MATERIALS AND METHODS ENVISION is an ongoing, multinational, single-arm, Phase 3 study in patients with a biopsy-proven recurrence of untreated low-grade intermediate-risk nonmuscle-invasive bladder cancer. Patients received 6 weekly intravesical instillations of UGN-102 (mitomycin; outpatient setting) and were evaluated at 3 months. Patients achieving complete response (CR; negative cystoscopic examination, cytology, and for-cause biopsy) were surveilled regularly until recurrence, progression, or death. Patients who remain disease-free will be followed up to 5 years, and further results will be reported in the future. RESULTS Of 240 patients enrolled, 228 (95%) received all 6 planned doses; 191 (79.6%; 95% CI, 73.9-84.5) achieved CR at 3 months, with an 82.3% (95% CI, 75.9-87.1) probability of response 12 months later. Median duration of response was not estimable over a median 13.9-month follow-up period. The most common adverse events (≥5.0% of patients) were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention; generally mild/moderate and resolved/resolving. Serious adverse events were observed in 29/240 (12.1%), 2 were treatment-related (urinary retention/urethral stenosis), both resolved. CONCLUSIONS Primary chemoablation with UGN-102 in patients with recurrent low-grade intermediate-risk nonmuscle-invasive bladder cancer resulted in a 79.6% CR rate. Patients achieving a CR had an 82.3% likelihood of remaining disease-free 1 year later. The benefit-risk profile was favorable, supporting UGN-102 as a nonsurgical alternative for transurethral resection of bladder tumors in this patient population. Limitations of this study included lack of tumor sizing after the diagnostic biopsy. TRIAL REGISTRATION ClinicalTrials.gov Identifier: NCT05243550.

中文翻译：

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使用 UGN-102 对复发性低级别中危非肌层浸润性膀胱癌进行原发性消融：一项单臂、开放标签、3 期试验 （ENVISION）。


目的 评价 UGN-102 化疗消融术对复发性低级别中危非肌层浸润性膀胱癌患者初步治疗的疗效和安全性。材料和方法 ENVISION 是一项正在进行的、多国的、单臂的 3 期研究，研究对象是未经治疗的低级别中危非肌层浸润性膀胱癌活检证实复发的患者。患者每周接受 6 次 UGN-102 膀胱内滴注 （丝裂霉素;门诊情况），并在 3 个月时进行评估。达到完全缓解 （CR;膀胱镜检查、细胞学和因果活检阴性） 的患者定期监测，直至复发、进展或死亡。保持无病的患者将接受长达 5 年的随访，并将在未来报告进一步的结果。结果 在入组的 240 名患者中，228 名 （95%） 接受了所有 6 次计划剂量;191 例 （79.6%;95% CI，73.9-84.5） 在 3 个月时达到 CR，12 个月后的反应概率为 82.3% （95% CI，75.9-87.1）。在中位 13.9 个月的随访期内，中位缓解持续时间无法估计。最常见的不良事件 （≥5.0% 的患者） 是排尿困难、血尿、尿路感染、尿路尿症、疲劳和尿潴留;通常为轻度/中度和缓解/缓解。29/240 （12.1%） 观察到严重不良事件，2 例与治疗相关 （尿潴留/尿道狭窄），均已解决。结论 在复发性低级别中危非肌层浸润性膀胱癌患者中，使用 UGN-102 进行初次化疗消融的 CR 率为 79.6%。达到 CR 的患者在 1 年后保持无病的可能性为 82.3%。 获益-风险状况良好，支持 UGN-102 作为该患者群体经尿道膀胱肿瘤切除术的非手术替代方案。这项研究的局限性包括诊断性活检后缺乏肿瘤大小。TRIAL REGISTRATION ClinicalTrials.gov 标识符：NCT05243550。