Department of Health Policy and Management, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina.
J Urol. 2022 Sep;208(3):580-588. doi: 10.1097/JU.0000000000002747. Epub 2022 May 31.
Optima II ("OPTimized Instillation of Mitomycin for Bladder Cancer Treatment," clinicaltrials.gov: NCT03558503) was a phase 2b trial evaluating a nonsurgical alternative as a primary treatment for nonmuscle-invasive bladder cancer (NMIBC). Patients received 6 weekly instillations of UGN-102, a mitomycin-containing reverse thermal gel. This is the first study to report on patient-reported side effects of UGN-102.
Sixty-three patients enrolled in Optima II from 20 sites. Of these 63 patients, 44 were in the cohort completing a quarterly patient-reported outcome measure assessing side effects. Changes in side effects were evaluated using the Wilcoxon signed-rank test. Associations of 3-month outcomes with demographic and clinical characteristics were examined with regression, controlling for baseline values. Ten of 44 patients (23%) were interviewed after the trial to understand tolerability for future patients making treatment decisions. Transcripts were double-coded using standard methods.
In the patient-reported outcome measure cohort (44), 61% were men, 57% aged 65+ years and 89% were non-Hispanic White. UGN-102 did not cause decrements in patient-reported urinary symptoms, bloating/flatulence or malaise at the primary endpoint of 3 months. Sexual function mildly worsened. Future health worries improved. Demographics were not correlated with changes. Clinically, sexual function was correlated with new NMIBC and bloating/flatulence was associated with transurethral resection of bladder tumor within 12 months. In interviews, patients appreciated a nonsurgical alternative, would recommend the gel to other patients and would choose the gel over surgery.
A nonsurgical, chemoablative gel (UGN-102) used as a primary treatment for NMIBC offers a more patient-centered therapeutic approach than standard treatments.
Optima II（“膀胱癌治疗中丝裂霉素优化给药的优化”，clinicaltrials.gov：NCT03558503）是一项评估非手术替代方案作为非肌肉浸润性膀胱癌（NMIBC）主要治疗方法的 2b 期试验。患者接受了 6 次每周一次的 UGN-102 膀胱内灌注，UGN-102 是一种含有丝裂霉素的逆向热凝胶。这是第一项报告 UGN-102 患者报告副作用的研究。
63 名患者从 20 个地点参加了 Optima II。在这 63 名患者中，有 44 名患者在队列中完成了一项每季度一次的患者报告结局测量，以评估副作用。使用 Wilcoxon 符号秩检验评估副作用的变化。使用回归分析，控制基线值，检查 3 个月结局与人口统计学和临床特征的相关性。10 名患者（23%）在试验后接受了访谈，以了解未来患者做出治疗决策时的耐受性。使用标准方法对转录本进行双编码。
在患者报告结局测量队列（44 名）中，61%为男性，57%年龄在 65 岁以上，89%为非西班牙裔白人。在 3 个月的主要终点时，UGN-102 并未导致患者报告的尿症状、腹胀/气胀或不适恶化。性功能略有恶化。对未来健康的担忧有所改善。人口统计学与变化无关。临床上，性功能与新的 NMIBC 相关，腹胀/气胀与 12 个月内膀胱肿瘤经尿道切除术相关。在访谈中，患者赞赏非手术替代方案，会向其他患者推荐该凝胶，并且会选择该凝胶而不是手术。
作为 NMIBC 的主要治疗方法，非手术、化学消融凝胶（UGN-102）提供了一种比标准治疗更以患者为中心的治疗方法。
