Kawahara Takashi, Kandori Shuya, Kojima Takahiro, Mathis Bryan J, Shiga Masanobu, Nishiyama Hiroyuki
Department of Urology, Faculty of Medicine, University of Tsukuba, 1-1-1 Tennodai, Tsukuba, Ibaraki, 305-8575, Japan.
Department of Urology, Aichi Cancer Center, Nagoya, Japan.
BMC Res Notes. 2025 Jan 31;18(1):47. doi: 10.1186/s13104-025-07128-z.
Recent preclinical and retrospective clinical evidence shows that androgen receptor (AR)-mediated signals have significant roles in development of non-muscle invasive bladder cancer (NMIBC). Here, we conducted a single-center, phase I study to assess the feasibility and efficacy of enzalutamide in patients having recurrent NMIBC with marker tumors. Patients with NMIBC who cannot achieve complete transurethral resection (TUR) or with recurrence within a year after the TUR, were enrolled. The patients were administered oral enzalutamide at 160 mg dose, once daily for four weeks. Clinical response at the end of the treatment was evaluated using cystoscopy.
Of the six patients enrolled, two experienced multiple recurrences. All the patients received the planned administration of enzalutamide. Enzalutamide was tolerable and all patients were able to complete the planed treatment, although four patients experienced mild treatment-related adverse events (AEs), but AEs with grade 2 or more were not observed. As for efficacy, three patients showed no change while the remaining three showed disease progression. Immunohistochemical analysis did not showed the strong staining of AR in the latest tumors. This is the first clinical study on enzalutamide treatment for NMIBC patients. In this study, four weeks of enzalutamide administration was well tolerated, however showed no clinical response for non-strong staining of AR.
University Hospital Medical Information Network UMIN000026520 (date registration: 2017/3/13).
近期的临床前和回顾性临床证据表明，雄激素受体（AR）介导的信号在非肌层浸润性膀胱癌（NMIBC）的发展中具有重要作用。在此，我们进行了一项单中心I期研究，以评估恩杂鲁胺对复发性NMIBC伴标记肿瘤患者的可行性和疗效。纳入了无法实现完全经尿道切除术（TUR）或TUR术后一年内复发的NMIBC患者。患者口服恩杂鲁胺，剂量为160毫克，每日一次，共四周。治疗结束时使用膀胱镜检查评估临床反应。
在纳入的6名患者中，2名经历了多次复发。所有患者均接受了计划的恩杂鲁胺给药。恩杂鲁胺耐受性良好，所有患者均能够完成计划的治疗，尽管有4名患者经历了轻度的治疗相关不良事件（AE），但未观察到2级或更高级别的AE。至于疗效，3名患者无变化，其余3名显示疾病进展。免疫组织化学分析未显示最新肿瘤中AR的强染色。这是关于恩杂鲁胺治疗NMIBC患者的第一项临床研究。在本研究中，四周的恩杂鲁胺给药耐受性良好，但对AR染色不强的患者未显示出临床反应。
大学医院医学信息网络UMIN000026520（注册日期：2017/3/13）。
