Guerrero-Ramos Félix, Boormans Joost L, Daneshmand Siamak, Gontero Paolo, Kamat Ashish M, Rouprêt Morgan, Vilaseca Antoni, Shariat Shahrokh F
Department of Urology, Hospital Universitario 12 de Octubre, Madrid, Spain.
Department of Urology, Erasmus University Medical Center, Rotterdam, The Netherlands.
Eur Urol Oncol. 2024 Dec;7(6):1267-1279. doi: 10.1016/j.euo.2024.05.012. Epub 2024 Jun 6.
Therapeutic options for patients with non-muscle-invasive bladder cancer (NMIBC) have traditionally been limited to intravesical immunotherapy or chemotherapy. A considerable number of new options have been investigated in recent years. Our aim was to review the efficacy and toxicity of novel therapeutic options (results already reported or currently under investigation) for patients with NMIBC.
We assessed the efficacy of various novel therapeutic options by examining key endpoints in diverse settings, including recurrence, progression, overall survival, disease-specific survival, and complete response. We identified the principal advantages and limitations for each option. Safety was predominantly evaluated as the incidence of grade ≥3 adverse events. Our investigation focused on evidence from scientific articles and congress abstracts published in English within the past 5 yr.
To date, pembrolizumab, nadofaragene firadenovec, and the combination of BCG with N-803 have received US Food and Drug administration approval for the treatment of BCG-unresponsive carcinoma in situ of the bladder (with or without papillary tumours). Five phase 3 trials are recruiting BCG-naïve patients with high-risk NMIBC. There is increasing interest in an ablative rather than an adjuvant approach for patients with intermediate-risk NMIBC.
Novel drugs and device-assisted drug delivery systems are on the verge of changing the treatment of NMIBC. Novel intravesical options seem to have the same efficacy with fewer adverse events in comparison to systemic therapies.
We reviewed new therapy options for non-muscle-invasive bladder cancer. Two agents (pembrolizumab and nadofaragene firadenovec) have been approved to date. Ongoing trials are assessing direct delivery of drugs in solution into the bladder. This route seems to have similar efficacy and fewer side effects than intravenous immunotherapy.
非肌层浸润性膀胱癌（NMIBC）患者的治疗选择传统上局限于膀胱内免疫疗法或化疗。近年来，人们研究了大量新的治疗选择。我们的目的是综述NMIBC患者新型治疗选择（已报道结果或正在研究中的结果）的疗效和毒性。
我们通过检查不同情况下的关键终点，包括复发、进展、总生存期、疾病特异性生存期和完全缓解，来评估各种新型治疗选择的疗效。我们确定了每种治疗选择的主要优势和局限性。安全性主要评估为≥3级不良事件的发生率。我们的研究重点是过去5年内以英文发表的科学文章和大会摘要中的证据。
迄今为止，帕博利珠单抗、纳多柔比星腺病毒载体和卡介苗与N-803联合用药已获得美国食品药品监督管理局批准，用于治疗对卡介苗无反应的膀胱原位癌（伴或不伴乳头状肿瘤）。五项3期试验正在招募初治的高危NMIBC患者。对于中危NMIBC患者，采用消融而非辅助治疗方法的兴趣日益增加。
新型药物和设备辅助给药系统即将改变NMIBC的治疗方式。与全身治疗相比，新型膀胱内治疗选择似乎具有相同的疗效且不良事件更少。
我们综述了非肌层浸润性膀胱癌的新治疗选择。迄今为止，已有两种药物（帕博利珠单抗和纳多柔比星腺病毒载体）获得批准。正在进行的试验正在评估将药物溶液直接注入膀胱的方法。与静脉内免疫疗法相比，这种给药途径似乎具有相似的疗效且副作用更少。
