Ghodoussipour Saum, Bivalacqua Trinity, Bryan Richard T, Li Roger, Mir M Carmen, Palou Joan, Psutka Sarah P, Sundi Debasish, Tyson Mark D, Inman Brant A
Section of Urologic Oncology, Rutgers Cancer Institute and Rutgers Robert Wood Johnson Medical School, New Brunswick, NJ, USA.
Department of Urology, University of Pennsylvania, Philadelphia, PA, USA.
Eur Urol. 2025 Jul;88(1):33-55. doi: 10.1016/j.eururo.2025.02.010. Epub 2025 Apr 18.
Intravesical therapy is central to managing non-muscle-invasive bladder cancer (NMIBC); yet, recurrence and progression remain common, underscoring the need for new treatments. This systematic review evaluates clinical trials of novel intravesical therapies for all risk categories of NMIBC.
A comprehensive literature search was conducted to identify the clinical trials assessing the effectiveness, safety, and tolerability of intravesical therapies for NMIBC. The search focused on studies published from 2020 to 2024, including trials on bacillus Calmette-Guérin (BCG)-unresponsive/refractory disease as well as on BCG-naïve and intermediate-risk patients. Mechanisms of action and drug delivery methods were summarized. No statistical syntheses were performed due to limited comparative data.
Out of 2998 studies identified, 36 reported on efficacy and safety, and six provided patient-reported outcomes (PROs). Intravesical therapies included BCG-based therapies, chemotherapy combinations, chemical-drug conjugates, thermogels, hyperthermic chemotherapy, osmotic pumps, and gene therapy. Initial response rates ranged from 42% to 85% for BCG-unresponsive/refractory patients and from 65% to 100% for treatment-naïve patients. The 12-mo recurrence-free survival rates ranged from 22% to 83% and 39% to 92%, respectively. Progression and severe toxicity (grade ≥3) were rare (0-17% and 0-20%, respectively). PROs were stable. The limitations included early-phase studies, heterogeneous outcome assessments, and a need for research on long-term durability, comparative effectiveness, quality of life, and cost.
This systematic review highlights the promising efficacy and tolerability of novel intravesical therapies for NMIBC. However, further research is needed to refine treatment strategies and assess long-term outcomes, quality of life, and economic factors. Future studies should include multiarm, multistage designs with a focus on patient-centered outcomes.
膀胱内灌注治疗是管理非肌层浸润性膀胱癌（NMIBC）的核心方法；然而，复发和进展仍然很常见，这凸显了对新治疗方法的需求。本系统评价评估了针对所有风险类别的NMIBC的新型膀胱内灌注治疗的临床试验。
进行了全面的文献检索，以确定评估膀胱内灌注治疗对NMIBC的有效性、安全性和耐受性的临床试验。检索重点是2020年至2024年发表的研究，包括关于卡介苗（BCG）无反应/难治性疾病以及初治和中危患者的试验。总结了作用机制和药物递送方法。由于比较数据有限，未进行统计综合分析。
在识别出的2998项研究中，36项报告了疗效和安全性，6项提供了患者报告结局（PROs）。膀胱内灌注治疗包括基于BCG的治疗、化疗联合方案、化学药物偶联物、热凝胶、热化疗、渗透泵和基因治疗。BCG无反应/难治性患者的初始缓解率为42%至85%，初治患者为65%至100%。12个月无复发生存率分别为22%至83%和39%至92%。进展和严重毒性（≥3级）罕见（分别为0至17%和0至20%）。PROs稳定。局限性包括早期研究、异质性结局评估，以及需要对长期疗效、比较有效性、生活质量和成本进行研究。
本系统评价突出了新型膀胱内灌注治疗对NMIBC的有前景的疗效和耐受性。然而，需要进一步研究以完善治疗策略并评估长期结局、生活质量和经济因素。未来研究应包括多组、多阶段设计，重点关注以患者为中心的结局。
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