Shore Neal D, Powles Thomas B, Bedke Jens, Galsky Matthew D, Palou Redorta Joan, Ku Ja Hyeon, Kretkowski Michal, Xylinas Evanguelos, Alekseev Boris, Ye Dingwei, Guerrero-Ramos Félix, Briganti Alberto, Kulkarni Girish S, Brinkmann Julia, Calella Anna-Maria, Cesari Rossano, Eccleston Anthony, Michelon Elisabete, Vermette Jennifer, Wei Caimiao, Steinberg Gary D
START Carolinas/Carolina Urologic Research Center, Myrtle Beach, SC, USA.
Barts Health Biomedical NIHR Research Centre, Queen Mary University of London, London, UK.
Nat Med. 2025 May 31. doi: 10.1038/s41591-025-03738-z.
Bacillus Calmette-Guérin (BCG) induction and maintenance (I+M) after transurethral resection of bladder tumor is standard of care (SOC) in high-risk non-muscle invasive bladder cancer (NMIBC). However, disease recurrence/progression occurs in approximately 40% of patients at 2 years, with unfavorable prognosis. Limited bladder-sparing therapeutic options exist, and no improvements to response durability have been observed in decades. CREST is a global, phase 3, randomized trial evaluating subcutaneous sasanlimab in combination with BCG-I+M (Arm A), sasanlimab in combination with BCG-I (Arm B) or BCG-I+M (Arm C) in BCG-naive high-risk NMIBC. The primary endpoint was investigator-assessed event-free survival (EFS) for Arm A versus Arm C; key secondary endpoints were EFS (Arm B versus Arm C) and overall survival. Patients were randomized 1:1:1 to Arm A (N = 352), Arm B (N = 352) and Arm C (N = 351). The trial met its primary endpoint with a statistically significant and clinically meaningful prolongation of EFS (Arm A versus Arm C); hazard ratio, 0.68 (95% confidence interval: 0.49-0.94); one-sided P = 0.0095. The 36-month estimated EFS rates were 82.1% (Arm A) and 74.8% (Arm C). EFS benefit for Arm A versus Arm C was observed across prespecified subgroups, including carcinoma in situ (CIS) and T1. The safety profile of the combination was consistent with the known profiles. To our knowledge, sasanlimab is the first anti-PD-1 antibody to show a clinically meaningful prolongation of EFS when combined with BCG-I+M versus SOC in patients with BCG-naive high-risk NMIBC. Sasanlimab combined with BCG-I+M has the potential to redefine the treatment paradigm and clinical decision-making for patients with BCG-naive high-risk NMIBC. ClinicalTrials.gov identifier: NCT04165317 .
卡介苗(BCG)诱导和维持治疗(I+M)用于经尿道膀胱肿瘤切除术后的高危非肌层浸润性膀胱癌(NMIBC)是标准治疗方案(SOC)。然而,约40%的患者在2年时会出现疾病复发/进展,预后不佳。膀胱保留治疗选择有限,且数十年来未观察到反应持久性有改善。CREST是一项全球性3期随机试验,评估皮下注射sasanlimab联合BCG-I+M(A组)、sasanlimab联合BCG-I(B组)或BCG-I+M(C组)用于初治高危NMIBC患者的疗效。主要终点是研究者评估的A组与C组的无事件生存期(EFS);关键次要终点是EFS(B组与C组)和总生存期。患者按1:1:1随机分为A组(N = 352)、B组(N = 352)和C组(N = 351)。该试验达到了其主要终点,EFS有统计学显著且临床有意义的延长(A组与C组);风险比为0.68(95%置信区间:0.49 - 0.94);单侧P = 0.0095。36个月时的估计EFS率分别为82.1%(A组)和74.8%(C组)。在包括原位癌(CIS)和T1期在内的预先指定亚组中均观察到A组相对于C组的EFS获益。联合治疗的安全性与已知情况一致。据我们所知,sasanlimab是首个在初治高危NMIBC患者中与BCG-I+M联合使用相对于SOC能使EFS有临床意义延长的抗PD-1抗体。Sasanlimab联合BCG-I+M有可能重新定义初治高危NMIBC患者的治疗模式和临床决策。ClinicalTrials.gov标识符:NCT04165317 。