Jagannath Sundar, Martin Thomas G, Lin Yi, Cohen Adam D, Raje Noopur, Htut Myo, Deol Abhinav, Agha Mounzer, Berdeja Jesus G, Lesokhin Alexander M, Liegel Jessica J, Rossi Adriana, Lieberman-Cribbin Alex, Usmani Saad Z, Dhakal Binod, Parekh Samir, Li Hui, Wang Feng, Montes de Oca Rocio, Plaks Vicki, Sun Huabin, Banerjee Arnob, Schecter Jordan M, Lendvai Nikoletta, Madduri Deepu, Lengil Tamar, Zhu Jieqing, Koneru Mythili, Akram Muhammad, Patel Nitin, Costa Filho Octavio, Jakubowiak Andrzej J, Voorhees Peter M
Icahn School of Medicine at Mount Sinai, New York, NY.
University of California, San Francisco, San Francisco, CA.
J Clin Oncol. 2025 Sep;43(25):2766-2771. doi: 10.1200/JCO-25-00760. Epub 2025 Jun 3.
CARTITUDE-1 evaluated ciltacabtagene autoleucel (cilta-cel) in patients with heavily pretreated relapsed/refractory multiple myeloma (RRMM). We describe overall survival (OS), ≥5-year progression-free outcomes, associated biomarkers, and safety, with a median study follow-up of 61.3 months. For the 97 treated patients, median OS was 60.7 months (95% CI, 41.9 to not estimable). One third (32/97) of patients remain alive and progression-free for ≥5 years after a single cilta-cel infusion, without maintenance treatment. Twelve of these patients treated at a single center underwent serial minimal residual disease (MRD) and positron emission tomography-computed tomography assessments, and all (100%) were MRD-negative (at least 10 threshold) and imaging-negative at year 5 or later after cilta-cel. Baseline characteristics, including the presence of high-risk cytogenetics and extramedullary disease, were generally comparable for the 32 patients who were progression-free for ≥5 years versus patients who had progressive disease by year 5. A trend of lower baseline tumor burden, higher fraction of naïve T-cells in the cilta-cel drug product, higher T cell-to-neutrophil ratio, higher hemoglobin and platelets at baseline, and higher effector-to-target ratio were associated with ≥5-year progression-free status. The safety profile of cilta-cel remained consistent with previous reports. To our knowledge, our data provide the first evidence that cilta-cel is potentially curative in patients with RRMM.
CARTITUDE-1研究评估了西达基奥仑赛（cilta-cel）在经过大量预处理的复发/难治性多发性骨髓瘤（RRMM）患者中的疗效。我们描述了总生存期（OS）、≥5年的无进展结局、相关生物标志物和安全性，研究的中位随访时间为61.3个月。对于97例接受治疗的患者，中位OS为60.7个月（95%CI，41.9至无法估计）。三分之一（32/97）的患者在单次输注cilta-cel后，无需维持治疗，存活且无进展≥5年。其中在单一中心接受治疗的12例患者接受了系列微小残留病（MRD）和正电子发射断层扫描-计算机断层扫描评估，所有患者（100%）在cilta-cel治疗后5年或更晚时MRD阴性（至少10阈值）且影像学阴性。≥5年无进展的32例患者与5年时疾病进展的患者相比，包括高危细胞遗传学和髓外疾病的存在情况在内的基线特征总体相当。基线肿瘤负荷较低、cilta-cel药品中幼稚T细胞比例较高、T细胞与中性粒细胞比例较高、基线时血红蛋白和血小板较高以及效应细胞与靶细胞比例较高的趋势与≥5年无进展状态相关。cilta-cel的安全性概况与既往报告一致。据我们所知，我们的数据首次证明cilta-cel对RRMM患者可能具有治愈作用。
