肿瘤细胞治疗研究
英文原题：Real-World Efficacy Outcomes of Ciltacabtagene Autoleucel in Relapsed Refractory Multiple Myeloma: A Comparative Study with the Cartitude-1 Trial.
Real-World Efficacy Outcomes of Ciltacabtagene Autoleucel in Relapsed Refractory Multiple Myeloma: A Comparative Study with the Cartitude-1 Trial.
我们的研究表明，cilta-cel 在真实世界（RW）环境下治疗的 RRMM 患者中疗效相当。
目的：CARTITUDE-1试验（C-1）促成靶向BCMA的CAR-T细胞疗法ciltacabtagene autoleucel（cilta-cel）获批，用于接受过3线治疗的复发或难治性多发性骨髓瘤（RRMM）患者。然而，试验入选标准可能无法充分代表真实世界（RW）患者。方法：研究者利用全国多中心RRMM患者队列开展回顾性分析，比较真实世界中接受cilta-cel的73例患者与C-1试验中的97例患者，评估临床特征、缓解深度、生存结局和毒性。结果：真实世界患者的基线人口学特征，包括年龄、少数族裔比例、性别、既往治疗，以及高危细胞遗传学等疾病特征，总体上与C-1人群相近。真实世界组ECOG体能状态为0分的患者较少（16%比40%，P<0.001），髓外病变患者较多（29%比13%，P=0.02），对硼替佐米（47%比68%，P=0.007）和抗CD38单克隆抗体（88%比97%，P=0.03）耐药的患者较少。真实世界患者中有45%不符合C-1入选标准。真实世界组总缓解率较低（88%比97%，P=0.015）。24个月生存结局相近，包括无进展生存率（真实世界58%比C-1组62%，P=0.98）和总生存率（72%比74%，P=0.9）。真实世界患者的安全性特征更佳，治疗相关不良事件较少。结论：本研究显示，真实世界环境中cilta-cel治疗RRMM患者具有与临床试验相当的疗效。
OBJECTIVE: The CARTITUDE-1 Trial (C-1) led to the approval of ciltacabtagene autoleucel (cilta-cel), a BCMA-directed CAR-T cell therapy, for patients with relapsed or refractory multiple myeloma (RRMM) after 3 lines of therapy. However, the inclusion criteria may not fully represent patients in the real-world (RW). METHODS: Leveraging a national multicenter cohort of patients with RRMM, we conducted a retrospective analysis comparing 73 patients who received cilta-cel in the RW to 97 patients treated in C-1 by evaluating clinical characteristics, depth of response, survival outcomes, and toxicity. RESULTS: Results showed that the RW baseline demographics such as age, proportion of racial minorities, gender, prior therapies, and disease characteristics, including high-risk cytogenetics, were broadly similar to the C-1 population. In the RW group, fewer patients had Eastern Cooperative Oncology Group 0 (16% vs. 40%, P < .001), more patients had extramedullary disease (29% vs. 13%, P = .02), and fewer were refractory to bortezomib (47% vs. 68%, P = .007) and anti-CD38 monoclonal antibody (88% vs. 97%, P = .03). Forty-five percent of RW patients did not meet the inclusion criteria for C-1. The overall response rate was lower in the RW group (88% vs. 97%, P = .015). The survival outcomes at 24-months including the progression-free survival (58% in RW vs. 62% in C1, P = .98) and overall survival (72% vs. 74%, P = .9) were similar. The safety profile of RW patients was better with fewer treatment-related adverse events. CONCLUSION: Our study demonstrates the comparable efficacy of cilta-cel in patients with RRMM treated in a RW setting.
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