Department of Medicine, Baylor College of Medicine, Houston, TX, USA.
Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital, Texas Children's Hospital, Houston, TX, USA.
Hum Vaccin Immunother. 2024 Dec 31;20(1):2378543. doi: 10.1080/21645515.2024.2378543. Epub 2024 Aug 5.
CAR-T cell therapy has established itself as a highly effective treatment for hematological malignancies. There are currently six commercial CAR-T products that have been FDA approved for diseases such as B-ALL, LBCL, MCL, FL, MM, and CLL/SLL. "Real-world" studies allow us to evaluate outcomes from the general population to determine their efficacy and safety compared to those who were included in the original trials. Based on several well conducted "Real-world" studies that represent diverse populations, we report that outcomes from the original trials that led to the approval of these therapies are comparable to those in practice.
嵌合抗原受体 T 细胞（CAR-T）疗法已被确立为血液系统恶性肿瘤的一种非常有效的治疗方法。目前已有六种 CAR-T 产品获得美国食品药品监督管理局（FDA）批准，可用于治疗 B-ALL、LBCL、MCL、FL、MM 和 CLL/SLL 等疾病。“真实世界”研究使我们能够评估一般人群的结果，以确定与原始试验入组患者相比，其疗效和安全性。基于几项针对不同人群的精心设计的“真实世界”研究，我们报告称，这些疗法获批所依据的原始试验结果与实际应用中的结果相当。
