Hematologic Malignancies and Cellular Therapy, Department of Medicine, Duke University Medical Center, 2301 Erwin Dr, Durham, NC, 27705, USA.
Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Curr Hematol Malig Rep. 2020 Aug;15(4):254-260. doi: 10.1007/s11899-020-00584-3.
This review discusses important findings from real-world evidence (RWE) studies impacting the field of chronic lymphocytic leukemia (CLL), from prognostication to long-term management and sequencing of novel agents.
RWE data, which represents a heterogeneous cohort of patients treated at non-academic centers and off-clinical trial, demonstrate several key differences between findings in randomized controlled trials (RCT) of novel agents in CLL. While toxicity and discontinuation rates are higher, survival outcomes are encouraging in clinical practice, with often equivalent outcomes from reduced-dose or time-limited therapy of drug. Though sequencing of novel agents lacks robust RCT data, RWE data provide important guidance for selection and sequencing of novel agents. RWE has critically informed our practice by providing diverse patient data and real-time analysis for important clinical questions outpacing the rapidly growing field of CLL.
本综述讨论了真实世界证据（RWE）研究对慢性淋巴细胞白血病（CLL）领域的重要发现，从预后到新型药物的长期管理和序贯治疗。
RWE 数据代表了在非学术中心和临床试验之外治疗的异质性患者队列，这些数据显示了 CLL 新型药物的随机对照试验（RCT）中发现的几个关键差异。虽然新型药物的毒性和停药率较高，但临床实践中的生存结果令人鼓舞，药物的低剂量或限时治疗通常也能取得等效的结果。尽管新型药物的序贯治疗缺乏强有力的 RCT 数据，但 RWE 数据为新型药物的选择和序贯治疗提供了重要指导。RWE 通过提供多样化的患者数据和实时分析，为重要的临床问题提供了关键信息，这些问题的发展速度超过了 CLL 领域的快速发展。
