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Real-World Evidence for Chronic Lymphocytic Leukemia in the Era of Targeted Therapies

Islam, Prioty MD*; Mato, Anthony R. MD

doi: 10.1097/PPO.0000000000000407
Review Articles

The landscape of chronic lymphocytic leukemia has transformed in the era of small molecule inhibitor targeted therapies. While randomized controlled trials remain the criterion standard in evaluating new therapies, they are often unable to keep pace with the clinical questions that arise during the use of novel agents. Real-world evidence is generated through analysis of data such as electronic medical records, payer claims, and patient registry databases and can provide invaluable information to supplement randomized controlled trials, such as outcomes in patient populations excluded from clinical trials, rates of discontinuation or dose reductions in clinical practice, survival outcomes, and optimal sequencing of novel agents. This review aims to discuss major findings from recent, relevant, real-world evidence publications that have greatly informed our understanding of chronic lymphocytic leukemia as it is treated in clinical practice.

From the *Hematologic Malignancies and Cellular Therapy, Department of Medicine, Duke University Medical Center, Durham, NC

Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.

Reprints: Prioty Islam, MD, Duke University Medical Center, 2301 Erwin Dr, Durham, NC 27705. E-mail:

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