Utilizing Real-World Evidence (RWE) to Improve Care in Chronic Lymphocytic Leukemia: Challenges and Opportunities

Abstract

Purpose of Review

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.

Recent Findings

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.

Summary

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.

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Correspondence to Prioty Islam.

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P.I. declares no conflicts of interest.

A.R.M. holds a consultancy role for TG Therapeutics (in addition DSMB), Abbvie, Pharmacyclics, Johnson & Johnson, Regeneron, Astra Zeneca, Genentech, LOXO, Celgene (including DSMB), Sunesis, and Adaptive and has received research funding from TG Therapeutics, Abbvie, Pharmacyclics, Johnson & Johnson, Regeneron, Genentech, LOXO, Portola, DTRM, Adaptive, and Acerta.

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Islam, P., Mato, A.R. Utilizing Real-World Evidence (RWE) to Improve Care in Chronic Lymphocytic Leukemia: Challenges and Opportunities. Curr Hematol Malig Rep 15, 254–260 (2020). https://doi.org/10.1007/s11899-020-00584-3

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Keywords

  • Real-world evidence
  • Chronic lymphocytic leukemia
  • Small-molecule inhibitor
  • Bruton’s tyrosine kinase inhibitor
  • PI3K inhibitor
  • BCL-2 inhibitor
  • Novel agents