End points and outcomes in follicular lymphoma: what should we measure, how, and why?

Journal: Blood

This publication reviews the challenges and considerations in selecting appropriate clinical end points for follicular lymphoma (FL) trials.

Given the long median overall survival (~20 years) in FL, traditional overall survival is often impractical as a primary end point.

The authors discuss the limitations of currently used end points, including delayed results and insufficient sensitivity to detect therapeutic differences.

They advocate for:

  • Validated surrogate markers, such as complete remission at 30 months post-frontline immunochemotherapy, as potential primary or co-primary end points in randomized trials.
  • Emerging markers like minimal residual disease, which may be valuable in early-phase studies and for accelerated approval processes.

Additionally, the review emphasizes incorporating quality of life and nonmedical burdens (e.g., time, financial toxicity) as critical secondary end points to ensure patient-centered care and better evaluate treatment impact beyond survival.

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