What clinicians should know about surrogate end points in hematologic malignancies

Author:

Bommier Côme12,Maurer Matthew John13ORCID,Lambert Jerome2

Affiliation:

1. 1Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN

2. 2Epidemiology and Clinical Statistics for Tumor, Respiratory, and Resuscitation Assessments Team, INSERM, U1153, Assistance Publique-Hôpitaux de Paris Hôpital St Louis, Université Paris Cité, Paris, France

3. 3Division of Hematology, Mayo Clinic, Rochester, MN

Abstract

Abstract Use of surrogates as primary end points is commonplace in hematology/oncology clinical trials. As opposed to prognostic markers, surrogates are end points that can be measured early and yet can still capture the full effect of treatment, because it would be captured by the true outcome (eg, overall survival). We discuss the level of evidence of the most commonly used end points in hematology and share recommendations on how to apply and evaluate surrogate end points in research and clinical practice. Based on the statistical literature, this clinician-friendly review intends to build a bridge between clinicians and surrogacy specialists.

Publisher

American Society of Hematology

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