Patient-Reported Outcomes in Clinical Trials: Lessons from the Blood and Marrow Transplant Clinical Trials Network

The Blood and Marrow Transplant Clinical Trials Network performs multi-center Phase 2 and Phase 3 clinical trials and has incorporated patient reported outcome (PRO) measures into study protocols since its inception. As with clinical data, collection and analysis of this type of information requires rigor to allow valid conclusions. This paper summarizes our experience with PROs over the last 25 years and reports on the quality of PRO reporting as well as lessons learned in this academic network setting.

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Publication Details

Journal
Blood Advances
Published
2026-08-25
DOI
https://doi.org/10.1182/bloodadvances.2026021114
Primary Topic
Cancer survivorship and care
Type
article
Field-Weighted Citation Impact
0.00
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article

Patient-Reported Outcomes in Clinical Trials: Lessons from the Blood and Marrow Transplant Clinical Trials Network

Rachel Cusatis, Catherine J. Lee, Stephanie J. Lee, Deborah Mattila et al.
Blood Advances
Cancer survivorship and care
article

Patient-Reported Outcomes in Clinical Trials: Lessons from the Blood and Marrow Transplant Clinical Trials Network

Rachel Cusatis, Catherine J. Lee, Stephanie J. Lee, Deborah Mattila, Betty K. Hamilton, Ruta Brazauskas, Areej El-Jawahri, Rachel Phelan, Anthony D. Sung, Mary M Horowitz
article en

Abstract

The Blood and Marrow Transplant Clinical Trials Network performs multi-center Phase 2 and Phase 3 clinical trials and has incorporated patient reported outcome (PRO) measures into study protocols since its inception. As with clinical data, collection and analysis of this type of information requires rigor to allow valid conclusions. This paper summarizes our experience with PROs over the last 25 years and reports on the quality of PRO reporting as well as lessons learned in this academic network setting.

Blood Advances
Cleveland Clinic (US), National Marrow Donor Program (US), Medical College of Wisconsin (US), Massachusetts General Hospital (US), Fred Hutch Cancer Center (US), Versiti Blood Center of Wisconsin (US), University of Kansas Medical Center (US)
Openalex Percentile: Top 13%
Cancer survivorship and care
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