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Graft Versus Host Disease Clinical Trials: Is it Time for Patients Centered Outcomes to Be the Primary Objective?

  • Stem Cell Transplantation (R Maziarz, Section Editor)
  • Published:
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Abstract

Purpose of Review

Graft versus host disease (GVHD) is a common complication following hematopoietic cell transplant and is associated with a high symptom burden, reduced functional status, and impaired quality of life (QOL). QOL is best assessed by patient-reported outcomes (PRO). Numerous clinical trials for the prevention and treatment of GVHD are available. This review aims to understand the landscape of PRO inclusion in clinical trials for GVHD over the last decade.

Recent Findings

Consensus bodies, including experts in GVHD, PRO, and clinical trials have made recommendations for a standardized approach for the inclusion of PRO in clinical trials including as primary outcomes, however, these have yet to be implemented in a consistent manner in practice.

Summary

Consistently applying consensus recommendation in chronic GVHD will ensure that PROs are appropriately included in clinical trials. Development of validated measures in acute GVHD and composite outcomes for all GVHD trials are required.

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Papers of particular interest, published recently, have been highlighted as: •• Of major importance

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  1. CIBMTR (Center for International Blood and Marrow Transplant Research)/Froedtert, Department of Medicine, Medical College of Wisconsin, 9200 W. Wisconsin Ave., Suite C5500, Milwaukee, WI, 53226, USA

    Bronwen E. Shaw

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Shaw, B.E. Graft Versus Host Disease Clinical Trials: Is it Time for Patients Centered Outcomes to Be the Primary Objective?. Curr Hematol Malig Rep 14, 22–30 (2019). https://doi.org/10.1007/s11899-019-0494-x

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