Graft-versus-Host Disease: The State of the Science.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, Volume 19, Issue S1, p.S102-S08 (2013)


2013, Center-Authored Paper, Clinical Research Division, November 2012


Graft-versus-Host Disease (GVHD) is the major limitation of allogeneic hematopoietic cellular transplantation. Despite concerted research efforts, GVHD prevention and treatment strategies have not substantially changed over the past three decades. This session will review the state the science of GVHD with respect to GVHD prophylaxis, acute GVHD therapy, and the assessment of chronic GVHD. A review of the latest novel GVHD prophylaxis strategies will be presented along with how these will be incorporated into a multi-center phase II trial in the upcoming year. Exploring ways to improve outcomes for patients with newly diagnosed acute GVHD over that which is achieved by steroids-alone has been a major research focus. The largest of these efforts has been the serial randomized phase II and phase III trials conducted through the Blood and Marrow Transplant Clinical Trials Network: Trials 0302 and 0802. These two trials will be extensively reviewed including the latest results from 0802. Lastly, one of the greatest challenges with respect to chronic GVHD has been formulating guidelines to capture disease severity and response. Clinical trials assessing chronic GVHD treatments are un-interpretable without these standards, which had been lacking prior to the NIH Consensus papers. However, the NIH severity index and response assessments were based on expert opinion and to date have not been prospectively validated. Resultsfrom the Chronic GVHD consortium evaluating the NIH disease severity and response assessment tools will be presented along with an overview of how this knowledge can be used for future clinical trials.