Patient-reported outcomes for acute graft-versus-host disease prevention and treatment trials

Patient-reported outcomes (PROs) such as health-related quality of life, functional status, and symptom burden have been recognized by the U.S. Food and Drug Administration (FDA) as legitimate measures of clinical benefit for sponsors seeking drug approval. However, in practice, very few agents have been approved based on these endpoints. Successful use of PROs in registration trials requires rigorous methods to overcome numerous logistic and analytic barriers. Acute graft-versus-host disease (aGVHD) is associated with high morbidity and mortality, and its prevention and treatment are the goals of many clinical trials in the hematopoietic cell transplantation (HCT) research community. This article summarizes issues to be considered in the use of PROs as endpoints in aGVHD prevention and treatment trials.