National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2020 Treatment of Chronic GVHD Report.

Journal Article (Journal Article)

Positive results from recent clinical trials have significantly expanded current therapeutic options for patients with chronic graft-versus-host disease (GVHD). However, new insights into the associations between clinical characteristics of chronic GVHD, pathophysiologic mechanisms of disease, and the clinical and biological effects of novel therapeutic agents are required to allow for a more individualized approach to treatment. The current report is focused on setting research priorities and direction in the treatment of chronic GVHD. Detailed correlative scientific studies should be conducted in the context of clinical trials to evaluate associations between clinical outcomes and the biological effect of systemic therapeutics. For patients who require systemic therapy but not urgent initiation of glucocorticoids, clinical trials for initial systemic treatment of chronic GVHD should investigate novel agents as monotherapy without concurrently starting glucocorticoids, to avoid confounding biological, pathological, and clinical assessments. Clinical trials for treatment-refractory disease should specifically target patients with incomplete or suboptimal responses to most recent therapy who are early in their disease course. Close collaboration between academic medical centers, medical societies, and industry is needed to support an individualized, biology-based strategic approach to chronic GVHD therapy.

Full Text

Duke Authors

Cited Authors

  • DeFilipp, Z; Couriel, DR; Lazaryan, A; Bhatt, VR; Buxbaum, NP; Alousi, AM; Olivieri, A; Pulanic, D; Halter, JP; Henderson, LA; Zeiser, R; Gooley, TA; MacDonald, KPA; Wolff, D; Schultz, KR; Paczesny, S; Inamoto, Y; Cutler, CS; Kitko, CL; Pidala, JA; Lee, SJ; Socie, G; Sarantopoulos, S; Pavletic, SZ; Martin, PJ; Blazar, BR; Greinix, HT

Published Date

  • September 2021

Published In

Volume / Issue

  • 27 / 9

Start / End Page

  • 729 - 737

PubMed ID

  • 34147469

Pubmed Central ID

  • PMC8944207

Electronic International Standard Serial Number (EISSN)

  • 2666-6367

Digital Object Identifier (DOI)

  • 10.1016/j.jtct.2021.05.004


  • eng

Conference Location

  • United States