Current and Emerging Targeted Therapies for Acute Graft-Versus-Host Disease

Acute graft-versus-host disease (GVHD), the major complication after allogeneic hematopoietic cell transplant (HCT), develops in approximately 50% of patients. The primary treatment is high-dose systemic steroids, but treatment failure is common, and steroid-refractory (SR) GVHD is the leading cause of non-relapse mortality after allogeneic HCT. Ruxolitinib became the first treatment for SR GVHD to obtain US Food and Drug Administration approval, and other new treatments are actively being studied. We searched the literature using the PubMed database and clinical trials using ClinicalTrials.gov to identify the most promising new treatments for GVHD. In this review, we categorize potential new treatments for GVHD by their mechanism of action (e.g., antibodies that deplete T cells or prevent their trafficking to target tissues, proteasome inhibitors, tyrosine kinase inhibitors, and other agents) and summarize the results from clinical trials.

Automated summary

GVHD, a common complication of HCT, is often treated with high-dose steroids, but treatment failure and steroid-refractory cases are common. Ruxolitinib is the first FDA-approved treatment for SR GVHD, with other new treatments currently under study.