Despite U.S. Food and Drug Administration (FDA) approval of new therapies for refractory acute graft-versus-host disease (GVHD), such as ruxolitinib, in recent years, finding an effective treatment remains an unmet need in the field. On Feb. 12, three experts will discuss the underlying mechanisms of the disease that may guide the design of future clinical trials or illuminate new pathways with significant translational potential for GVHD. Concurrent: Refractory Acute GVHD begins at 10:30 a.m. in Ballroom C.

“GVHD continues to be a significant clinical challenge. It still has a complete response rate ranging from 30-60%, and GVHD involving the gastrointestinal (GI) tract has the poorest outcomes, even to second-line medicine,” said Session Chair Victor Tkachev, PhD. “Finding an effective treatment for this disease without making patients more susceptible to viral disease, opportunistic infections, and cancer relapse won’t be possible without advancing our mechanistic understanding of how steroid-refractory GVHD evolves.”

Tkachev, an assistant professor of surgery at Harvard Medical School and an assistant investigator at the Center for Transplantation Sciences at Massachusetts General Hospital, will explore how to maintain a healthy immune system without flaring GVHD based on lessons from a non-human primate model. Viktor Arnhold, MD, Memorial Sloan Kettering Cancer Center, will discuss the role of corticosteroids on the dysregulation of epithelial regeneration in the GI tract.

“The GI tract is highly susceptible to GVHD, and we know from clinical practice that it’s the hardest to treat,” Tkachev noted. “There is evidence from many clinical trials that the development of GI GVHD predicts the outcome of transplantation.”

While Tkachev and Arnhold will present information most applicable to basic and translational researchers, Kristina Maas-Bauer, MD, a hematologist/oncologist and physician-scientist at Medical Center Freiburg University, Freiburg, Germany, will delve into clinical considerations, outlining Rho kinase (ROCK) type 1/2 inhibition for steroid-refractory acute GVHD.

“This ROCK inhibitor has recently been approved for chronic GVHD, and it seems like this drug has some beneficial effect in acute GVHD, which can be explored further,” Tkachev said. “As it already exists as an FDA-approved drug, it has a more straightforward path to the clinic in acute disease because we know its safety profile and it has been studied in different settings.”

This and other sessions at the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR® will be available for on-demand viewing for registered attendees following the live presentation.