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Five Future Possibilities for Treating Acute GVHD That Is Refractory Steroids

June 5, 2025

By Peter Wehrwein, Managing Editor

News

Article

Researchers at MD Anderson discussed the five possible approaches to treating graft-versus-host disease in a review article published in the American Journal of Hematology.

Portia Smallbone, M.B.B.S.

The FDA approved Jakafi (ruxolitinib) in 2019 as a treatment for graft-versus-host disease (GVHD) that doesn’t respond to steroids. The addition of the oral Janus kinase 1/2 inhibitor to the GVHD armamentarium has been a major advance, but some patients don’t respond to Jakafi. Moreover, in a review paper published in the American Journal of Hematology, lead and corresponding author Portia Smallbone, M.B.B.S., of the University of Texas MD Anderson Cancer Center, and her MD Anderson colleagues, Rohtesh S. Mehta, M.P.H., M.S., and Amin Alousi, M.D., say the benefits of Jakafi have not translated into improved overall survival. There is, they wrote, “a clear and urgent need for better therapies in patients suffering from SR-AGVHD [steroid-refractory graft-versus-host-disease].” In the latter half of the review, they discuss five experimental “third line” approaches to treating acute GVHD.

Mesenchymal stem cells

Mesenchymal stem cells are stem cells that can be obtained from bone marrow, adipose tissue, or umbilical cord, explain Smallbone and colleagues. They can be infused intravenously and have been shown to have immunological properties that suggest they could be used as treatment of steroid-refractory GVHD. But Smallbone, Mehta and Alousi say the trial data concerning efficacy has been “contentious.” Case reports and European studies showed remarkable results that couldn’t be reproduced. They cite a Cochrane review of 12 randomized studies conducted before 2018 that, they say, found no evidence of efficacy. But they also cite a real-world evidence study of 64 patients that they characterize as having “encouraging” results with respect to overall survival. The inconsistency, they say, could be chalked up to differences in study design, manufacturing differences and variation in how respond is defined.

The FDA approved Ryoncil (remestemcel-L) in December 2024 for steroid-refractory acute GVHD graft in pediatric patients. Smallbone and her colleagues said that approval was “a significant milestone and may pave the way for more robust studies in the adult population.”