Experimental Pharmaceuticals for Steroid-Refractory Acute Graft-versus-Host Disease

Acute GVHD (aGVHD) is a significant complication after allogeneic hematopoietic cell transplantation (HCT), occurring in up to 70% of HCT recipients. Steroid-refractory aGVHD represents a subset of patients failing initial therapy and is particularly morbid, with only 30% of patients surviving long term. Better therapies are urgently required for these patients. Here, we discuss recent advancements in the management of SR-aGVHD. We review the currently available therapies for SR-aGVHD including the results of the REACH1 and REACH2 trials, which provide the basis for the use of ruxolitinib for the treatment of SR-aGVHD. We additionally discuss newer agents under clinical investigation and will highlight the niche these agents may fill to further improve outcomes in aGVHD patient care.