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

J Exp Pharmacol. 2020 Nov 26;12:549-557. doi: 10.2147/JEP.S259290. eCollection 2020.


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.

Keywords: graft-vs-host disease; hematopoietic; ruxolitinib; stem cell transplantation; steroid refractory.

Publication types

  • Review