Novel developments in the prophylaxis and treatment of acute GVHD

Acute graft-versus-host disease (GVHD) is a major life-threatening complication after allogeneic hematopoietic cell transplantation (allo-HCT). Traditional standard prophylaxis for acute GVHD has included a calcineurin-inhibitor plus an antimetabolite, while treatment has relied mainly on corticosteroids followed by multiple non-standard second line options. In the last decade this basic framework has been reshaped by approval of ATG products, the emergence of post-transplant cyclophosphamide and recent pivotal trials studying abatacept and vedolizumab for GVHD prophylaxis while ruxolitinib was approved for steroid refractory acute GVHD treatment. Due to this progress, routine acute GVHD prophylaxis and treatment practices are starting to shift and results of ongoing trials are eagerly awaited. Here, we review recent developments in acute GVHD prevention and therapy along with ongoing and future planned clinical trials in this space, outlining what future goals should be and the limitations of current clinical trial designs and endpoints.