Novel strategies for steroid-refractory acute graft-versus-host disease

Graft-versus-host disease is one of the commonest complications of allogeneic bone marrow or peripheral blood stem cell transplantation. This review will cover advances in the pathophysiology of graft-versus-host disease and new agents under investigation for the treatment of this disorder. Patients developing graft-versus-host disease who fail to respond to steroids have a poor prognosis. In this group of people, morbidity and mortality are very high.

Novel agents are currently under investigation for the treatment of such devastating disorders. Pentostatin, denileukin diftitox, mycophenolate mofetil, extracorporeal photopheresis, and several monoclonal antibodies have been used, some of them with encouraging results.

As supportive care improves and new agents are added to the armamentarium against steroid-refractory acute graft-versus-host disease, the prognosis of this entity may start to change. Patients with this complication after transplantation should be enrolled, whenever possible, in clinical trials to find effective therapies.