Purpose of review: Chronic graft-versus-host disease is an important cause of late morbidity and mortality after allogeneic stem cell transplantation. With the renewed interest in its pathophysiology and treatment, this review discusses recent clinical and laboratory advances in this disease. Advances in pathophysiology, the relationship between chronic graft-versus-host disease and relapse incidence, and recent developments in the prophylaxis, initial therapy, and therapy for refractory disease are discussed.
Recent findings: A better understanding of the pathophysiology of chronic graft-versus-host disease, including the potential role of a coordinated B-cell and T-cell response, is demonstrated. Corticosteroids and cyclosporine or tacrolimus remain the standard as initial therapy. This combination is effective in the majority of affected patients, although therapy is often required for longer than 1 year. Although no strategy has been demonstrated to be effective in specifically preventing chronic graft-versus-host disease, several drugs have recently been demonstrated to be effective therapeutic agents for steroid-refractory disease. Agents such as mycophenolate mofetil, sirolimus, and rituximab have demonstrated response rates of greater than 60% in patients with steroid-refractory disease.
Summary: Renewed interest and understanding of chronic graft-versus-host disease have led to novel treatment strategies for steroid-refractory disease. A focus on the initial therapy and prophylaxis against chronic graft-versus-host disease is now warranted.