A Phase II/III Study of Sirolimus plus Prednisone, Sirolimus/Extracorporeal Photopheresis plus Prednisone, and Sirolimus/Calcineurin Inhibitor plus Prednisone for the Treatment of Chronic Graft-versus-Host-Disease

Graft-versus-host disease (GVHD) is a complication of stem cell and bone marrow transplantation in which cells from the donor attack tissues of the recipient. Symptoms of GVHD include rash, digestive problems, weight loss, and liver dysfunction. GVHD is usually treated with a corticosteroid (such as prednisone) and additional drugs called calcineurin inhibitors — such as cyclosporine or tacrolimus — but in some patients the condition persists despite this therapy.

In this study, investigators at multiple centers are comparing three different new approaches to treating chronic GVHD. Each center is evaluating two of the approaches. At Memorial Sloan-Kettering, physicians are comparing treatment with prednisone, sirolimus, and a calcineurin inhibitor to treatment with prednisone, sirolimus, and extracorporeal photopheresis (ECP) . Patients will be randomly assigned to receive one of these treatments (but not both).

ECP is a procedure that uses a machine to separate white blood cells from the rest of the blood, and adds methoxsalen, a photoactive chemical, to the separated white blood cells. The rest of the blood is returned to the patient. The white blood cells that were treated with methoxsalen are exposed to ultraviolet light and then returned to the patient. These treated white blood cells may stimulate the patient’s immune system and help the body control the cells that may cause GVHD.