Salvage therapy for refractory chronic graft-versus-host disease with mycophenolate mofetil and tacrolimus

Chronic graft-versus-host disease (GVHD) is a common late complication of a llogeneic bone marrow transplantation (BMT) and is the principal cause of m orbidity and non-relapse mortality. The improved management of acute GVHD h as not translated into lower rates of chronic GVHD as older patients underg o allogeneic BR IT, more patients receive unrelated or related mismatched a llogeneic BMT, and donor lymphocyte infusion is increasingly used for treat ment of post-BMT relapses. Patients with high risk chronic GVHD or those wh o fail on standard therapy have a bad prognosis, Salvage therapies have pro duced disappointing results. Here, we present a retrospective analysis of 2 6 patients where a steroid sparing synergistic combination of mycophenolate mofetil (MMF) and tacrolimus was used in refractory chronic GVHD, 46% pati ents showed an objective response, 11.5 % had stable disease, 34.6 % had pr ogression and 7.7 % were not evaluable. The combination was well tolerated. This promising preliminary result has prompted a trial to assess the safet y and efficacy of this regimen in patients with chronic GVHD who have faile d prior therapy and to determine if it would improve survival as well as qu ality of life in such patients.