title: Salvage therapy for refractory chronic graft-versus-host disease with mycophenolate mofetil and tacrolimus.
abstract: Chronic graft-versus-host disease (GVHD) is a common late complication of allogeneic bone marrow transplantation (BMT) and is the principal cause of morbidity and non-relapse mortality. The improved management of acute GVHD has not translated into lower rates of chronic GVHD as older patients undergo allogeneic BMT, more patients receive unrelated or related mismatched allogeneic BMT, and donor lymphocyte infusion is increasingly used for treatment of post-BMT relapses. Patients with high risk chronic GVHD or those who fail on standard therapy have a bad prognosis. Salvage therapies have produced disappointing results. Here, we present a retrospective analysis of 26 patients where a steroid sparing synergistic combination of mycophenolate mofetil (MMF) and tacrolimus was used in refractory chronic GVHD. 46% patients showed an objective response, 11.5% had stable disease, 34.6% had progression and 7.7% were not evaluable. The combination was well tolerated. This promising preliminary result has prompted a trial to assess the safety and efficacy of this regimen in patients with chronic GVHD who have failed prior therapy and to determine if it would improve survival as well as quality of life in such patients.
authors.affiliation: The Bone Marrow Transplant Program, Johns Hopkins Oncology Center, Baltimore, MD, USA.
authors.forename: B
authors.initials: B
authors.lastname: Mookerjee
authors.forename: V
authors.initials: V
authors.lastname: Altomonte
authors.forename: G
authors.initials: G
authors.lastname: Vogelsang
mesh_terms: D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D004357:Drug Synergism; D004359:Drug Therapy, Combination; D004791:Enzyme Inhibitors / Q000008:administration & dosage / Q000627:therapeutic use; D005260:Female; D006082:Graft Enhancement, Immunologic; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000209:etiology; D006801:Humans; D007168:IMP Dehydrogenase / Q000037:antagonists & inhibitors; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D017710:Lymphocyte Transfusion; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011355:Prodrugs / Q000627:therapeutic use*; D012189:Retrospective Studies; D016879:Salvage Therapy; D016559:Tacrolimus / Q000008:administration & dosage / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D004791:Enzyme Inhibitors; D007166:Immunosuppressive Agents; D011355:Prodrugs; D007168:IMP Dehydrogenase; D009173:Mycophenolic Acid; D016559:Tacrolimus