Safety and potential efficacy of low-dose methotrexate for treatment of chronic graft-versus-host disease

• Published in: Bone marrow transplantation (Basingstoke) Vol. 36; no. 4; pp. 337 - 341
• Main Authors: GIACCONE, L, MARTIN, P, CARPENTER, P, MORAVEC, C, HOOPER, H, FUNKE, V. A. M, STORB, R, FLOWERS, M. E. D
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01-08-2005
• Subjects: Adult; Aged; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Anti-inflammatory agents; Autoimmune diseases; Biological and medical sciences; Blood transfusion; Bone marrow; Bone marrow, stem cells transplantation. Graft versus host reaction; Chronic Disease; Clinical trials; Complications and side effects; Diagnosis; Disease control; Dosage and administration; Drug Evaluation; Drug therapy; Drug Therapy, Combination; Fasciitis; Female; Graft versus host disease; Graft versus host reaction; Graft vs Host Disease - drug therapy; Graft vs Host Disease - pathology; Growth factors; Health services; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic stem cells; Humans; Inflammation; Male; Medical sciences; Methotrexate; Methotrexate - administration & dosage; Methotrexate - toxicity; Middle Aged; Patient outcomes; Patients; Prednisone; Prednisone - administration & dosage; Reagents; Retrospective Studies; Risk factors; Salvage Therapy; Scleroderma; Stem cell transplantation; Toxicity; Transfusion; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Transplantation; Treatment Outcome; United States; Antineoplastic agent; Hematology; Toxicity; Chronic disease; Stem cell; Low dose; Treatment efficiency; chronic graft-versus-host disease; Hematopoietic cell; Homograft; Graft versus host reaction; Antifolate; Treatment; Antimetabolic; hematopoietic cell transplantation; Graft; Methotrexate
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/sj.bmt.1705022

Low-dose methotrexate (MTX) is widely used in autoimmune diseases because of its anti-inflammatory activity. We report here the results of a retrospective study to review the outcomes of low-dose MTX used for treatment of refractory chronic graft-versus-host disease GVHD, with the goal of reducing the amount of prednisone needed to control the disease. In all, 14 patients with refractory chronic GVHD received MTX at a dose of 7.5 mg/m(2)/weekly for 3--0 weeks. Also, 11 patients had skin involvement, often with scleroderma or fasciitis. The median duration of chronic GVHD at the start of MTX was 38 (range 1--35) months. In this retrospective review, we found no grade 3-- toxicities, and none of the patients needed blood transfusion or growth factors. In 10 patients (71%), GVHD could be adequately controlled with prednisone at doses below 1 mg/kg every other day without the addition of other agents. Four patients decreased the amount of concomitant immunosuppressive treatment, five continued with the same regimen, four required an increase in immunosuppressive treatment, and one decided to discontinue all treatment. From this preliminary analysis, MTX appears to be a well-tolerated, inexpensive and possibly steroid-sparing agent that is worthy of further evaluation in prospective trials for treatment of chronic GVHD.