First-line therapy for chronic graft-versus-host disease that includes low-dose methotrexate is associated with a high response rate.

We report the results of low-dose methotrexate (MTX) as first-line therapy mostly in combination with other immunosuppressive agents in patients with chronic graft-versus-host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Between November 2001 and March 2008, 86 patients with cGVHD after allo-HSCT received low-dose MTX therapy until a complete or partial response (CR, PR) was achieved, or until treatment failure or intolerable side effects were found. The median time from HSCT to the start of MTX was 154 (range: 80-993) days. The median number of MTX administrations was 4 (range: 2-18). The overall response rate among all enrolled patients was 83% (71 of 86 patients). The response rate for GVHD involving various organs was 90% (45 of 50) in the skin, 75% (39 of 52) in the liver, 42% (5 of 12) in the mouth, 3 of 7 in the eye, and 2 of 2 in the gut. In addition, MTX treatment allowed for a significant reduction in the prednisone dosage (median 90%) from 20 (2.5-100) mg at the start of MTX administration to 5 (0-30) mg 1 month after MTX was last used. Multivariate analysis showed that the only significant factor related to higher CR rate was sole organ involvement (P = .007). Grade 3 toxicities occurred in only 3 patients presenting cytopenias or oral mucositis. From this analysis, MTX appears to be a well-tolerated, effective, and inexpensive agent when used as a first-line treatment in combination with other immunosuppressive agents for cGVHD, especially for skin or sole organ involvement without concomitant thrombocytopenia.

[Methotrexate for treatment of graft versus host disease after allogeneic hematopoietic stem cell transplantation].

OBJECTIVE: To evaluate the efficacy and safety of low-dose methotrexate in patients with graft versus host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (Allo-HSCT). METHODS: Thirty-one patients with minor or moderate grade acute GVHD (aGVHD), chronic GVHD (cGVHD) or GVHD post donor lymphocyte infusion (post-DLI GVHD) after Allo-HSCT received intravenously administrated methotrexate at a dose of 5 or 10mg every 5 to 7 days until achieving complete or partial responses, treatment failure or intolerable side effects. RESULTS: The overall response rate was 93.8% (15/16 patients) in patients with aGVHD, 75% (12/16 patients) in patients with cGVHD and 100% (2/2 in patients) with post-DLI GVHD. The response rate for GVHD involving organs was 100% in skin, 60% in gut, 71% in liver, 75% in mouth and 100% in eyes. Side effects were minor. During the therapy, the other immunosuppressive agents were reduced. CONCLUSION: Short-term low-dose methotrexate is a tolerable and effective regimen for patients with minor or moderate grade aGVHD, cGVHD or post-DLI GVHD after Allo-HSCT.