Low-dose methotrexate for the treatment of graft-versus-host disease after allogeneic hematopoietic stem cell transplantation

Methotrexate (MTX) is commonly used in the prophylaxis of graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (alloHSCT). In order to evaluate the efficacy and safety of low-dose MTX treatment in patients with GVHD after allo-HSCT, 38 patients with acute GVHD (aGVHD), chronic GVHD (cGVHD) or GVHD post-donor lymphocyte infusion (post-DLI GVHD) after allo-HSCT received intravenous MTX at a dose of 5 or 10mg every 5-7 days until a complete or partial response was seen, or there was treatment failure or intolerable side effects. The overall response rate was 94.7% (18/19 patients) in patients with aGVHD, 76.2% (16/21 patients) in patients with cGVHD and 2/2 in patients with post-DLI GVHD. The response rate for GVHD involving various organs was 100% in skin, 75% in gut, 55.6% in liver, 75% in mouth and 100% in the eye, among all enrolled patients. Side effects were minor. Short-term, low-dose MTX is a tolerable and an effective regimen for patients with aGVHD, cGVHD or post-DLI GVHD after allo-HSCT.