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Methotrexate combined with methylprednisolone is an investigational therapeutic regimen being evaluated as a first-line treatment for grade II-IV acute graft-versus-host disease (aGVHD) in patients who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). Developed and studied primarily by researchers at Peking University People's Hospital, this combination therapy utilizes low-dose (mini-dose) methotrexate as an adjunct to standard corticosteroid therapy (methylprednisolone). Methotrexate is a small molecule antimetabolite that inhibits dihydrofolate reductase, thereby interfering with DNA synthesis and suppressing the proliferation of T-cells involved in the alloimmune response. Methylprednisolone, a synthetic glucocorticoid, provides potent anti-inflammatory and immunosuppressive effects by activating the glucocorticoid receptor. The combination is intended to enhance the efficacy of standard steroid therapy and improve complete response rates in patients with aGVHD.
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