thalidomide + methotrexate

thalidomide + methotrexate is a combination therapy regimen being investigated by the Institute of Hematology & Blood Diseases Hospital in China for the treatment of symptomatic large granular lymphocytic leukemia (LGLL). This regimen typically involves the immunomodulatory agent thalidomide and the antimetabolite methotrexate, often administered alongside prednisone as part of a TPM regimen or as a step-up TM regimen for patients with insufficient response to thalidomide monotherapy. Thalidomide acts by modulating the immune system through the Cereblon E3 ubiquitin ligase complex, while methotrexate serves as an anti-inflammatory and immunosuppressive agent that inhibits dihydrofolate reductase and suppresses aberrant JAK/STAT pathway signaling, which is a hallmark of LGLL pathogenesis. Clinical studies have demonstrated high complete remission and overall response rates with this combination, significantly outperforming historical benchmarks for standard immunosuppressive monotherapies in this patient population.

Other names

Thalidomide and methotrexate