Triptolide (TPL) is a diterpenoid isolated from the traditional Chinese medicine Tripterygium wilfordii. It has powerful antitumor, immunosuppressive and anti-inflammatory properties. Recent studies have shown that TPL can induce apoptosis of hematological tumor cells, inhibit their proliferation and survival, promote autophagy and ferroptosis, and enhance the efficacy of traditional chemotherapy and targeted therapies. Various molecules and signaling pathways, such as NF-κB, BCR-ABL, and Caspase, are involved in inducing apoptosis of leukemia cells. To solve the water solubility and toxic side effects of TPL, low-dose TPL (IC20) combined with chemotherapy drugs and various TPL derivatives have entered preclinical studies. This review discusses advances in molecular mechanism, the development and utilization of structural analogues of TPL in hematologic tumors in the past two decades, and clinical applications.
Keywords: TPL; Triptolide; derivatives; hematological malignancies; molecular mechanism.