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This therapeutic combination consists of the hypomethylating agent decitabine administered in conjunction with allogeneic hematopoietic stem cell transplantation (allo-HSCT). Primarily developed and investigated by the Bone Marrow Transplantation Center of the First Affiliated Hospital, Zhejiang University, this approach is utilized for patients with high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Decitabine is typically integrated into the conditioning regimen prior to the infusion of donor stem cells. Its mechanism of action involves the inhibition of DNA methyltransferases, leading to DNA hypomethylation and the reactivation of tumor suppressor genes. This epigenetic remodeling is thought to sensitize malignant cells to the graft-versus-leukemia (GVL) effect, upregulate tumor-associated antigens, and modulate the immune environment to reduce post-transplant relapse rates and improve overall survival in advanced myeloid malignancies.
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