Decitabine With or Without Micro-Transplantation for the Treatment of Intermediate or High-Risk Myelodysplastic Syndrome: A Chinese Single-Center Retrospective Study of 22 Patients

Abstract

The treatment outcomes of intermediate or high-risk myelodysplastic syndrome (MDS) remain unsatisfactory. This study was designed to evaluate the safety and efficacy of human leukocyte antigen (HLA)-mismatched hematopoietic stem cell micro-transplantation (MST) in patients with MDS. A total of 22 patients with MDS, ranging between the ages of 39 and 74, were enrolled in this study. Eleven patients were given decitabine (DAC), a DNA methyltransferase inhibitor, combined with HLA-mismatched MST (MST-DAC group), and the remaining patients were given decitabine only (DAC group). The median overall survival (OS) of the MST-DAC group was higher than that of the DAC group (24 vs. 14.3 months; HR 0.32; 95% CI: 0.11–0.96; p = 0.04), although it is a study with small samples. The overall response rate (ORR), marrow complete remission (mCR), plus hematological improvement (HI) rates of the MST-DAC group were higher than that of the DAC group (81.8 vs. 54.5%, p = 0.36; 63.6 vs. 27.3%, p = 0.09, respectively); however, there were no statistical differences between the two groups, which may be attributed to the limited number of cases evaluated in this study. No graft-vs.-host disease was observed in the MST-DAC group. Patients in the MST-DAC group demonstrated a slightly lower incidence of hematological and non-hematological adverse events (AEs). DAC combined with HLA-mismatched MST may provide a novel, effective, and safe treatment for use in intermediate or high-risk MDS pathologies.

Generalist in allogeneic hematopoietic stem cell transplantation for MDS or AML: Epigenetic therapy

“…The report by Li et al. also showed that DAC combined with MST could increase the overall response rate ( 40 ). This is a new scheme of DAC combined with micro-transplantation (MST), which can effectively prolong the survival of patients with high-risk MDS and TAML, especially for MDS patients, and may become an effective bridging method for allogeneic transplantation.…”

Outcomes of intermediate or high‐risk CMML patients treated with hypomethylating agents combined with venetoclax: A single center experience

“…The WT1 is a tumor marker found in leukemic blasts 27 and is closely associated with a poorer long‐term prognosis in AML. In our previous studies, 28 , 29 WT1 transcript levels proved to be effective in assessing disease progression in patients with MDS (including CMML). In this study, we also found a strong correlation between changes in WT1 mRNA and treatment response which indicates the effectiveness of Vene combination therapy indirectly.…”