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.
Highlights
Myelodysplastic syndrome (MDS) represents a group of heterogeneous myeloid clonal diseases that originate from hematopoietic stem cells and are characterized as having an abnormal development of myeloid cells
Hypermethylation in DNA is associated with tumor progression and differentiation arrest, which has been detected in myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) [3, 4]
Decitabine (DAC), a DNA hypomethylating agent, is considered a frontline therapy for intermediate or high-risk patients who were not candidates for allo-HSCT according to National Comprehensive Cancer Network (NCCN) Guidelines (Version 2.2020) for myelodysplastic syndromes
Summary
Myelodysplastic syndrome (MDS) represents a group of heterogeneous myeloid clonal diseases that originate from hematopoietic stem cells and are characterized as having an abnormal development of myeloid cells. It is known that allogeneic hematopoietic stem cell transplantation (allo-HSCT) promotes curative effects in patients with. MDS; clinicians often face impediments to its widespread application, concerning infectious and other toxicities associated with conditioning regimens, the development of significant graft-vs.-host disease (GVHD) with resultant organ dysfunction, infection from prolonged immunosuppression, and in some cases, a high rate of disease progression [2]. Hypermethylation in DNA is associated with tumor progression and differentiation arrest, which has been detected in myelodysplastic syndrome (MDS) and AML [3, 4]. Decitabine (DAC), a DNA hypomethylating agent, is considered a frontline therapy for intermediate or high-risk patients who were not candidates for allo-HSCT according to National Comprehensive Cancer Network (NCCN) Guidelines (Version 2.2020) for myelodysplastic syndromes. The clinical efficacy of demethylation drugs to treat patients with MDS remains limited
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