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No benefit of hypomethylating agents compared to supportive care for higher risk myelodysplastic syndrome.

This study evaluated the role of hypomethylating agents (HMA) compared to best supportive care (BSC) for patients with high or very-high (H/VH) risk myelodysplastic syndrome (MDS) according to the Revised International Prognostic Scoring System. Methods: A total of 279 H/VH risk MDS patients registered in the Korean MDS Working Party database were retrospectively analyzed. Results: HMA therapy was administered to 205 patients (73.5%), including 31 patients (11.1%) who then received allogeneic hematopoietic cell transplantation (allo-HCT), while 74 patients (26.5%) received BSC or allo-HCT without HMA. The 3-year overall survival (OS) rates were 53.1% ± 10.7% for allo-HCT with HMA, 75% ± 21.7% for allo-HCT without HMA, 17.3% ± 3.6% for HMA, and 20.8% ± 6.9% for BSC groups (p
Source: The Korean Journal of Internal Medicine - Category: Internal Medicine Authors: Tags: Korean J Intern Med Source Type: research

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Background: Studies of bone marrow cell clonal alterations in patients with Fanconi anemia (FA), a cancer-prone inherited bone marrow failure (BMF) syndrome, have focused on their role in progression from BMF to myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The role of such alterations on patient outcomes after hematopoietic cell transplantation (HCT) is unknown.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background: Azacitidine (Aza) and donor lymphocyte infusions (DLI) confer survival advantage with improved tolerability for patients with relapsed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) following allogenic stem cell transplantation (SCT).
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background: We tried to clarify the clinical significance of Wilms tumor gene-1 expression in peripheral blood cells (PB-WT1) for early detection of relapse in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Myelodysplastic syndrome (MDS) is an aging-associated group of clonal disorders characterized by ineffective hematopoiesis, leading to cytopenias and an increased risk of developing acute myeloid leukemia (AML). MDS arises from abnormal hematopoietic stem cells (HSCs). The only potentially curative therapy available for MDS patients is hematopoietic cell transplantation (HCT), but relapse is common, likely due to the inability of current therapies to effectively eliminate disease-initiating MDS HSCs.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background: There are very limited data on the impact of BM fibrosis on prognosis and survival of AML and MDS especially after allogeneic HSCT. The current system of Reticulin Score for grading fibrosis according to (WHO 2008) is only semi-quantitative and subject to inter-observer inconsistency and no performance data. We evaluated BM fibrosis by a new objective and highly quantitative method of Computer-assisted Image Analysis (CIA) and correlated it to cytogenetics, survival and treatment-related mortality after allo-sib HSCT.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
CPX-351 is a dual-drug liposomal encapsulation of cytarabine (C) and daunorubicin (D) that delivers a synergistic 5:1 drug ratio and is FDA-approved for adults with newly diagnosed therapy-related AML or AML with MDS-related changes. In a randomized, phase 3 study (NCT01696084) in patients (pts) 60-75 y with newly diagnosed sAML, CPX-351 significantly improved overall survival (OS) and remission rates vs 7  + 3. RAEB-t AML, often defined as bone marrow blasts 20%-29%, shares many features with myelodysplastic syndrome (MDS).
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background: Patients (pts) with High-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) often proceed to allogeneic hematopoietic stem cell transplant (alloHSCT) due to poorer outcomes. This is the only chance of cure; however, relapse rates after alloHSCT remain high and range between 25-50%. Maintenance approaches post-HSCT, such as hypomethylating agents or tyrosine kinase inhibitors, have been used to decrease the relapse rate with mixed results. Lenalidomide is known to augment T/NK cell activity/proliferation which lead us to hypothesize that it will enhance the Graft-versus-Leukemia (GvL) effect wit...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background: Allogeneic hematopoietic stem cell transplantation (HCT) is a highly effective therapy for acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS), but can be associated with significant morbidity and mortality. Low pre-HCT diffusing capacity for carbon monoxide (DLCO) is a risk factor for death post-HCT. We sought to identify additional patient- or HCT-related variables that might modulate this risk.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background/Objective: Allogeneic hematopoietic cell transplantation (HCT) is a potential curative option for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) but carries a high risk of relapse. Azacitidine (aza) may enhance the graft-versus-leukemia effect without an increase in graft-versus-host disease (GVHD) and has been evaluated for relapse prevention following HCT with reduced intensity conditioning. Given the poor outcomes associated with relapsed AML and MDS post-HCT, our institution administered maintenance aza to these patients.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Background: Outcomes of allogeneic stem cell transplantation (AHSCT) vary based on both disease and patient characteristics. Our group developed a model to predict survival in patients with AML/MDS using 3 factors, 2 disease-related (cytogenetics, disease status at transplant) and 1 patient-related (HCT-CI) (Bachegowda et  al.Blood.2017). This model effectively stratified patients into 3 risk groups with very different survival. Here we aim to validate this model in a large cohort of AML/MDS patients receiving AHSCT with different donors.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
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