Current and emerging strategies for management of myelodysplastic syndromes

The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia

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International scoring system for evaluating prognosis in myelodysplastic syndromes

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Acute Myeloid Leukemia

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Revised International Prognostic Scoring System for Myelodysplastic Syndromes

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Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study

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Frequent pathway mutations of splicing machinery in myelodysplasia

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Clonal hematopoiesis of indeterminate potential and its distinction from myelodysplastic syndromes

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Clinical and biological implications of driver mutations in myelodysplastic syndromes

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Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia

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Randomized Controlled Trial of Azacitidine in Patients With the Myelodysplastic Syndrome: A Study of the Cancer and Leukemia Group B

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Clinical Effect of Point Mutations in Myelodysplastic Syndromes

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Landscape of genetic lesions in 944 patients with myelodysplastic syndromes

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Decitabine improves patient outcomes in myelodysplastic syndromes

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Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia

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Durable Remissions with Ivosidenib in IDH1 -Mutated Relapsed or Refractory AML

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Lenalidomide in the Myelodysplastic Syndrome with Chromosome 5q Deletion

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Bone progenitor dysfunction induces myelodysplasia and secondary leukaemia

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Efficacy of Lenalidomide in Myelodysplastic Syndromes

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Lenalidomide induces ubiquitination and degradation of CK1α in del(5q) MDS

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<i>TP53</i> and Decitabine in Acute Myeloid Leukemia and Myelodysplastic Syndromes

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New insights into the prognostic impact of the karyotype in MDS and correlation with subtypes: evidence from a core dataset of 2124 patients

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Venetoclax with azacitidine disrupts energy metabolism and targets leukemia stem cells in patients with acute myeloid leukemia

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Prognostic Mutations in Myelodysplastic Syndrome after Stem-Cell Transplantation

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Results of a randomized study of 3 schedules of low-dose decitabine in higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia

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Myeloablative Versus Reduced-Intensity Hematopoietic Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes

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A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome

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Low-Dose Decitabine Versus Best Supportive Care in Elderly Patients With Intermediate- or High-Risk Myelodysplastic Syndrome (MDS) Ineligible for Intensive Chemotherapy: Final Results of the Randomized Phase III Study of the European Organisation for Research and Treatment of Cancer Leukemia Group and the German MDS Study Group

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Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes

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Leukaemogenesis induced by an activating β-catenin mutation in osteoblasts

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Targeting Cancer Stemness in the Clinic: From Hype to Hope

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H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers

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A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q

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Clinical significance of somatic mutation in unexplained blood cytopenia

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A validated decision model for treating the anaemia of myelodysplastic syndromes with erythropoietin + granulocyte colony‐stimulating factor: significant effects on quality of life

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SF3B1 mutation identifies a distinct subset of myelodysplastic syndrome with ring sideroblasts

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Prognostic impact of elevated pretransplantation serum ferritin in patients undergoing myeloablative stem cell transplantation

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Phase 2 study of lenalidomide in transfusion-dependent, low-risk, and intermediate-1–risk myelodysplastic syndromes with karyotypes other than deletion 5q

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Dynamics of clonal evolution in myelodysplastic syndromes

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Haploinsufficiency for ribosomal protein genes causes selective activation of p53 in human erythroid progenitor cells

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Myelodysplastic Cells in Patients Reprogram Mesenchymal Stromal Cells to Establish a Transplantable Stem Cell Niche Disease Unit

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Measurable residual disease-guided treatment with azacitidine to prevent haematological relapse in patients with myelodysplastic syndrome and acute myeloid leukaemia (RELAZA2): an open-label, multicentre, phase 2 trial

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Absence of nucleolar disruption after impairment of 40S ribosome biogenesis reveals an rpL11-translation-dependent mechanism of p53 induction

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A prognostic score for patients with lower risk myelodysplastic syndrome

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Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes

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SF3B1-mutant MDS as a distinct disease subtype: a proposal from the International Working Group for the Prognosis of MDS

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Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study

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Clonal diversity of recurrently mutated genes in myelodysplastic syndromes

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A Small Molecule RAS-Mimetic Disrupts RAS Association with Effector Proteins to Block Signaling

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Hematopoietic stem cell and progenitor cell mechanisms in myelodysplastic syndromes

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The central role of inflammatory signaling in the pathogenesis of myelodysplastic syndromes

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Leukaemogenic effects of Ptpn11 activating mutations in the stem cell microenvironment

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TP53 mutation status divides myelodysplastic syndromes with complex karyotypes into distinct prognostic subgroups

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Phase I Study of Oral Azacitidine in Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, and Acute Myeloid Leukemia

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Dose-Reduced Versus Standard Conditioning Followed by Allogeneic Stem-Cell Transplantation for Patients With Myelodysplastic Syndrome: A Prospective Randomized Phase III Study of the EBMT (RICMAC Trial)

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Genetic alterations of the cohesin complex genes in myeloid malignancies

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Epidemiology of Myelodysplastic Syndromes

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Randomized Phase II Study of Azacitidine Alone or in Combination With Lenalidomide or With Vorinostat in Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: North American Intergroup Study SWOG S1117

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Impact of TP53 mutation variant allele frequency on phenotype and outcomes in myelodysplastic syndromes

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Synthetic Lethal and Convergent Biological Effects of Cancer-Associated Spliceosomal Gene Mutations

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Randomized Phase III Study of Lenalidomide Versus Placebo in RBC Transfusion-Dependent Patients With Lower-Risk Non-del(5q) Myelodysplastic Syndromes and Ineligible for or Refractory to Erythropoiesis-Stimulating Agents

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The First-in-Class Anti-CD47 Antibody Magrolimab (5F9) in Combination with Azacitidine Is Effective in MDS and AML Patients: Ongoing Phase 1b Results

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Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996)

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A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-α in anemic patients with low-risk MDS

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Retrospective comparison of bone marrow and granulocyte colony-stimulating factor-mobilized peripheral blood progenitor cells for allogeneic stem cell transplantation using HLA identical sibling donors in myelodysplastic syndromes

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Synergistic effects of PRIMA-1^Met (APR-246) and 5-azacitidine in <i>TP53</i>-mutated myelodysplastic syndromes and acute myeloid leukemia

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Iron Chelation in Transfusion-Dependent Patients With Low- to Intermediate-1–Risk Myelodysplastic Syndromes: A Randomized Trial

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Stem and progenitor cell alterations in myelodysplastic syndromes.

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Long-term follow-up for up to 5 years on the risk of leukaemic progression in thrombocytopenic patients with lower-risk myelodysplastic syndromes treated with romiplostim or placebo in a randomised double-blind trial

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High‐dose darbepoetin alpha in the treatment of anaemia of lower risk myelodysplastic syndrome results of a phase II study

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Extended survival and reduced risk of AML progression in erythroid-responsive lenalidomide-treated patients with lower-risk del(5q) MDS

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Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents

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MDS overlap disorders and diagnostic boundaries

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Azacitidine with or without eltrombopag for first-line treatment of intermediate- or high-risk MDS with thrombocytopenia

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Molecular strategies for detection and quantitation of clonal cytotoxic T-cell responses in aplastic anemia and myelodysplastic syndrome

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Allogeneic Hematopoietic Cell Transplantation in Patients Age 60-70 Years with De Novo High-Risk Myelodysplastic Syndrome or Secondary Acute Myelogenous Leukemia: Comparison with Patients Lacking Donors Who Received Azacitidine

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Antisense STAT3 inhibitor decreases viability of myelodysplastic and leukemic stem cells

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Results of a Clinical Trial of H3B-8800, a Splicing Modulator, in Patients with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML) or Chronic Myelomonocytic Leukemia (CMML)

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Integrating the “Immunome” in the Stratification of Myelodysplastic Syndromes and Future Clinical Trial Design

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Comparative analysis between azacitidine and decitabine for the treatment of myelodysplastic syndromes

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Efficacy and Safety of Azacitidine (AZA) in Combination with the Anti-PD-L1 Durvalumab (durva) for the Front-Line Treatment of Older Patients (pts) with Acute Myeloid Leukemia (AML) Who Are Unfit for Intensive Chemotherapy (IC) and Pts with Higher-Risk Myelodysplastic Syndromes (HR-MDS): Results from a Large, International, Randomized Phase 2 Study

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How I treat MDS after hypomethylating agent failure

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The mesenchymal niche in MDS

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Phase 2 Results of APR-246 and Azacitidine (AZA) in Patients with TP53 mutant Myelodysplastic Syndromes (MDS) and Oligoblastic Acute Myeloid Leukemia (AML)

Sallman, D. A., Dezern, A. E., Garcia‐Manero, G., Steensma, D. P., Roboz, G. J., Sekeres, M. A., Cluzeau, T., Sweet, K. L., McLemore, A. F., McGraw, K. L., et al. (2019) · Blood · doi:10.1182/blood-2019-131055 · Cited by 72 · see citing works

A Phase II Study of Nivolumab or Ipilimumab with or without Azacitidine for Patients with Myelodysplastic Syndrome (MDS)

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Preliminary Results of a Phase 1 Study of Flotetuzumab, a CD123 x CD3 Bispecific Dart® Protein, in Patients with Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndrome

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Invariant patterns of clonal succession determine specific clinical features of myelodysplastic syndromes

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APR-246 Combined with Azacitidine (AZA) in TP53 Mutated Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML). a Phase 2 Study By the Groupe Francophone Des Myélodysplasies (GFM)

Cluzeau, T., Sébert, M., Rahmé, R., Cuzzubbo, S., Walter‐Petrich, A., Lehmann‐Che, J., Péterlin, P., Bève, B., Attalah, H. L., Chermat, F., et al. (2019) · Blood · doi:10.1182/blood-2019-125579 · Cited by 65 · see citing works

A randomized phase II trial of azacitidine +/- epoetin- in lower-risk myelodysplastic syndromes resistant to erythropoietic stimulating agents

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PAK1 is a therapeutic target in acute myeloid leukemia and myelodysplastic syndrome

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Pharmacokinetic Exposure Equivalence and Preliminary Efficacy and Safety from a Randomized Cross over Phase 3 Study (ASCERTAIN study) of an Oral Hypomethylating Agent ASTX727 (cedazuridine/decitabine) Compared to IV Decitabine

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The Clinical Management of Clonal Hematopoiesis

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A Phase I Study of Oral ARRY-614, a p38 MAPK/Tie2 Dual Inhibitor, in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes

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Limited clinical efficacy of azacitidine in transfusion-dependent, growth factor-resistant, low- and Int-1-risk MDS: Results from the nordic NMDSG08A phase II trial

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A phase II study of guadecitabine in higher-risk myelodysplastic syndrome and low blast count acute myeloid leukemia after azacitidine failure

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Pexmetinib: A Novel Dual Inhibitor of Tie2 and p38 MAPK with Efficacy in Preclinical Models of Myelodysplastic Syndromes and Acute Myeloid Leukemia

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Progression in patients with low- and intermediate-1-risk del(5q) myelodysplastic syndromes is predicted by a limited subset of mutations

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A Phase 1b Study Evaluating the Safety and Efficacy of Venetoclax in Combination with Azacitidine in Treatment-Naïve Patients with Higher-Risk Myelodysplastic Syndrome

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Macrophage Checkpoint Blockade in Cancer — Back to the Future

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Prognosis of patients with intermediate risk IPSS‐R myelodysplastic syndrome indicates variable outcomes and need for models beyond IPSS‐R

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A phase II multicenter rabbit anti-thymocyte globulin trial in patients with myelodysplastic syndromes identifying a novel model for response prediction

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Preclinical modeling of myelodysplastic syndromes

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Iron Chelation in Transfusion-Dependent Patients With Low- to Intermediate-1–Risk Myelodysplastic Syndromes

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Phase 1/1b Study of the Stapled Peptide ALRN-6924, a Dual Inhibitor of MDMX and MDM2, As Monotherapy or in Combination with Cytarabine for the Treatment of Relapsed/Refractory AML and Advanced MDS with TP53 Wild-Type

Sallman, D. A., Borate, U., Cull, E. H., Donnellan, W. B., Komrokji, R. S., Steidl, U. G., Corvez, M. M., Payton, M., Annis, D. A., Pinchasik, D. E., et al. (2018) · Blood · doi:10.1182/blood-2018-99-118780 · Cited by 35 · see citing works

Lenalidomide Induces Lipid Raft Assembly to Enhance Erythropoietin Receptor Signaling in Myelodysplastic Syndrome Progenitors

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Lenalidomide Stabilizes the Erythropoietin Receptor by Inhibiting the E3 Ubiquitin Ligase RNF41

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A phase 2 trial of the oral smoothened inhibitor glasdegib in refractory myelodysplastic syndromes (MDS)

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Azacitidine/Decitabine Synergism with the NEDD8-Activating Enzyme Inhibitor MLN4924 in Pre-Clinical AML Models

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A phase 1 dose escalation study of the IDH1m inhibitor, FT-2102, in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

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A Clinical Study of OPN-305, a Toll-like Receptor 2 (TLR-2) Antibody, in Patients with Lower Risk Myelodysplastic Syndromes (MDS) That Have Received Prior Hypomethylating Agent (HMA) Therapy

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Roxadustat (FG4592; ASP1517; AZD9941) in the Treatment of Anemia in Patients with Lower Risk Myelodysplastic Syndrome (LR-MDS) and Low Red Blood Cell (RBC) Transfusion Burden (LTB)

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TP53 State Dictates Genome Stability, Clinical Presentation and Outcomes in Myelodysplastic Syndromes

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