ASH 2021: Highlights MDS

Prof. dr. Arjan van de Loosdrecht vat het belangrijkste ASH-nieuws samen op het gebied van myelodysplastisch syndroom.

Directe links naar de ASH-abstracts:

Basaal / pathogenese

• Single-Cell Multi-Omics Defines the Cell-Type Specific Impact of SF3B1 Splicing Factor Mutations on Hematopoietic Differentiation in Human Clonal Hematopoiesis and Myelodysplastic Syndromes
• Integrative Analysis of Primary SF3B1mt Ring Sideroblasts Provides Fundamental Insights into MDS-RS Pathogenesis and Dyserythropoiesis
• Modeling Clonal Progression in SF3B1-Mutant Myelodysplastic Syndrome

Diagnostiek

• Multicenter Prospective Evaluation of Diagnostic Potential of Flow Cytometric Aberrancies in Myelodysplastic Syndromes
• ELN IMDS Flow Working Group Validation of the Monocyte Assay for Chronic Myelomonocytic Leukemia Diagnosis By Flow Cytometry

Prognostiek

• Molecular International Prognosis Scoring System for Myelodysplastic Syndromes

Therapie: laag risico

• Ivosidenib Monotherapy Is Effective in Patients with IDH1 Mutated Myelodysplastic Syndrome (MDS): The Idiome Phase 2 Study By the GFM Group
• Enasidenib (ENA) Is Effective in Patients with IDH2 Mutated Myelodysplastic Syndrome (MDS) : The Ideal Phase 2 Study By the GFM Group
• Lenalidomide and Eltrombopag for Treatment in Low or Intermediate Risk Myelodysplastic Syndrome: Result of a Phase 2 Study Combination Clinical Trial
• Oral Decitabine/Cedazuridine in Patients with Lower Risk Myelodysplastic Syndrome: A Longer-Term Follow-up of from the Ascertain Study

Therapie: hoog risico

• Pevonedistat (PEV) + Azacitidine (AZA) Versus AZA Alone As First-Line Treatment for Patients with Higher-Risk Myelodysplastic Syndromes (MDS)/Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML) with 20–30% Marrow Blasts: The Randomized Phase 3 PANTHER Trial (NCT03268954)
• CPX 351 As First Line Treatment in Higher Risk MDS. a Phase II Trial By the GFM
• Molecular Responses Are Observed across Mutational Spectrum in Treatment-Naïve Higher-Risk Myelodysplastic Syndrome Patients Treated with Venetoclax Plus Azacitidine
• Efficacy and Safety of Sabatolimab (MBG453) in Combination with Hypomethylating Agents (HMAs) in Patients (Pts) with Very High/High-Risk Myelodysplastic Syndrome (vHR/HR-MDS) and Acute Myeloid Leukemia (AML): Final Analysis from a Phase Ib Study