Enhancing our chances of picking a winner in higher-risk myelodysplastic syndromes

Article Hematology

Molecular characterization of mutant TP53 acute myeloid leukemia and high-risk myelodysplastic syndrome

Tim Grob et al.

Summary: Mutant TP53 AML and MDS-EB share similar molecular characteristics and survival outcomes, suggesting that they should be considered as distinct molecular disease entities.

BLOOD (2022)

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Article Hematology

A randomised phase II study of azacitidine (AZA) alone or with Lenalidomide (LEN), Valproic acid (VPA) or Idarubicin (IDA) in higher-Risk MDS or low blast AML: GFM's pick a winner trial, with the impact of somatic mutations

Lionel Ades et al.

Summary: The combination of AZA with other drugs did not improve the outcome observed in higher-risk MDS patients. There was no significant difference in survival and progression-free survival among the different treatment arms. Some combinations led to increased infection rates and hospitalization due to myelosuppression. Factors associated with better treatment response and survival included certain clinical indicators and gene mutations.

BRITISH JOURNAL OF HAEMATOLOGY (2022)

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Letter Oncology

Randomized phase 2 trial of pevonedistat plus azacitidine versus azacitidine for higher-risk MDS/CMML or low-blast AML

Mikkael A. Sekeres et al.

LEUKEMIA (2021)

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Article Oncology

Validation of International Working Group response criteria in higher-risk myelodysplastic syndromes: A report on behalf of the MDS Clinical Research Consortium

Rami S. Komrokji et al.

Summary: The utility of the IWG 2006 response criteria for MDS as a surrogate endpoint for outcomes is unclear, but it can be used as a surrogate endpoint for OS in higher-risk MDS pts. Any response associated with restoration of effective hematopoiesis is associated with better outcome.

CANCER MEDICINE (2021)

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Article Hematology