Venetoclax and hypomethylating agent therapy in myelodysplastic syndromes: Big picture perspective

Gangat, Naseema; Komrokji, Rami; Tefferi, Ayalew

doi:10.1002/ajh.26781

Cited by 2 publications

(1 citation statement)

References 25 publications

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“…Recently, it was reported that the combination of HMA (azacitidine) with venetoclax in high-risk MDS might lead to a higher response rate. The combination of HMA and venetoclax was approved for high-risk MDS patients ineligible for allo-SCT [ 106 ].…”

Section: Treatment Of Myelodysplastic Neoplasmmentioning

confidence: 99%

The Genetic Landscape of Myelodysplastic Neoplasm Progression to Acute Myeloid Leukemia

Bănescu

Tripon

Muntean

2023

IJMS

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Myelodysplastic neoplasm (MDS) represents a heterogeneous group of myeloid disorders that originate from the hematopoietic stem and progenitor cells that lead to the development of clonal hematopoiesis. MDS was characterized by an increased risk of transformation into acute myeloid leukemia (AML). In recent years, with the aid of next-generation sequencing (NGS), an increasing number of molecular aberrations were discovered, such as recurrent mutations in FLT3, NPM1, DNMT3A, TP53, NRAS, and RUNX1 genes. During MDS progression to leukemia, the order of gene mutation acquisition is not random and is important when considering the prognostic impact. Moreover, the co-occurrence of certain gene mutations is not random; some of the combinations of gene mutations seem to have a high frequency (ASXL1 and U2AF1), while the co-occurrence of mutations in splicing factor genes is rarely observed. Recent progress in the understanding of molecular events has led to MDS transformation into AML and unraveling the genetic signature has paved the way for developing novel targeted and personalized treatments. This article reviews the genetic abnormalities that increase the risk of MDS transformation to AML, and the impact of genetic changes on evolution. Selected therapies for MDS and MDS progression to AML are also discussed.

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Section: Treatment Of Myelodysplastic Neoplasmmentioning

confidence: 99%

The Genetic Landscape of Myelodysplastic Neoplasm Progression to Acute Myeloid Leukemia

Bănescu

Tripon

Muntean

2023

IJMS

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Combination therapy with venetoclax and azacitidine for the treatment of myelodysplastic syndromes with DDX41 mutations

Wang,

Xiao,

Qin

et al. 2024

Hematology

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Venetoclax and hypomethylating agent therapy in myelodysplastic syndromes: Big picture perspective

Abstract: The importance of the biological and clinical continuum of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) was officially acknowledged through the designation of the MDS/AML category in the International Consensus Classification (ICC) of Mye-

Cited by 2 publications

References 25 publications

The Genetic Landscape of Myelodysplastic Neoplasm Progression to Acute Myeloid Leukemia

The Genetic Landscape of Myelodysplastic Neoplasm Progression to Acute Myeloid Leukemia

Combination therapy with venetoclax and azacitidine for the treatment of myelodysplastic syndromes with DDX41 mutations

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