Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome

Myelodysplastic syndrome (MDS) is a heterogeneous, clonal hematological disorder characterized by ineffective hematopoiesis, cytopenia, morphologic dysplasia, and predisposition to acute myeloid leukemia (AML). Stem cell genomic instability, microenvironmental aberrations, and somatic mutations cont...

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Veröffentlicht in:International journal of molecular sciences 2021-10, Vol.22 (19), p.10232
Hauptverfasser: Lee, Paul, Yim, Rita, Yung, Yammy, Chu, Hiu-Tung, Yip, Pui-Kwan, Gill, Harinder
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container_issue 19
container_start_page 10232
container_title International journal of molecular sciences
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creator Lee, Paul
Yim, Rita
Yung, Yammy
Chu, Hiu-Tung
Yip, Pui-Kwan
Gill, Harinder
description Myelodysplastic syndrome (MDS) is a heterogeneous, clonal hematological disorder characterized by ineffective hematopoiesis, cytopenia, morphologic dysplasia, and predisposition to acute myeloid leukemia (AML). Stem cell genomic instability, microenvironmental aberrations, and somatic mutations contribute to leukemic transformation. The hypomethylating agents (HMAs), azacitidine and decitabine are the standard of care for patients with higher-risk MDS. Although these agents induce responses in up to 40–60% of patients, primary or secondary drug resistance is relatively common. To improve the treatment outcome, combinational therapies comprising HMA with targeted therapy or immunotherapy are being evaluated and are under continuous development. This review provides a comprehensive update of the molecular pathogenesis and immune-dysregulations involved in MDS, mechanisms of resistance to HMA, and strategies to overcome HMA resistance.
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source MDPI - Multidisciplinary Digital Publishing Institute; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; PubMed Central
subjects 5-aza-2'-deoxycytidine
Acute myeloid leukemia
Anemia
Cell division
Chromosomes
Drug resistance
Dysplasia
Epigenetics
Genomic instability
Hematological diseases
Hematology
Hematopoiesis
Immunotherapy
Kinases
Leukemia
Medical prognosis
Mutation
Myelodysplastic syndrome
Myelodysplastic syndromes
Myeloid leukemia
Patients
Proteins
Review
Signal transduction
Stem cells
title Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome
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