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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8508686/ https://www.ncbi.nlm.nih.gov/pubmed/34638574 http://dx.doi.org/10.3390/ijms221910232 |
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author | Lee, Paul Yim, Rita Yung, Yammy Chu, Hiu-Tung Yip, Pui-Kwan Gill, Harinder |
author_facet | Lee, Paul Yim, Rita Yung, Yammy Chu, Hiu-Tung Yip, Pui-Kwan Gill, Harinder |
author_sort | Lee, Paul |
collection | PubMed |
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. |
format | Online Article Text |
id | pubmed-8508686 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-85086862021-10-13 Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome Lee, Paul Yim, Rita Yung, Yammy Chu, Hiu-Tung Yip, Pui-Kwan Gill, Harinder Int J Mol Sci Review 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. MDPI 2021-09-23 /pmc/articles/PMC8508686/ /pubmed/34638574 http://dx.doi.org/10.3390/ijms221910232 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Lee, Paul Yim, Rita Yung, Yammy Chu, Hiu-Tung Yip, Pui-Kwan Gill, Harinder Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome |
title | Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome |
title_full | Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome |
title_fullStr | Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome |
title_full_unstemmed | Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome |
title_short | Molecular Targeted Therapy and Immunotherapy for Myelodysplastic Syndrome |
title_sort | molecular targeted therapy and immunotherapy for myelodysplastic syndrome |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8508686/ https://www.ncbi.nlm.nih.gov/pubmed/34638574 http://dx.doi.org/10.3390/ijms221910232 |
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