Abstract
Myelodysplastic syndromes (MDS) are a heterogeneous group of hematologic diseases, mainly affecting the elderly, characterized by unilinear or multilinear peripheral cytopenia, bone marrow ineffective haemopoiesis, and a varying risk of progression to acute myeloid leukemia (AML). On the basis of the prognostic score systems currently used, MDS patients are generally classified as having higher risk (HR) or lower risk (LR) MDS. Two drugs, azacitidine (AZA) and decitabine (DAC), defined, because of their proven mechanism of action, as DNA methyltransferase inhibitors (DNMTIs), or hypomethylating agents (HMAs), have proven effective in improving peripheral cytopenias and quality of life, reducing or eliminating transfusion need, delaying leukemic evolution, and (only for AZA) prolonging overall survival (OS). HMAs are currently the first therapeutic choice for MDS patients who are not candidates for allogeneic hematopoietic stem cell transplantation (allo-HSCT). HMAs have also been used before and after allo-HSCT, but their role in this setting needs to be confirmed by larger studies. Although data from several clinical and biological studies might help to identify patients with a higher probability to respond to HMAs, to date this treatment should not be denied to any HR MDS patient. Several attempts have been made to combine HMAs with other therapeutic agents, and these results await confirmation by further studies.
Keywords: Azacitidine, decitabine, DNA methyltransferase inhibitors, hypomethylating agents, myelodysplastic syndromes.
Current Pharmaceutical Design
Title:Clinical Impact of Hypomethylating Agents in the Treatment of Myelodysplastic Syndromes
Volume: 22 Issue: 16
Author(s): Carlo Finelli, Matilde Y. Follo, Marta Stanzani, Sarah Parisi, Cristina Clissa, Sara Mongiorgi, Marilena Barraco and Lucio Cocco
Affiliation:
Keywords: Azacitidine, decitabine, DNA methyltransferase inhibitors, hypomethylating agents, myelodysplastic syndromes.
Abstract: Myelodysplastic syndromes (MDS) are a heterogeneous group of hematologic diseases, mainly affecting the elderly, characterized by unilinear or multilinear peripheral cytopenia, bone marrow ineffective haemopoiesis, and a varying risk of progression to acute myeloid leukemia (AML). On the basis of the prognostic score systems currently used, MDS patients are generally classified as having higher risk (HR) or lower risk (LR) MDS. Two drugs, azacitidine (AZA) and decitabine (DAC), defined, because of their proven mechanism of action, as DNA methyltransferase inhibitors (DNMTIs), or hypomethylating agents (HMAs), have proven effective in improving peripheral cytopenias and quality of life, reducing or eliminating transfusion need, delaying leukemic evolution, and (only for AZA) prolonging overall survival (OS). HMAs are currently the first therapeutic choice for MDS patients who are not candidates for allogeneic hematopoietic stem cell transplantation (allo-HSCT). HMAs have also been used before and after allo-HSCT, but their role in this setting needs to be confirmed by larger studies. Although data from several clinical and biological studies might help to identify patients with a higher probability to respond to HMAs, to date this treatment should not be denied to any HR MDS patient. Several attempts have been made to combine HMAs with other therapeutic agents, and these results await confirmation by further studies.
Export Options
About this article
Cite this article as:
Finelli Carlo, Y. Follo Matilde, Stanzani Marta, Parisi Sarah, Clissa Cristina, Mongiorgi Sara, Barraco Marilena and Cocco Lucio, Clinical Impact of Hypomethylating Agents in the Treatment of Myelodysplastic Syndromes, Current Pharmaceutical Design 2016; 22 (16) . https://dx.doi.org/10.2174/1381612822666160310145040
DOI https://dx.doi.org/10.2174/1381612822666160310145040 |
Print ISSN 1381-6128 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4286 |
Call for Papers in Thematic Issues
"Tuberculosis Prevention, Diagnosis and Drug Discovery"
The Nobel Prize-winning discoveries of Mycobacterium tuberculosis and streptomycin have enabled an appropriate diagnosis and an effective treatment of tuberculosis (TB). Since then, many newer diagnosis methods and drugs have been saving millions of lives. Despite advances in the past, TB is still a leading cause of infectious disease mortality ...read more
Current Pharmaceutical challenges in the treatment and diagnosis of neurological dysfunctions
Neurological dysfunctions (MND, ALS, MS, PD, AD, HD, ALS, Autism, OCD etc..) present significant challenges in both diagnosis and treatment, often necessitating innovative approaches and therapeutic interventions. This thematic issue aims to explore the current pharmaceutical landscape surrounding neurological disorders, shedding light on the challenges faced by researchers, clinicians, and ...read more
Emerging and re-emerging diseases
Faced with a possible endemic situation of COVID-19, the world has experienced two important phenomena, the emergence of new infectious diseases and/or the resurgence of previously eradicated infectious diseases. Furthermore, the geographic distribution of such diseases has also undergone changes. This context, in turn, may have a strong relationship with ...read more
Melanoma and Non-Melanoma Skin Cancer Treatment: Standard of Care and Recent Advances
In this thematic issue, we aim to provide a standard of care of the diagnosis and treatment of melanoma and non-melanoma skin cancer. The editor will invite authors from different countries who will write review articles of melanoma and non-melanoma skin cancers. The Diagnosis, Staging, Surgical Treatment, Non-Surgical Treatment all ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
PI3K/Akt/mTOR Pathway Inhibitors in Cancer: A Perspective on Clinical Progress
Current Medicinal Chemistry Tyrosine Kinases as Therapeutic Targets in BCR-ABL Negative Chronic Myeloproliferative Disorders
Current Drug Targets Clinical Significance of Breast Cancer Resistance Protein/ABCG2 in the Management of Malignancy
Clinical Cancer Drugs A Medicinal Chemist’s Perspective Towards Structure Activity Relationship of Heterocycle Based Anticancer Agents
Current Topics in Medicinal Chemistry De Novo Design of New Inhibitor of Mutated Tyrosine-Kinase for the Myeloid Leukemia Treatment
Current Pharmaceutical Design Polo-Like Kinases Inhibitors
Current Medicinal Chemistry Structure-activity Relationship Studies of New Marine Anticancer Agents and their Synthetic Analogues
Current Medicinal Chemistry ANN-QSAR Model for Virtual Screening of Androstenedione C-Skeleton Containing Phytomolecules and Analogues for Cytotoxic Activity Against Human Breast Cancer Cell Line MCF-7
Combinatorial Chemistry & High Throughput Screening Base Excision Repair, the Redox Environment and Therapeutic Implications
Current Molecular Pharmacology Novel Drug-Induced Pulmonary Complications in Cancer Patients You Can Save Life!
Current Respiratory Medicine Reviews MiR-125b Inhibits Cell Proliferation and Induces Apoptosis in Human Colon Cancer SW480 Cells <i>via</i> Targeting STAT3
Recent Patents on Anti-Cancer Drug Discovery Adhesion Dependent Signalling in the Tumour Microenvironment: The Future of Drug Targetting
Current Pharmaceutical Design Economics of Pharmacogenomics: Rethinking Beyond QALYs?
Current Pharmacogenomics and Personalized Medicine Benzimidazoles: An Ideal Privileged Drug Scaffold for the Design of Multitargeted Anti-inflammatory Ligands
Mini-Reviews in Medicinal Chemistry Optimizing Outcomes Following Allogeneic Hematopoietic Progenitor Cell Transplantation in AML: The Role of Hypomethylating Agents
Current Cancer Drug Targets Drug-Induced Thromboembolic Events in Patients with Malignancy
Cardiovascular & Hematological Disorders-Drug Targets Chemistry and Health Effects of Bioactive Compounds in Selected Culinary Aromatic Herbs
Current Nutrition & Food Science Cell Cycle as a Target of Antineoplastic Drugs
Current Pharmaceutical Design In Vivo Optical Imaging in Gene & Cell Therapy
Current Gene Therapy Respiratory Stem Cells and Progenitors: Overview, Derivation, Differentiation, Carcinogenesis, Regeneration and Therapeutic Application
Current Stem Cell Research & Therapy