Myelodysplastic Neoplasms (MDS): The Current and Future Treatment Landscape
Document Type
Journal Article
Publication Date
4-3-2024
Journal
Current oncology (Toronto, Ont.)
Volume
31
Issue
4
DOI
10.3390/curroncol31040148
Keywords
MDS; myelodysplastic neoplasm; myelodysplastic syndrome; precision medicine; targeted therapy
Abstract
Myelodysplastic neoplasms (MDS) are a heterogenous clonal disorder of hemopoietic stem cells characterized by cytomorphologic dysplasia, ineffective hematopoiesis, peripheral cytopenias and risk of progression to acute myeloid leukemia (AML). Our understanding of this disease has continued to evolve over the last century. More recently, prognostication and treatment have been determined by cytogenetic and molecular data. Specific genetic abnormalities, such as deletion of the long arm of chromosome 5 (del(5q)), TP53 inactivation and SF3B1 mutation, are increasingly associated with disease phenotype and outcome, as reflected in the recently updated fifth edition of the World Health Organization Classification of Hematolymphoid Tumors (WHO5) and the International Consensus Classification 2022 (ICC 2022) classification systems. Treatment of lower-risk MDS is primarily symptom directed to ameliorate cytopenias. Higher-risk disease warrants disease-directed therapy at diagnosis; however, the only possible cure is an allogenic bone marrow transplant. Novel treatments aimed at rational molecular and cellular pathway targets have yielded a number of candidate drugs over recent years; however few new approvals have been granted. With ongoing research, we hope to increasingly offer our MDS patients tailored therapeutic approaches, ultimately decreasing morbidity and mortality.
APA Citation
Karel, Daniel; Valburg, Claire; Woddor, Navitha; Nava, Victor E.; and Aggarwal, Anita, "Myelodysplastic Neoplasms (MDS): The Current and Future Treatment Landscape" (2024). GW Authored Works. Paper 4768.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/4768
Department
Pathology