Advances in the management of higher-risk myelodysplastic syndromes: future prospects | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

Advances in the management of higher-risk myelodysplastic syndromes: future prospects

Journal Title: 
Leukemia & Lymphoma
Primary Author: 
Gener-Ricos G
Author(s): 
Gener-Ricos G, Rodriguez-Sevilla JJ, Urrutia S, Bataller A, Bazinet A, Garcia-Manero G
Original Publication Date: 
Monday, September 30, 2024
Bone Marrow Disease(s): 

Higher-risk myelodysplastic syndromes (HR-MDS) are defined using a number of prognostic scoring systems that include the degree of cytopenias, percentage of blasts, cytogenetic alterations, and more recently genomic data. HR-MDS encompasses characteristics such as progressive cytopenias, increased bone marrow blasts, unfavorable cytogenetics, and an adverse mutational profile. Survival is generally poor, and patients require therapy to improve outcomes. Hypomethylating agents (HMAs), such as azacitidine, decitabine, and more recently, oral decitabine/cedazuridine, are the only approved therapies for HR-MDS. These are often continued until loss of response, progression, or unacceptable toxicity. Combinations including an HMA plus other drugs have been investigated but have not demonstrated better outcomes compared to single-agent HMA. Moreover, in a disease of high genomic complexity such as HR-MDS, therapy targeting specific genomic abnormalities is of interest. This review will examine the biological underpinnings of HR-MDS, its therapeutic landscape in the frontline and relapsed settings, as well as the impact of hematopoietic stem cell transplantation, the only known curative intervention for this disease.