TY - JOUR
T1 - Advances in the management of higher-risk myelodysplastic syndromes
T2 - future prospects
AU - Gener-Ricos, Georgina
AU - Rodriguez-Sevilla, Juan Jose
AU - Urrutia, Samuel
AU - Bataller, Alex
AU - Bazinet, Alexandre
AU - Garcia-Manero, Guillermo
N1 - Publisher Copyright:
© 2024 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2024
Y1 - 2024
N2 - 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.
AB - 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.
KW - Higher-risk myelodysplastic syndromes
KW - allogeneic stem cell transplant
KW - hypomethylating agents
KW - targeted therapies
UR - http://www.scopus.com/inward/record.url?scp=85192378735&partnerID=8YFLogxK
U2 - 10.1080/10428194.2024.2344061
DO - 10.1080/10428194.2024.2344061
M3 - Review article
C2 - 38712556
AN - SCOPUS:85192378735
SN - 1042-8194
VL - 65
SP - 1233
EP - 1244
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
IS - 9
ER -