Next-generation therapy for lower-risk MDS.
In: Hematology. American Society of Hematology. Education Program, Jg. 2023 (2023-12-08), Heft 1, S. 59-64
academicJournal
Zugriff:
Myelodysplastic syndromes (MDS) are malignant myeloid neoplasms characterized by ineffective clonal hematopoiesis leading to peripheral blood cytopenia and a variable risk of transformation to acute myeloid leukemia. In lower-risk (LR) MDS, as defined by prognostic scoring systems recently updated with the addition of a mutation profile, therapeutic options aim to reduce cytopenia, mainly anemia. Although options for reducing the transfusion burden have recently been improved, erythropoiesis-stimulating agents (ESAs), lenalidomide, hypomethylating agents, and, more recently, luspatercept have shown efficacy in rarely more than 50% of patients with a duration of response often far inferior to the patient's life expectancy. Nevertheless, several new therapies are currently under investigation aiming at improving cytopenia in patients with LR-MDS, mostly by targeting different biological pathways. Targeting ligands of the transforming growth factor β pathway has led to the approval of luspatercept in LR-MDS with ring sideroblasts or SF3B1 mutation, potentially replacing first-line ESAs in this population. Here, we also discuss the evolving standard of care for the treatment of LR-MDS and explore some of the most promising next-generation agents under investigation.
(Copyright © 2023 by The American Society of Hematology.)
Titel: |
Next-generation therapy for lower-risk MDS.
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Autor/in / Beteiligte Person: | Sébert, M |
Zeitschrift: | Hematology. American Society of Hematology. Education Program, Jg. 2023 (2023-12-08), Heft 1, S. 59-64 |
Veröffentlichung: | Washington, DC : American Society of Hematology ; <i>Original Publication</i>: Washington, DC : The Society,, 2023 |
Medientyp: | academicJournal |
ISSN: | 1520-4383 (electronic) |
DOI: | 10.1182/hematology.2023000520 |
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