Introduction: The aim of treatment in very low-, low- and intermediate-1-risk myelodysplastic syndrome (MDS) is mainly to relieve symptoms due to cytopenias. Only a few therapeutic drugs are currently available, but novel drugs are under clinical investigations. In this setting, imetelstat, a telomerase inhibitor, is a promising new agent.
Areas Covered: This review summarizes promising emerging strategies using imetelstat for the treatment of lower-risk MDS.
Expert Opinion: Favorable results were demonstrated in the IMerge phase 3 clinical trial using imetelstat in transfusion-dependent patients with lower-risk MDS relapsed or refractory to erythropoiesis-stimulating agents (ESAs). This study led to imetelstat approval by the United States Food and Drug Administration (FDA) in June 2024.
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Research and analysis of differential gene expression in CD34 hematopoietic stem cells in myelodysplastic syndromes.
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Inflammatory Signatures in VEXAS Syndrome, Myelodysplasia Cutis, and Sweet Syndrome.
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March 2025
Service de Dermatologie et Allergologie, Faculté de Médecine, Sorbonne Université, Hôpital Tenon, Assistance Publique-Hôpitaux de Paris, Paris, France.
Importance: VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) is a monogenic disease caused by UBA1 somatic variants in hematopoietic progenitor cells, mostly involving adult men. It is associated with inflammatory-related symptoms, frequently involving the skin and hematological disorders. Recently described myelodysplasia cutis (MDS-cutis) is a cutaneous manifestation of myelodysplasia in which clonal myelodysplastic cells infiltrate the skin.
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Department of Clinical Haematology, Christian Medical College, Vellore, India.
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