{"title":"Examining the safety and efficacy of imetelstat in low-risk myelodysplastic syndrome.","authors":"Xavier Thomas","doi":"10.1080/14656566.2025.2471518","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Areas covered: </strong>This review summarizes promising emerging strategies using imetelstat for the treatment of lower-risk MDS.</p><p><strong>Expert opinion: </strong>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.</p>","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":""},"PeriodicalIF":2.5000,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Opinion on Pharmacotherapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/14656566.2025.2471518","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
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.
期刊介绍：
Expert Opinion on Pharmacotherapy is a MEDLINE-indexed, peer-reviewed, international journal publishing review articles and original papers on newly approved/near to launch compounds mainly of chemical/synthetic origin, providing expert opinion on the likely impact of these new agents on existing pharmacotherapy of specific diseases.
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