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Opinion|Videos|December 12, 2025

Imetelstat as a Treatment Option for Low-risk MDS

Discover how a metal statin offers a promising backup treatment for MDS patients, enhancing transfusion outcomes while requiring careful monitoring.

metelstat, a telomerase inhibitor, offers a novel treatment option for patients with low-risk MDS who are heavily transfusion-dependent and have limited responses to conventional therapies. In clinical practice, it is often considered for patients who are refractory to ESAs and, in some cases, luspatercept, particularly when reducing transfusion burden is a key priority. Its mechanism targets the underlying malignant clone, offering potential disease-modifying benefits beyond symptomatic anemia management. Conversely, imetelstat is generally avoided in patients with adequate response to ESAs or luspatercept, those with significant comorbidities, or individuals at higher risk for cytopenias or hepatic toxicity. In these scenarios, ESAs or luspatercept remain preferred due to favorable safety profiles, ease of administration, and proven efficacy in improving hemoglobin levels or achieving transfusion independence. Therapy selection is therefore individualized based on patient characteristics, prior treatment r

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