The National Comprehensive Cancer Network (NCCN) has updated its Clinical Practice Guidelines for myelodysplastic neoplasms (MDS) to recommend imetelstat for treating symptomatic anemia in patients with lower-risk MDS.
In patients who are negative for ring sideroblasts (RS) and have lower-risk MDS with symptomatic anemia, imetelstat is suggested as a category 1 second-line treatment for patients with serum erythropoietin (EPO) levels ≤500 mU/mL after treatment with erythropoiesis-stimulating agents (ESAs) or luspatercept. Additionally, imetelstat is recommended as a category 2A first-line treatment for patients with serum EPO levels >500 mU/mL who are unlikely to respond to immunosuppressive therapy.
In patients who are RS-positive and have lower-risk MDS with symptomatic anemia, imetelstat is recommended as a category 1 second-line treatment for patients with serum EPO levels ≤500 mU/mL after luspatercept. It is also recommended as a category 2A first-line treatment for those with serum EPO levels >500 mU/mL
In June 2024, the US Food and Drug Administration granted approval to imetelstat based on data from the pivotal phase III IMerge trial.
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