In-depth [randomized-controlled trial]: Between Sept 11, 2019, and Oct 13, 2021, 317 patients were screened for eligibility across 118 tertiary sites in 17 countries. Included were patients ≥ 18 years with red blood cell transfusion-dependent LR-MDS ineligible for or unresponsive to ESAs. Altogether, 178 patients (118 in imetelstat and 60 in placebo) were included in the study. The primary outcome of RBC-TI at 8 weeks was significantly higher in imetelstat versus placebo (40% vs. 15%, p=0.0008). However, the secondary outcome of grade 3-4 treatment-emergent adverse events was also higher for imetelstat than placebo (91% vs. 47%), with neutropenia (68%) and thrombocytopenia (62%) being most common. Overall, findings from this study suggest that imetelstat provides a novel mechanism of action, offering durable transfusion independence and disease-modifying activity for heavily transfused LR-MDS patients unresponsive to or ineligible for erythropoiesis-stimulating agents.

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