Imetelstat improves patient-reported outcomes and quality of life in lower-risk myelodysplastic syndromes: results from the phase III IMerge study

作者：Mikkael A. Sekeres, Amer M. Zeidan, V. Santini, Rami S. Komrokji, Pierre Fenaux, Michael R. Savona, Yazan F. Madanat, David Valcárcel, Antoine Regnault, Kristin Creel, Libo Sun, Ying Wan, Shyamala Navada, Tymara Berry, Faye Feller, Uwe Platzbecker, María Diez‐Campelo, Esther Natalie Olíva · 发表于：Haematologica · 年份：2026 · DOI：10.3324/haematol.2025.288956 · 被引用次数：1 · 研究领域：Acute Myeloid Leukemia Research、Hemoglobinopathies and Related Disorders、Erythropoietin and Anemia Treatment

Red blood cell (RBC) transfusions for anemia associated with lower-risk myelodysplastic syndromes/neoplasms (LR-MDS) often contribute to reduced quality of life (QOL). Thus, reduction in RBC transfusion dependency (TD) is a primary therapeutic goal. Imetelstat is a first-in-class, competitive telomerase inhibitor approved to treat certain adult patients with LRMDS with RBC-TD anemia who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents. In the phase III IMerge study (clinicaltrials.gov identifier: NCT02598661), treatment with imetelstat resulted in clinically meaningful, statistically significant increases in the primary endpoint of ≥8-week RBC transfusion independence (TI) versus placebo. Because patients with LR-MDS experience detrimental effects on numerous facets of QOL (physical, emotional, social, and functional), these exploratory analyses assessed patient-reported outcomes using the Functional Assessment of Chronic Illness Therapy-Fatigue, Quality of Life in Myelodysplasia Scale, and Functional Assessment of Cancer Therapy-Anemia questionnaires as part of the phase III IMerge study. Nominal P values were reported. Fewer imetelstat-treated patients experienced deterioration in fatigue levels and more imetelstat-treated patients experienced sustained improvement in fatigue levels and QOL versus placebo. In the imetelstat group, 8-week, 24-week, and 1-year RBC-TI responders had sustained improvements in predefined signi...