Elritercept’s Effect on Transfusion Independence in Patients With Myelodysplastic Syndromes скачать в хорошем качестве

Elritercept’s Effect on Transfusion Independence in Patients With Myelodysplastic Syndromes

Lynette Chee, PhD, Hematologist at The Royal Melbourne Hospital/ Peter MacCallum Cancer Centre, discusses elritercept’s effect on transfusion independence (TI) in patients with myelodysplastic syndromes (MDS), MDS are a group of blood disorders characterized by abnormal development of blood cells within the bone marrow. People with MDS have abnormally low blood cell levels. Signs and symptoms may include dizziness, fatigue, weakness, shortness of breath, bruising and bleeding, frequent infections, and headaches. In some people with MDS, the condition progresses to bone marrow failure or develops into acute leukemia. MDS develops when a cell with a genetic change replicates, and the resulting copies begin to predominate in the bone marrow and suppress healthy stem cells. The genetic change may result from a genetic predisposition, or from injury to the DNA caused by an exposure such as chemotherapy or radiation. In many people with MDS there is no obvious exposure or cause. Results from the phase 2 trial of elritercept treatment in lower risk- (LR) MDS (NCT04419649) were recently presented at the 2025 American Society of Hematology (ASH) meeting. Elritercept is an investigational modified activin receptor type IIA/IgG1 fusion protein designed to bind and block select TGF-β superfamily ligands. As of April 3, 2025, 79 patients treated with elritercept starting at the recommended part 2 dose of 3.75 mg/kg were evaluable for the modified intention-to-treat-24 analysis of transfusion independence (TI) and median exposure was 11.9 months. In the first 24 weeks of treatment, 38.5% of patients achieved TI of 8 or more weeks. In the first 48 weeks, 26.9% achieved TI of 24 or more weeks, with 75.9% maintaining TI at 48 weeks. Sustained TI of 24 weeks or more was observed in 67.3% of patients with TI of 8 weeks or more. TI of 48 weeks or more was achieved in 20.5% of patients over the course of the entire study. Median duration of response was 110.9 weeks among patients who had TI of 8 weeks or more in the first 24 weeks. Median time to TI of 8 weeks or more was 0.4 weeks. Hematologic improvement-erythroid response was measured in 96 patients at the recommended part 2 dose and achieved in 61.5% with median time to response of 3.1 weeks. SF3B1 was the most frequently mutated gene and found in 91% of RS positive patients. Among the modified intention-to-treat 24-week population, TI of 8 weeks or more responses were observed in 52% of patients with and 25% without SF3B1 mutations. For more information on MDS and other rare hematologic conditions, visit https://checkrare.com/diseases/hemato... Chapters: Introduction 00:00 MDS Overview 00:39 Elritercept Clinical Trial 3:19 Take Home Message 7:53