Imetelstat for anemia in lower-risk myelodysplastic syndromes: A summary from the Institute for Clinical and Economic Review’s California Technology Assessment Forum

CLINICAL EFFECTIVENESSWe focused on 2 specific comparisons in this assessment: (1) imetelstat vs best supportive care for patients with lower-risk MDS who are transfusion dependent despite ESAs (overall low, low, or intermediate) or higher risk (high or very high) categories. [4][5]5][6] In lower-risk MDS, approximately 40% of patients require blood transfusions to manage their anemia.Erythropoiesisstimulating agents (ESAs) are the standard first-line treatment, but some patients do not respond, and others may lose efficacy over time. 7Luspatercept (Reblozyl; Bristol Myers Squibb) has been approved by the US Food and Drug Administration (FDA) as a first-line treatment for anemia in patients with lower-risk MDS as well as use after ESA failure in the approximately 35% of patients with RS-positive disease (where luspatercept has been found to be particularly effective). 8or patients with the del(5q) subtype, which makes up approximately 10% of cases, lenalidomide is an effective treatment option. 9Imetelstat (Rytelo; Geron Corporation) was approved by the FDA on June 6, 2024, as a treatment for transfusion-dependent anemia in patients with lower-risk MDS who have not responded to, lost response to, or are ineligible for ESAs. 10 The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate the clinical and economic outcomes of imetelstat for anemia in lower-risk MDS.This report presents a summary of our findings and highlights the key policy recommendations Imetelstat for anemia in lower-risk myelodysplastic syndromes: