Phase II Study of Luspatercept Treatment for Adults with Lower-risk Myelodysplastic Syndromes and Anemia Who Do Not Need Blood Transfusions

What this Trial Is About

This research aims to evaluate the effectiveness of luspatercept in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who have anemia but do not require blood transfusions and have not been treated with erythropoiesis-stimulating agents ESA. It focuses on patients who are transfusion-independent and have symptomatic anemia, studying the response to treatment in this specific group. All participants receive luspatercept administered as a subcutaneous injection at a dose of 1.75 mg per kg of body weight every 21 days. This treatment continues for 24 weeks initially, and for those who respond by week 25, therapy may extend for up to 1.5 years or until loss of response. This is a single-arm, open-label phase II study without a placebo group. Participants will have assessments at regular intervals, including blood tests and bone marrow sampling, to monitor blood cell counts and treatment response. The main outcome is erythroid response measured after eight treatment cycles each cycle is 21 days. Secondary outcomes include the duration of response, time to response, neutrophil and platelet responses, safety monitoring for adverse effects, and quality of life evaluations using validated questionnaires. The study lasts up to 48 months, including long-term follow-up.

Efficacy of Luspatercept in ESA-naive LR-MDS Patients With or Without Ring Sideroblasts Who do Not Require Transfusions