On April 3, 2020, the Food and Drug Administration approved luspatercept-aamt for the treatment of anaemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Efficacy was demonstrated in the MEDALIST trial (NCT02631070), a randomised, multi-center, double-blind, placebo-controlled trial in 229 patients with IPSS-R very low, low, or intermediate-risk myelodysplastic syndromes who had ring sideroblasts and required RBC transfusions (2 or more RBC units over 8 weeks).
Patients were randomised 2:1 to luspatercept-aamt or placebo.
All patients received best supportive care, which included RBC transfusions.
The main efficacy endpoint in MDS-RS and MDS-RS-T was the proportion of patients who were RBC-transfusion independent (RBC-TI), defined as the absence of any RBC transfusion during any consecutive 8-week period between Weeks 1 and 24.
Of the 153 patients who received luspatercept-aamt, 58 (37.9%, 95% CI: 30.2, 46.1) were RBC-TI for at least 8 weeks, compared to 10 patients (13.2%, 95% CI: 6.5, 22.9) who received placebo (treatment difference 24.6% (95% CI: 14.5, 34.6; p<0.0001.)
The most common (>10%) adverse reactions to luspatercept-aamt are fatigue, headache, musculoskeletal pain, arthralgia, dizziness/vertigo, nausea, diarrhoea, cough, abdominal pain, dyspnea, and hypersensitivity.
The recommended starting dose of luspatercept-aamt is 1 mg/kg once every 3 weeks by subcutaneous injection.
Review haemoglobin results prior to each administration.