On 3 April 2020, the US Food and Drug Administration (FDA) approved luspatercept-aamt (REBLOZYL, Celgene Corporation) 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, multicentre, double-blind, placebo-controlled trial in 229 patients with Revised International Prognostic Scoring System 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% confidence interval [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, dyspnoea, 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.
Full prescribing information for REBLOZYL is available here.
FDA granted luspatercept-aamt orphan drug and fast track designation.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System.