Efficacy was demonstrated in a study involving 153 patients
The U.S. Food and Drug Administration (FDA) has approved luspatercept-aamt (REBLOZYL®) for the treatment of anemia in patients: with myelodysplastic syndrome with ring sideroblasts (MDS-RS); or with myelodysplasia / myeloproliferative neoplasia with ring sideroblasts and thrombocytosis (SMD / NMP-RS-T). To be eligible, these patients must also be intolerant or ineligible for treatment with erythropoiesis-stimulating agents or even need red blood cell transfusions.
Efficacy was demonstrated in a randomized, multicenter, double-blind, placebo-controlled study (MEDALIST). Among 153 patients who received luspatercept-aamt, 58 (37.9%) were independent of transfusion for at least 8 weeks, compared with 10 patients (13.2%) who received placebo (difference in treatment 24.6% (p <0.0001).
The most common adverse reactions (> 10%) to luspatercept-aamt are fatigue, headache, musculoskeletal pain, arthralgia, dizziness / vertigo, nausea, diarrhea, cough, abdominal pain, dyspnea and hypersensitivity. The recommended starting dose of luspatercept-aamt is 1 mg / kg once every 3 weeks by subcutaneous injection.
See the full study at:
Platzbecker, U, Komrokji, RS, Fenaux, P, Garcia-Manero G, Mufti GJ, Mikkael A. Sekeres MA et al. A phase 3, randomized, double-blind study of luspatercept (ACE-536) in patients (pts) with Revised International Prognostic Scoring System (IPSS-R) very low- to intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts (RS) who require red blood cell (RBC) transfusions: The MEDALIST trial. JCO, 2016 34:15_suppl, TPS7076-TPS7076
https://clinicaltrials.gov/ct2/show/NCT02631070
