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Myelodysplastic Syndromes: FDA Approves Luspatercept-aamt for Anemia

Myelodysplastic syndrome.

Patients with anemia and lower-risk myelodysplastic syndromes (MDS) are usually treated with erythropoiesis-stimulating agent therapy; however, this treatment method is not always effective in this patient population, and many rely on blood transfusions to treat MDS-related anemia. But now with the recent FDA approval of luspatercept-aamt (Reblozyl®, Celgene Corporation), those patients may no longer be dependent on blood transfusions.

Luspatercept-aamt was recently FDA approved for the treatment of anemia in adult patients with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) who failed an erythropoiesis-stimulating agent and required 2 or more red blood cell units over 8 weeks.

Approval was based on a double-blind, placebo-controlled, phase 3 trial (NCT02631070) that enrolled 229 patients with very-low-risk, low-risk, or intermediate-risk MDS-RS. Prior to enrollment, patients had to have been receiving regular red blood cell transfusions. Participants were randomly assigned to receive either luspatercept at a dose of 1.0 up to 1.75 mg/kg of body weight or placebo, both of which were administered subcutaneously every 3 weeks.

The primary endpoint—transfusion independence—was found in 38% of patients who received luspatercept compared with 13% of patients who were in the placebo group. Treatment-related adverse events included fatigue, diarrhea, asthenia, nausea, and dizziness, and incidence decreased over time.

"Luspatercept reduced the severity of anemia in patients with lower-risk [MDS] with ring sideroblasts who had been receiving regular red-cell transfusions and who had disease that was refractory to or unlikely to respond to erythropoiesis-stimulating agents or who had discontinued such agents owing to an adverse event," study authors conclude.

The recommended starting dose of luspatercept-aamt is 1 mg/kg of body weight, administered by subcutaneous injection once every 3 weeks.

For More Information

Clinicaltrials.gov (2020). A study of luspatercept (ACE-536) to treat anemia due to very low, low, or intermediate risk myelodysplastic syndromes (MEDALIST). NLM identifier: NCT02631070

Fenaux P, Platzbecker U, Mufti GJ, et al (2020). Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med, 382:140-151. DOI:10.1056/NEJMoa1908892

Image Courtesy of TomskiiJA. Licensed under CC BY-SA 3.0

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