FOR adult patients suffering from anemia due to beta thalassemia, the European Commission has just expanded the indications of luspatercept. The drug had previously been approved, also in Italy, to treat anemia associated with transfusion-dependent beta-thalassemia and very low, low and intermediate risk transfusion-dependent myelodysplastic syndromes. Now, however, it can also be used for the treatment of adult patients with anemia associated with non-transfusion-dependent beta-thalassemia (NTD).
Beta-thalassemia and myelodysplastic syndromes: Innovative therapy approved that reduces blood transfusions
by Irma D’Aria
What is non-transfusion-dependent beta-thalassemia?
Beta-thalassemia is an inherited blood disorder caused by a genetic defect in the hemoglobin molecule. The condition is associated with decreased production of healthy red blood cells, often leading to severe anemia – a condition that can be debilitating and can lead to other serious health problems. Patients with non-transfusion-dependent beta-thalassemia are those who do not require regular lifelong transfusions to survive, but who may still develop a variety of anemia-related complications, and who require occasional or even frequent transfusions, usually for periods of time defined. For this reason specific treatments are needed even in these cases.
Myelodysplasias: with new personalized therapy, 48% of patients transfusion-free for 8 weeks
by Irma D’Aria
Lo studio BEYOND
The European Commission approval is based on results from the Phase 2 BEYOND study, which evaluated the efficacy and safety of luspatercept versus placebo in 145 adults with non-transfusion-dependent beta-thalassemia. According to the results of this study, the drug was effective in 77% of cases in increasing the concentration of hemoglobin above the threshold value of 1 g/dL. “Today’s announcement is important news in Europe for patients with anemia associated with non-transfusion-dependent beta-thalassemia who are waiting for new therapeutic options to reduce these problems”.