hematology and oncology
Thalassemias
Treatment of Thalassemias
Often red blood cell transfusion, with or without iron chelation therapy Splenectomy if splenomegaly is present Allogeneic stem cell transplantation if possible Luspaterceptfor treatment of transfusion-dependent beta thalassemia In patients with alpha-thalassemia trait or beta-thalassemia trait, no treatment is needed. In Hb H disease, splenectomy may be helpful if anemia is severe or splenomegaly is present. Patients with beta-thalassemia intermedia should receive as few transfusions as possible to avoid iron overload. However, suppression of abnormal hematopoiesis by periodic Red blood cells (RBCs) may be valuable in severely affected patients. In beta thalassemia major, give transfusions as needed to maintain the hemoglobin level around 9 to 10 g/dL (90 to 100 g/L) and avoid severe clinical manifestations. To prevent or delay complications due to iron overload, excess (transfusional) iron must be removed (eg, via chronic Treatment ). Chelation therapy is generally initiated when serum ferritin levels are > 1000 ng/mL ( is an option for treatment in transfusion-dependent patients ( Treatment reference ). Allogeneic Hematopoietic Stem Cell Transplantation is the only curative option and should be considered in all patients. 1.Cappellini MD, Viprakasat V, Taher A, et al: A phase 3 trial ofluspaterceptin patients with transfusion-dependent β-thalassemia.N Engl J Med382(13):1219–1231, 2020. doi: 10.1056/NEJMoa1910182 