From the Guidelines
Treatment for adult beta thalassemia major should include regular blood transfusions every 2-4 weeks, accompanied by iron chelation therapy with deferoxamine (Desferal, 20-60 mg/kg/day subcutaneously over 8-24 hours) to prevent iron overload and reduce the risk of cardiac complications, as recommended by the American Heart Association 1. The goal of treatment is to maintain hemoglobin levels above 9-10 g/dL and prevent iron overload, which can damage organs.
- Regular blood transfusions every 2-4 weeks are the cornerstone of treatment for beta thalassemia major.
- Iron chelation therapy with deferoxamine is recommended to prevent iron overload and reduce the risk of cardiac complications.
- Folic acid supplementation (1 mg daily) is also recommended to support erythropoiesis.
- For beta thalassemia intermedia, transfusions may be needed only during periods of increased anemia, such as during infections or pregnancy.
- Hydroxyurea (starting at 10-15 mg/kg/day) may help increase fetal hemoglobin production in some patients.
- Splenectomy might be considered for those with significant splenomegaly causing mechanical symptoms or increased transfusion requirements.
- Allogeneic hematopoietic stem cell transplantation remains the only curative option, particularly for younger patients with HLA-matched siblings.
- Newer therapies include luspatercept (Reblozyl, 1 mg/kg subcutaneously every 3 weeks) for transfusion-dependent patients and gene therapy approaches that are showing promise in clinical trials.
- Regular monitoring of iron levels, cardiac and liver function, bone density, and endocrine status is essential for all patients with beta thalassemia to manage complications effectively, as cardiac iron overload is a major predictor of heart failure in these patients 1.
From the FDA Drug Label
Deferiprone tablets are indicated for the treatment of transfusional iron overload in adult patients with thalassemia syndromes when current chelation therapy is inadequate The treatment for adult beta thalassemia is deferiprone when current chelation therapy is inadequate, as it is indicated for the treatment of transfusional iron overload in adult patients with thalassemia syndromes 2.
- The recommended starting oral dosage of deferiprone tablets is 75 mg/kg/day (actual body weight), in three divided doses per day.
- Another option is deferasirox, which was evaluated in patients with beta-thalassemia, but the provided text does not explicitly state its indication for adult beta thalassemia treatment 3.
From the Research
Treatment Options for Adult Beta Thalassemia
- The treatment for beta thalassemia major includes regular blood transfusions, iron chelation therapy, and in some cases, bone marrow transplantation or gene therapy 4
- Iron chelation therapy is essential to prevent cardiac failure and other complications due to iron accumulation 5, 6
- A combination of desferrioxamine (DFX) and deferiprone (L1) has been shown to be effective in reducing iron overload in patients with beta thalassemia major 5
- Deferasirox (Exjade) has also been found to be effective in modulating iron overload in patients with beta thalassemia intermedia 7
Management of Iron Overload
- Iron overload is a major cause of morbidity and mortality in patients with beta thalassemia major, and successful iron chelation is essential for optimal management 5
- Regular monitoring of serum ferritin levels is necessary to assess the effectiveness of iron chelation therapy 5, 7
- A significant reduction in serum ferritin levels has been observed with iron chelation therapy using DFX and L1 in patients with beta thalassemia major 5 and deferasirox in patients with beta thalassemia intermedia 7
Challenges in Treatment
- Patients with beta thalassemia major often have limited access to regular and safe blood transfusions, particularly in resource-poor countries 6
- Alloimmunization and transfusion-transmitted infections are significant risks for patients with beta thalassemia major, highlighting the need for improved blood transfusion services and safety practices 6