From the FDA Drug Label
The primary efficacy study, Study 1 (NCT00061750), was a multicenter, open-label, randomized, active-comparator control study to compare deferasirox tablets for oral suspension and deferoxamine in patients with beta-thalassemia and transfusional hemosiderosis Patients greater than or equal to 2 years of age were randomized in a 1:1 ratio to receive either oral deferasirox tablets for oral suspension at starting doses of 5,10,20, or 30 mg per kg once daily or deferoxamine at starting doses of 20 to 60 mg per kg for at least 5 days per week based on LIC at baseline Reduction of LIC and serum ferritin was observed with deferasirox tablet for oral suspension doses of 20 to 30 mg per kg per day. Therefore, a starting dose of 20 mg per kg per day is recommended [see Dosage and Administration (2. 1)].
The treatment for Beta-Thalassemia (Anemia Mediterranean) is deferasirox at a starting dose of 20 mg per kg per day.
- The recommended dose is based on the patient's liver iron concentration (LIC).
- Deferasirox has been shown to reduce LIC and serum ferritin levels in patients with beta-thalassemia and transfusional hemosiderosis 1.
From the Research
The treatment for Beta-Thalassemia, also known as Anemia Mediterranean, should prioritize regular blood transfusions and iron chelation therapy to prevent complications and improve quality of life, as supported by the most recent study 2. The cornerstone of therapy for moderate to severe Beta-Thalassemia is regular transfusions of packed red blood cells, typically given every 2-4 weeks to maintain hemoglobin levels above 9-10 g/dL. This approach prevents complications and supports normal growth and development.
- Key aspects of treatment include:
- Iron chelation therapy using medications such as deferasirox (20-40 mg/kg/day orally), deferiprone (75-100 mg/kg/day divided into three doses), or deferoxamine (20-40 mg/kg/day subcutaneously over 8-24 hours) to prevent iron overload, as discussed in 3.
- Hydroxyurea (10-20 mg/kg/day) may help increase fetal hemoglobin production in some patients.
- Folic acid supplementation (1 mg daily) supports increased erythropoiesis.
- For severe cases, hematopoietic stem cell transplantation offers the only curative option, with best outcomes when performed before complications develop, as noted in the general management of the disease.
- Gene therapy approaches are emerging treatments that may provide functional correction of the genetic defect.
- Comprehensive care, including regular monitoring of iron levels, cardiac and liver function, bone density, and endocrine status, is necessary to manage complications effectively, as emphasized in 2. The most recent and highest quality study 2 highlights the importance of adequate iron chelation therapy and regular assessment of adherence, adverse effects, and iron burden to optimize patient outcomes.