Conditions: Beta-thalassemia Major Hemosiderosis Iron Overload Rare Anemia
Keywords: Transfusional hemosiderosis Beta-thalassemia major Deferasirox iron overload rare anemia iron overload due to transfusion
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 233
Healthy volunteers: no

Brief summary:
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available. To evaluate the long-term safety and efficacy of deferasirox by measuring treatment success, change in liver iron content (LIC) and change in serum ferritin levels. Safety was mainly assessed by incidence of adverse events (AEs)and clinically significant lab parameters.

Detailed description:
Iron accumulation is an inevitable consequence of chronic blood transfusions and results in serious complications in the absence of chelation treatment to remove excess iron. Deferasirox (Exjade, ICL670) is an oral chelator with high iron-binding potency and selectivity. This extension study aimed at collecting efficacy and safety data during 2 years of treatment with deferasirox in the extension study or until deferasirox became commercially available in the countries where the centers were located, whichever came first. The population comprised of β-thalassemia patients with transfusional hemosiderosis who could not be satisfactorily treated with deferoxamine or deferiprone.

Arms:
arm 1: Deferasirox was given orally once daily (10 to 20 mg/kg) to participants 2 years and older based on participant's body weight.

Interventions: intervention 1: Deferasirox
Intervention descriptions: intervention 1: Deferasirox was administered orally once daily. Deferasirox was available as 125 mg, 250 mg, and 500 mg tablets.

Protocol document: