Conditions: Transfusion-dependent β-thalassemia Patients Cardiac Iron Overload
Keywords: Severe cardiac iron overload deferasirox β-thalassaemia cardiac dysfunction
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 32
Healthy volunteers: no

Brief summary:
The primary efficacy endpoint of this interventional study was to evaluate the number of patients achieving a complete response (CR), defined as patients switching from intensive deferasirox -DFO treatment, at any time point during the 24 months of study, to deferasirox monotherapy based on improvement in the cardiac magnetic resonance imaging (MRI) T2\* value to \>10ms, and continue to maintain their MRI T2\* to values \>10 msec.

Detailed description:
This study was planned to recruit 52 transfusion-dependent β-thalassemia patients with severe cardiac iron overload. However only 13 patients participated in the study during a 3 year and 5 month timeframe. The study was terminated due to the slow enrollment rate due to scarcity of the patient population with severe cardiac iron overload.

Arms:
arm 1: During Phase A, the induction treatment at entry, participants received Deferasirox -DFO combination. During Phase B, when participants transitioned to less intensive chelation therapy, participants received Deferasirox monotherapy.

Interventions: intervention 1: Deferasirox intervention 2: Deferoxamine (DFO)
Intervention descriptions: intervention 1: 20-40 mg/kg/day orally, once daily intervention 2: 40 mg/kg/day subcutaneous (sc) infusion, 3-4 days per week

Protocol document: