Apotransferrin in Patients With β-thalassemia

Efficacy and Safety of Human Apotransferrin in Patients With β-thalassemia Intermedia

The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced transfusion dependency.

Inclusion Criteria:

• Non-transfusion dependent β-thalassemia intermedia, defined as patients with microcytic anaemia in combination with an elevated HbA2 (>2.5%) and a haemoglobin of <6.2 mmol/L, or transfusion dependent β-thalassemia treated with a regular transfusion schedule.
• Age above≥ 17 years.
• Adequate renal and hepatic function tests
• WHO performance 0, 1 or 2.
• Signed informed consent.

Exclusion Criteria:

• Known with allergic reactions against human plasma or plasma products.
• Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease).
• Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias.
• Pregnant or lactating females.
• Known with IgA deficiency with anti-IgA antibodies