5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

Inclusion Criteria

• •Thalassemia major with progressive disease or complications of iron overload despite traditional transfusion and iron chelation therapy
• •Thalassemia major in which standard transfusion therapy or iron chelation therapy is contraindicated
• •ECOG performance status must be less than or equal to 2
• •NYHA less than or equal to class II status
• •Progressive disease is defined as 1) an increasing transfusion requirement or difficulty in maintenance of hemoglobin levels greater than 7g/dl as a consequence of autologous or allogeneic antibodies or 2) increasing extramedullary hematopoiesis causing compression phenomena.
• •Complications of iron overload despite iron chelation therapy is defined as difficulty in achieving negative iron balance when complications of iron overload exist. Complications of iron overload include heart failure, or decreased cardiac ejection fraction, endocrinopathy and evidence of progressive liver dysfunction.