Treatment of thalassemia major.

In industrialised countries, the use of regular blood transfusions and of c helation therapy with Deferoxamine (DFO) has led to the transformation of t halassemia major from a fatal disease in early childhood to a chronic illne ss associated with prolonged survival. Transfusion regimens maintaining pre transfusion hemoglobin >9-10 g/dl are effective in suppressing erythroid ma rrow expansion. Long term DFO therapy using subcutaneous infusions at least 4-6 d a week have clearly demonstrated major effects on iron overload comp lications. DFO treatment reduces excessive iron and prevents cardiac, hepat ic and endocrine diseases, Nonetheless, compliance is difficult for many pa tients and the cost of DFO limits its use in developing countries. The only oral iron chelating agent that has been investigated extensively is Deferi prone (L1). In France, this oral agent can be administered in patients expe riencing toxic side effects under DFO treatment. Since 1981 more than 1500 bone-marrow transplants have been performed word- wide, mostly in Italy. Allogenic BMT is currently able to cure 85% of thala ssemic children with an available HLA matched sibling donor.