[Therapeuetic management of patients with thalassemia major].
In industrialised countries, the use of regular blood transfusions and of chelation therapy with Deferoxamine (DFO) has led to the transformation of thalassemia major from a fatal disease in early childhood to a chronic illness associated with prolonged survival. Transfusion regimens maintaining pretransfusion hemoglobin > 9-10 g/dl are effective in suppressing erythroid marrow expansion. Long term DFO therapy using subcutaneous infusions at least 4-6 d a week have clearly demonstrated major effects on iron overload complications. DFO treatment reduces excessive iron and prevents cardiac, hepatic and endocrine diseases. Nonetheless, compliance is difficult for many patients and the cost of DFO limits its use in developing countries. The only oral iron chelating agent that has been investigated extensively is Deferiprone (L1). In France, this oral agent can be administered in patients experiencing 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 thalassemic children with an available HLA matched sibling donor.
Thuret
I
I
Service d'hématologie pédiatrique, Hôpital de la Timone, Marseille, France.
D001803:Blood Transfusion; D016026:Bone Marrow Transplantation; D003676:Deferoxamine / Q000627:therapeutic use; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D013156:Splenectomy; D017086:beta-Thalassemia / Q000628:therapy*
D007502:Iron Chelating Agents; D003676:Deferoxamine