[Program of hypertransfusion and chelation with desferrioxamine in 10 patients with thalassemia major].
OBJECTIVE
Thalassaemia maior is an inherited severe anaemia due to abnormal haemoglobin synthesis. Hypertransfusion therapy is based on the reduction of ineffective erythropoiesis and improvement of the anaemia. To prevent iron overload, continuous chelation therapy was performed with desferrioxamine (DFO). The efficacy of such management in 10 thalassaemia maior patients followed-up for 11 years has been evaluated.


METHODS
Packed red-cell transfusions were administered to maintain haemoglobin rates over 100 g/L; 40 mg/Kg DFO was given five days a week subcutaneously. The mean age at the start of such treatment was 9.3 years. Clinical problems due to iron overload were evaluated, such as heart function, growth and development, endocrinopathies, along with DFO toxicity and complications of transfusion (positive indirect antiglobulin test (IAT), hepatitis, HIV infection).


RESULTS
Two patients died of heart haemosiderosis. The mean ferritin values at the end of the study were around 3.031 mg/mL. Heart dysfunction was seen in 2 cases; 2 patients had retarded growth, decreased IGF-1 being found in both; 3 cases had hypogonadotropic hypogonadism, and 2 had clinical hypoparathyroidism. Bilateral opacification of the eye lens appeared in one patients as a consequence of prolonged DFO therapy. Positive IAT was found in 3 cases, and one patient developed positive hepatitis C serology.


CONCLUSIONS
Control of iron deposits by iron chelators can prevent the damages of iron overload. Adequate accomplishment of programmes may be difficult, especially in children.
Albo
C
C
Servicio de Hematología y Hemoterapia, Complexo Hospitalario Xeral-Cíes, Vigo.
Cabrera
J
J
Dios
A
A
Castro
M
M
Ares
C
C
Constenla
I
I
López
D
D
D000293:Adolescent; D001803:Blood Transfusion; D015913:Chelation Therapy; D002648:Child; D003131:Combined Modality Therapy; D003298:Coombs Test; D003676:Deferoxamine / Q000627:therapeutic use*; D005069:Evaluation Studies as Topic; D005260:Female; D005293:Ferritins / Q000032:analysis; D006130:Growth Disorders / Q000097:blood / Q000209:etiology; D006526:Hepatitis C / Q000635:transmission; D006801:Humans; D007501:Iron / Q000097:blood; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000209:etiology / Q000517:prevention & control*; D008297:Male; D013156:Splenectomy; D065227:Transfusion Reaction; D017086:beta-Thalassemia / Q000097:blood / Q000150:complications / Q000601:surgery / Q000628:therapy*
D007502:Iron Chelating Agents; D005293:Ferritins; D007501:Iron; D003676:Deferoxamine