Sustained normalization of cardiac function by chelation therapy in thalassaemia major.
The effect of iron chelation using subcutaneous desferrioxamine on the iron-overloaded heart was studied prospectively over 4 years in 23 asymptomatic patients (mean age 13.2 +/- 5.3 years) with thalassaemia major and transfusion-dependent anaemia. The haemoglobin was maintained greater than 10 g/dl by transfusion and chelation therapy to achieve a negative iron balance. Chelation was closely supervised to ensure compliance. Despite an increase in calculated iron load due to transfusion from 34 +/- 27 g to 63 +/- 28 g, there was a sequential fall in serum ferritin levels from 3148 +/- 1956 ng/ml to 2228 +/- 1825 ng/ml (P less than 0.05). Abnormalities of left ventricular (LV) function, assessed by radionuclide angiography, were present at rest or during exercise in 18 of 23 patients (78%) prior to therapy. Normal LV function was restored in 11 of these 18 patients, five continuing to have abnormal function and two dying. There was a significant increase in resting LVEF from 50 +/- 8% to 57 +/- 6% (P less than 0.01). Peak exercise LVEF rose from 51 +/- 11% to 65 +/- 7% (P less than 0.001). We conclude that the common subclinical abnormalities of LV function induced by iron overload in unchelated patients with thalassaemia major can be reversed with long-term subcutaneous infusions of desferrioxamine.
Freeman
A P
AP
Department of Nuclear Medicine, Prince of Wales Hospital, Sydney, Australia.
Giles
R W
RW
Berdoukas
V A
VA
Talley
P A
PA
Murray
I P
IP
D000293:Adolescent; D000328:Adult; D001315:Australia / Q000453:epidemiology; D001803:Blood Transfusion; D015913:Chelation Therapy; D002648:Child; D003131:Combined Modality Therapy; D003676:Deferoxamine / Q000627:therapeutic use*; D005260:Female; D005293:Ferritins / Q000097:blood; D006331:Heart Diseases / Q000188:drug therapy* / Q000209:etiology; D006333:Heart Failure / Q000209:etiology / Q000401:mortality; D006801:Humans; D007501:Iron; D008297:Male; D005082:Physical Exertion; D013318:Stroke Volume; D013789:Thalassemia / Q000150:complications / Q000188:drug therapy* / Q000628:therapy
D005293:Ferritins; D007501:Iron; D003676:Deferoxamine