title: High incidence of cardiomyopathy in beta-thalassaemia patients receiving regular transfusion and iron chelation: reversal by intensified chelation.
abstract: Cardiac scintigraphy has been performed in 60 beta-thalassaemia major patients aged 8-35 years who received regular blood transfusions and subcutaneous desferrioxamine (DFX) chelation. Fifty-seven showed no clinical, radiological or electrocardiographic evidence of heart disease and 3 had clinically apparent cardiac failure. Twenty-two patients (37%) showed severe cardiac functional impairment defined by a resting left ventricular ejection fraction (LVEF) less than 45% and/or a drop of greater than 12% on stress, while 19 were normal and 19 had a mild abnormality. There was no significant correlation between abnormality of LVEF and age, serum ferritin, number of units transfused, dose and duration of subcutaneous DFX therapy, liver disease or sexual maturation. Non-compliant patients (defined as the use of subcutaneous DFX less than 4 times weekly) generally showed worse cardiac function. Repeat study on 17 patients after 6-28 months of better compliance with subcutaneous or intravenous DFX (using an indwelling catheter) showed a significant overall improvement in LVEF associated with a significant drop in serum ferritin. We conclude that cardiac scintigraphy uncovers a high incidence of cardiac functional abnormality in asymptomatic, well-transfused thalassaemia patients, particularly those poorly compliant with chelation. Those with poor LVEF results should be offered intensive chelation therapy to improve cardiac function.
authors.affiliation: Royal Free Hospital, London, UK.
authors.forename: M A
authors.initials: MA
authors.lastname: Aldouri
authors.forename: B
authors.initials: B
authors.lastname: Wonke
authors.forename: A V
authors.initials: AV
authors.lastname: Hoffbrand
authors.forename: D M
authors.initials: DM
authors.lastname: Flynn
authors.forename: S E
authors.initials: SE
authors.lastname: Ward
authors.forename: J E
authors.initials: JE
authors.lastname: Agnew
authors.forename: A J
authors.initials: AJ
authors.lastname: Hilson
mesh_terms: D000293:Adolescent; D000328:Adult; D001803:Blood Transfusion; D015913:Chelation Therapy / Q000379:methods*; D002648:Child; D003131:Combined Modality Therapy; D003676:Deferoxamine / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D006331:Heart Diseases / Q000000981:diagnostic imaging / Q000209:etiology* / Q000628:therapy*; D006801:Humans; D015994:Incidence; D008297:Male; D011877:Radionuclide Imaging; D013318:Stroke Volume / Q000187:drug effects; D013789:Thalassemia / Q000150:complications* / Q000628:therapy*
chemical_list: D003676:Deferoxamine