title: Effectiveness of deferiprone in transfusion-independent beta-thalassemia/HbE patients.
abstract: Beta-thalassemia/HbE (beta-thal/HbE) is a thalassemia intermedia (TI) which encompasses a broad spectrum of severity. Here, we used deferiprone (DFP) as an iron chelating agent in TI patients receiving intermittent blood transfusion who are asymptomatic for cardiovascular disease in order to evaluate the effectiveness in iron overload and reduce the possibility of cardiovascular complications. Thirty transfusion-independent beta-thal/HbE patients with iron overload were treated with DFP for 1 year. Hematological, biochemical, oxidative stress and echocardiographic parameters were determined. Serum ferritin, non-transferrin-bound iron, and malondialdehyde decreased significantly (P<0·05) after 1-year treatment with DFP. For echocardiographic results, mean pulmonary arterial pressure and pulmonary vascular resistance were diminished significantly (P<0·05). All those parameters were still improved after subgroup analysis was done for the high ferritin group (>2500 ng/ml). DFP therapy alone improved iron overload and oxidative stress and compliance was good. We propose that prevention of pulmonary hypertension is also possible for TI undergoing intermittent blood transfusion.
authors.affiliation: Department of Biochemistry, Faculty of Medicine, Department of Internal Medicine, Siriraj Hospital, Mahidol University, Thailand.
authors.forename: Krittapoom
authors.initials: K
authors.lastname: Akrawinthawong
authors.forename: Nithima
authors.initials: N
authors.lastname: Chaowalit
authors.forename: Thanasaporn
authors.initials: T
authors.lastname: Chatuparisuth
authors.forename: Noppadol
authors.initials: N
authors.lastname: Siritanaratkul
mesh_terms: D000293:Adolescent; D000328:Adult; D018376:Cardiovascular Abnormalities / Q000517:prevention & control; D000077543:Deferiprone; D015150:Echocardiography, Doppler; D005260:Female; D005293:Ferritins / Q000097:blood; D005500:Follow-Up Studies; D006446:Hemoglobin E; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D008875:Middle Aged; D018384:Oxidative Stress; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D065227:Transfusion Reaction; D016896:Treatment Outcome; D055815:Young Adult; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000628:therapy
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D006446:Hemoglobin E