title: Clinical trial of deferiprone iron chelation therapy in beta-thalassaemia/haemoglobin E patients in Thailand.
abstract: Nine patients with either beta-thalassaemia/haemoglobin E (7) or homozygous beta-thalassaemia (2) not requiring regular transfusions were treated with the oral iron chelator, deferiprone 25-50 mg/kg/d for between 17 and 86 weeks (mean 49 weeks). There were significant decreases in serum ferritin (initial mean +/- standard deviation 2168 +/- 1142, final 418 +/- 247 micro g/l; t-test for paired samples, P = 0.005), hepatic iron (initial 20.3 +/- 6.26, final 11.7 +/- 4.83 mg/g/dry weight; P = < 0.02), red cell membrane iron (initial 76.2 +/- 3.64, final 7.2 +/- 0.56 mmol/mg protein; P = < 0.0005) and serum non-transferrin bound iron (initial 9.0 +/- 0.56, final 5.9 +/- 0.89 micro mol/l; P = < 0.0005). There was also a significant rise in serum erythropoietin (initial 240 +/- 195.1, final 433.2 +/- 269.2 U/l; P = 0.034). The haemoglobin level rose in three patients and transfusion requirements were reduced substantially in four patients. Serum thiobarbituric acid reactive substance (TBARS) also fell in six of eight patients. Patients generally improved clinically, with weight gain observed. Side-effects were mild and included gastrointestinal symptoms (6) and arthralgia (1), not requiring withdrawal of the drug. One patient died at 17 weeks of therapy as a result of an intercurrent infection. His neutrophil count was normal. We conclude that deferiprone is an effective, well-tolerated iron chelator for patients with thalassaemia intermedia. Further studies are needed to determine the optimum dose and length of treatment needed to reduce iron burden to a safe level in these patients.
authors.affiliation: Thalassaemia Research Centre, Institute of Science and Technology for Research and Development, Mahidol University, Salaya Campus, Salaya, UK.
authors.forename: Pensri
authors.initials: P
authors.lastname: Pootrakul
authors.forename: Pornpan
authors.initials: P
authors.lastname: Sirankapracha
authors.forename: Jongkal
authors.initials: J
authors.lastname: Sankote
authors.forename: Udom
authors.initials: U
authors.lastname: Kachintorn
authors.forename: Wanna
authors.initials: W
authors.lastname: Maungsub
authors.forename: Kosit
authors.initials: K
authors.lastname: Sriphen
authors.forename: Kleonphan
authors.initials: K
authors.lastname: Thakernpol
authors.forename: Kanit
authors.initials: K
authors.lastname: Atisuk
authors.forename: Suthat
authors.initials: S
authors.lastname: Fucharoen
authors.forename: Udom
authors.initials: U
authors.lastname: Chantraluksri
authors.forename: Oded
authors.initials: O
authors.lastname: Shalev
authors.forename: A Victor
authors.initials: AV
authors.lastname: Hoffbrand
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000077543:Deferiprone; D004910:Erythrocyte Membrane / Q000737:chemistry; D004921:Erythropoietin / Q000032:analysis; D005260:Female; D005293:Ferritins / Q000097:blood; D006446:Hemoglobin E; D006801:Humans; D007501:Iron / Q000032:analysis; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008099:Liver / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D011728:Pyridones / Q000627:therapeutic use*; D011990:Receptors, Transferrin / Q000097:blood; D013785:Thailand; D017392:Thiobarbituric Acid Reactive Substances / Q000032:analysis; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000378:metabolism
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D011990:Receptors, Transferrin; D017392:Thiobarbituric Acid Reactive Substances; D004921:Erythropoietin; D000077543:Deferiprone; D005293:Ferritins; D006446:Hemoglobin E; D007501:Iron