Clinical trial of deferiprone iron chelation therapy in β‐thalassaemia/haemoglobin E patients in Thailand

Nine patients with either β‐thalassaemia/haemoglobin E (7) or homozygous β‐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...

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Bibliographic Details
Published in:	British journal of haematology Vol. 122; no. 2; pp. 305 - 310
Main Authors:	Pootrakul, Pensri, Sirankapracha, Pornpan, Sankote, Jongkal, Kachintorn, Udom, Maungsub, Wanna, Sriphen, Kosit, Thakernpol, Kleonphan, Atisuk, Kanit, Fucharoen, Suthat, Chantraluksri, Udom, Shalev, Oded, Hoffbrand, A. Victor
Format:	Journal Article
Language:	English
Published:	Oxford, UK Blackwell Science Ltd 01-07-2003 Blackwell
Subjects:	Administration, Oral Adult Anemias. Hemoglobinopathies beta-Thalassemia - drug therapy beta-Thalassemia - metabolism Biological and medical sciences Blood. Blood coagulation. Reticuloendothelial system Deferiprone Diseases of red blood cells Erythrocyte Membrane - chemistry Erythropoietin - analysis Female Ferritins - blood haemoglobin E Hematologic and hematopoietic diseases Hemoglobin E Humans Iron - analysis Iron Chelating Agents - therapeutic use iron chelation Liver - metabolism Male Medical sciences Middle Aged Pharmacology. Drug treatments Pyridones - therapeutic use Receptors, Transferrin - blood Thailand thalassaemia intermedia Thiobarbituric Acid Reactive Substances - analysis Thailand Asia Human Hemoglobinopathy Hemopathy Deferiprone Iron Genetic disease β-Thalassemia intermedia haemoglobin E. thalassaemia intermedia Chemotherapy Hemolytic anemia Treatment Chelating agent Hemoglobin iron chelation Clinical trial
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Summary:	Nine patients with either β‐thalassaemia/haemoglobin E (7) or homozygous β‐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 µg/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 µmol/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.
Bibliography:	ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-1 content type line 23
ISSN:	0007-1048 1365-2141
DOI:	10.1046/j.1365-2141.2003.04412.x