Clinical trial of deferiprone iron chelation therapy in beta-thalassaemia/haemoglobin E patients in Thailand.
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.
Pootrakul
Pensri
P
Thalassaemia Research Centre, Institute of Science and Technology for Research and Development, Mahidol University, Salaya Campus, Salaya, UK.
Sirankapracha
Pornpan
P
Sankote
Jongkal
J
Kachintorn
Udom
U
Maungsub
Wanna
W
Sriphen
Kosit
K
Thakernpol
Kleonphan
K
Atisuk
Kanit
K
Fucharoen
Suthat
S
Chantraluksri
Udom
U
Shalev
Oded
O
Hoffbrand
A Victor
AV
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
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