title: Deferiprone as an oral iron chelator in sickle cell disease.
abstract: Iron overload is not uncommon in sickle cell disease (SCD) and requires regular chelation therapy in several instances. The present study evaluates the effect of deferiprone in 15 adult patients with SCD (ten beta(s)/beta(0)thalassemia and five beta(s)/beta(s)) and iron overload. Deferiprone was given at a dose of 75 mg/kg daily for 12 months. The evaluation considered pre- and post-treatment values of serum ferritin, urinary iron excretion, and T2 values of liver and heart obtained by magnetic resonance imaging (MRI). Eleven patients had a liver biopsy prior to starting therapy to evaluate iron concentration (LIC). Twelve patients completed the study with satisfactory compliance. In ten of them (83.3%) the serum ferritin levels decreased significantly at the end of the trial; in eight patients (66.6%) the reduction of serum ferritin was accompanied by a significant increase of their liver T2 values. All patients had a significant increase of urinary iron excretion in response to the drug. Ferritin levels and liver T2 values correlated with liver iron concentration; on the contrary, ferritin levels and liver T2 values failed to show any correlation with heart T2 values. Heart T2 values did not also show any correlation with left ventricular ejection fraction. Deferiprone was well tolerated and did not cause any significant adverse effects. These results suggest that deferiprone may effectively decrease the iron deposition in patients with SCD; moreover, T2 MRI proves to be a reliable and rapid, noninvasive method for assessing the liver iron load in patients with SCD.
authors.affiliation: Thalassemia Center, Laikon General Hospital, 16 Sevastoupoleos Street, 11526, Athens, Greece. ersi_voskaridou@yahoo.com
authors.forename: Ersi
authors.initials: E
authors.lastname: Voskaridou
authors.forename: Maroussa
authors.initials: M
authors.lastname: Douskou
authors.forename: Evangelos
authors.initials: E
authors.lastname: Terpos
authors.forename: Alexandra
authors.initials: A
authors.lastname: Stamoulakatou
authors.forename: John
authors.initials: J
authors.lastname: Meletis
authors.forename: Akis
authors.initials: A
authors.lastname: Ourailidis
authors.forename: Ioannis
authors.initials: I
authors.lastname: Papassotiriou
authors.forename: Dimitris
authors.initials: D
authors.lastname: Loukopoulos
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy* / Q000378:metabolism; D000077543:Deferiprone; D004341:Drug Evaluation; D005260:Female; D005293:Ferritins / Q000097:blood; D006321:Heart / Q000000981:diagnostic imaging / Q000503:physiopathology; D006801:Humans; D007501:Iron / Q000652:urine; D007502:Iron Chelating Agents / Q000008:administration & dosage*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology / Q000378:metabolism; D008099:Liver / Q000000981:diagnostic imaging / Q000378:metabolism; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D011728:Pyridones / Q000008:administration & dosage*; D011859:Radiography; D017086:beta-Thalassemia / Q000188:drug therapy / Q000378:metabolism
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D007501:Iron