title: Monitoring long-term efficacy of iron chelation therapy by deferiprone and desferrioxamine in patients with beta-thalassaemia major: application of SQUID biomagnetic liver susceptometry.
abstract: In this non-randomized prospective study, liver and spleen iron concentrations were monitored annually over a 4-year period by non-invasive Superconducting Quantum Interference Device biomagnetometry in 54 beta-thalassaemia major patients (age, 7-22 years) receiving treatment with deferiprone (75 mg/kg/d). Median liver iron concentrations increased significantly from 1456 to 2029 and 2449 microg/g(liver) at baseline, after 2.0 and 3.2 years respectively. Another group of 51 thalassaemic patients (aged 4-34 years) who received desferrioxamine s.c. for 1.9 years increased their liver iron concentration from 1076 to 1260 microg/g(liver). Taking into account the increase of the daily iron input from transfusions of 3.6 mg/d, caused by weight gain in 67% of the patients treated with deferiprone, a larger total body iron elimination rate was achieved after 2 years than at baseline. A negative ferritin change was observed in 51% of the patients. In 15 non-splenectomized patients, liver iron significantly increased from 1260 to 1937 microg/g(liver) (P < 0.01), but serum ferritin remained stable at 2100 microg/l, as did the spleen iron concentration at 1200 microg/g(spleen). A two-compartment model may predict an average chelation efficacy for desferrioxamine and deferiprone, with a saturation effect of the latter, for a certain chelation and transfusion regimen by a single liver iron quantification.
authors.affiliation: Institut für Molekulare Zellbiologie, Universitätsklinikum Hamburg-Eppendorf, Germany. fischer@uke.uni-hamburg.de
authors.forename: Roland
authors.initials: R
authors.lastname: Fischer
authors.forename: Filomena
authors.initials: F
authors.lastname: Longo
authors.forename: Peter
authors.initials: P
authors.lastname: Nielsen
authors.forename: Rainer
authors.initials: R
authors.lastname: Engelhardt
authors.forename: Robert C
authors.initials: RC
authors.lastname: Hider
authors.forename: Antonio
authors.initials: A
authors.lastname: Piga
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use*; D006801:Humans; D007501:Iron / Q000032:analysis; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008099:Liver / Q000737:chemistry; D008280:Magnetics; D011446:Prospective Studies; D011728:Pyridones / Q000627:therapeutic use*; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000188:drug therapy*
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron; D003676:Deferoxamine