Monitoring long-term efficacy of iron chelation therapy by deferiprone and desferrioxamine in patients with beta-thalassaemia major: application of SQUID biomagnetic liver susceptometry

Br J Haematol. 2003 Jun;121(6):938-48. doi: 10.1046/j.1365-2141.2003.04297.x.

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Deferiprone
  • Deferoxamine / therapeutic use*
  • Humans
  • Iron / analysis
  • Iron Chelating Agents / therapeutic use*
  • Liver / chemistry
  • Magnetics
  • Prospective Studies
  • Pyridones / therapeutic use*
  • Treatment Outcome
  • beta-Thalassemia / drug therapy*

Substances

  • Iron Chelating Agents
  • Pyridones
  • Deferiprone
  • Iron
  • Deferoxamine