Iron-chelation therapy with oral chelators in patients with thalassemia major

Hematology. 2013 Jan;18(1):50-5. doi: 10.1179/1607845412Y.0000000046. Epub 2012 Nov 19.

Abstract

In thalassemia major (TM), without iron chelation therapy, iron-mediated free radical damage causes liver, endocrine, and myocardial toxicities. Deferoxamine has universally been the standard therapeutic option for iron chelation therapy; however, its usage is troublesome, leading to suboptimal patient compliance. In order to maximize the effectiveness of iron chelation therapy, oral iron chelators deferiprone and deferasirox constitute an important development, offering a potential to improve compliance. Although both oral drugs are effective, they have differences including different pharmacokinetics and side-effect profiles. Our retrospective evaluation of TM patients using oral chelators showed that oral chelators are effective in reducing iron overload regarding ferritin level and partially in cardiac T2* value. However, in our study side effects and discontinuation rates were unexpectedly high and close follow-up of TM patients using oral chelators should be carefully done. The variability in rate of side effects and drug discontinuation in spelenectomized patients using oral chelators suggests that spleen may have a role in pharmacokinetics of these drugs, as well.

MeSH terms

  • Adolescent
  • Adult
  • Benzoates / adverse effects
  • Benzoates / therapeutic use
  • Blood Transfusion
  • Chelation Therapy / adverse effects
  • Chelation Therapy / methods
  • Child
  • Child, Preschool
  • Deferasirox
  • Deferiprone
  • Deferoxamine / adverse effects
  • Deferoxamine / therapeutic use
  • Humans
  • Iron Chelating Agents / adverse effects
  • Iron Chelating Agents / therapeutic use*
  • Middle Aged
  • Pyridones / adverse effects
  • Pyridones / therapeutic use
  • Retrospective Studies
  • Splenectomy
  • Triazoles / adverse effects
  • Triazoles / therapeutic use
  • Young Adult
  • beta-Thalassemia / drug therapy*
  • beta-Thalassemia / therapy

Substances

  • Benzoates
  • Iron Chelating Agents
  • Pyridones
  • Triazoles
  • Deferiprone
  • Deferoxamine
  • Deferasirox