Clinical Challenges with Iron Chelation in Beta Thalassemia

Hematol Oncol Clin North Am. 2023 Apr;37(2):379-391. doi: 10.1016/j.hoc.2022.12.013.

Abstract

Conventional therapy for severe thalassemia includes regular red cell transfusions and iron chelation therapy to prevent and treat complications of iron overload. Iron chelation is very effective when appropriately used, but inadequate iron chelation therapy continues to contribute to preventable morbidity and mortality in transfusion-dependent thalassemia. Factors that contribute to suboptimal iron chelation include poor adherence, variable pharmacokinetics, chelator adverse effects, and difficulties with precise monitoring of response. The regular assessment of adherence, adverse effects, and iron burden with appropriate treatment adjustments is necessary to optimize patient outcomes.

Keywords: Adherence; Adverse effects; Deferasirox; Deferiprone; Deferoxamine; Iron chelation.

Publication types

  • Review

MeSH terms

  • Deferiprone / therapeutic use
  • Deferoxamine / therapeutic use
  • Humans
  • Iron / therapeutic use
  • Iron Chelating Agents / therapeutic use
  • Iron Overload* / etiology
  • Pyridones / therapeutic use
  • Thalassemia* / therapy
  • beta-Thalassemia* / therapy

Substances

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