Oral iron chelation and the treatment of iron overload in a pediatric hematology center

Pediatr Blood Cancer. 2009 May;52(5):616-20. doi: 10.1002/pbc.21929.


Background: Recent advances have led to the development of oral iron chelators, which have changed clinical practice. The objective of this study was to descriptively assess the use of one such agent, deferasirox, as standard of care treatment in a large pediatric hematology center.

Procedure: We retrospectively studied all patients at the Texas Children's Hematology Center who were previously or currently treated with deferasirox. We gathered data on demographics, clinical diagnoses, length of time on chronic transfusions, previous use of deferoxamine, adherence to therapy, and reasons for discontinuation. We also assessed changes in serum ferritin, liver function tests, and creatinine for those on deferasirox for a minimum of 12 months.

Results: Fifty-nine patients were studied. Eighty-one percent of patients treated with deferasirox had a diagnosis of sickle cell disease. The mean baseline ferritin level for our study population was 2,117 ng/ml (range 754-7,211). Fifty-three percent of patients had been previously treated with deferoxamine. Adherence to oral therapy was documented in 76% of patients. For those on deferasirox for a minimum of 12 months, serum ferritin decreased in 30% of patients (44% of compliant patients, 11% of poorly compliant patients). Changes in creatinine and liver function tests were mild and did not result in long-term discontinuation of deferasirox in any cases.

Conclusions: Outside of controlled clinical trials, deferasirox can be utilized safely as an oral iron chelator in children although adherence to therapy and the complex interaction of factors that contribute to iron overload still present challenges for clinicians.

MeSH terms

  • Administration, Oral
  • Benzoates / administration & dosage*
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Child
  • Child, Preschool
  • Deferasirox
  • Female
  • Ferritins / blood
  • Hematology
  • Hospitals*
  • Humans
  • Infant
  • Infant, Newborn
  • Iron Chelating Agents / administration & dosage*
  • Iron Chelating Agents / adverse effects
  • Iron Chelating Agents / therapeutic use*
  • Iron Overload / blood
  • Iron Overload / drug therapy*
  • Male
  • Pediatrics*
  • Triazoles / administration & dosage*
  • Triazoles / adverse effects
  • Triazoles / therapeutic use*


  • Benzoates
  • Iron Chelating Agents
  • Triazoles
  • Ferritins
  • Deferasirox