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


METHODS
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.
Raphael
Jean L
JL
Department of Pediatrics, Baylor College of Medicine, Texas Children's Sickle Cell Center, Houston, Texas, USA. Raphael@bcm.edu
Bernhardt
M Brooke
MB
Mahoney
Donald H
DH
Mueller
Brigitta U
BU
D000284:Administration, Oral; D001565:Benzoates / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006405:Hematology; D006761:Hospitals; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy*; D008297:Male; D010372:Pediatrics; D014230:Triazoles / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use*
D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000077588:Deferasirox