Deferasirox (DFX) is a relatively new
iron chelator approved by the US Food and Drug Administration for treatment of children >2 years of age. Prospective studies in Asian Indian children are limited. The β-
thalassemia patients receiving regular transfusions in the
thalassemia ward of an advanced pediatric center were included in this study. Monitoring of side effects was carried out by assessing monthly
transaminases and serum
creatinine levels.
Hemoglobin levels were determined before
blood transfusion. Thirty patients of transfusion-dependent
thalassemia were eligible for the final analysis. The male:female ratio was 3.3:1, and ages ranged from 2.0 to 21 years. The serum
ferritin (SF) level at the start of
therapy was 2657.7±1414.6 (mean±SD). The mean dose of DFX was 21.57 mg/kg/d (range, 17.2 to 27.2 mg/kg/d). Common side effects noted were gastrointestinal manifestations in 5 (16.6%) and
skin rash in 2 (6.6%) patients. There was an increase in serum
creatinine in 2 patients, and treatment was interrupted in 1. Reversible
cytopenia was observed in 1 patient. In 13/30 patients, an initial increase in SF was observed. A decrease in SF levels compared with initial value was seen in only 8 patients at a follow-up of 24 months, at a median dose of 28.8 mg/kg/d. Thus, DFX is a relatively safe oral
iron chelator that can be used in Asian Indians, with gastrointestinal problems like
diarrhea and
abdominal pain as the most common side effects. Treatment requires individualization with careful dose escalation and proper monitoring.