Efficacy of deferasirox in North Indian β-thalassemia major patients: a preliminary report.

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.