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Indian J Med Paediatr Oncol. 2013 Jan;34(1):16-20. doi: 10.4103/0971-5851.113407.

Deferasirox in Indian children with thalassemia major: 3 years experience.

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Department of Pediatric Hematology and Oncology, Rajiv Gandhi Cancer Institute and Research Centre, Rohini, New Delhi, India.



To evaluate the efficacy and safety of the oral iron chelator deferasirox in treating transfusional hemosiderosis in a cohort of Indian children with thalassemia major with high iron load.


The first 50 children (age 2-18 yrs) with thalassemia major to commence deferasirox at our center were enrolled and followed up for a period of 36 months between April 2008 and March 2011. The dose of deferasirox was determined by their baseline serum ferritin and was adjusted to a maximum of 40 mg/kg/day depending on response. Ferritin levels, SGOT, SGPT, serum creatinine and urine albumin were regularly monitored.


Of the 50 patients, 76% documented a significant decline in serum ferritin (P<0.05). Seven (14%) patients had a stable ferritin whilst 5 patients (10%) documented an increase over the study period. The mean serum ferritin at baseline, 12, 24 and 36 months was 4354, 3260, 3290 and 3042, respectively (P<0.05). The median serum ferritin at the same time points was 3555, 2810, 2079 and 2271, respectively (P<0.05). No severe toxicity was seen.


Deferasirox, when given in doses ≥30 mg/kg, was found to be an effective and safe drug in reducing transfusional hemosiderosis. Thirty five (70%) needed dose escalation upto 40 mg/kg/day. Fifteen (30%), however did not achieve a negative iron balance despite maximally permissible doses.


Deferasirox; serum ferritin; thalassemia major

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