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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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1
Department of Pediatric Hematology and Oncology, Rajiv Gandhi Cancer Institute and Research Centre, Rohini, New Delhi, India.

Abstract

OBJECTIVE:

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.

MATERIALS AND METHODS:

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.

RESULTS:

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.

CONCLUSIONS:

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.

KEYWORDS:

Deferasirox; serum ferritin; thalassemia major

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