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

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.

• Authors: Mayank Dhamija, Amita Mahajan, Manas Kalra, Anju Virmani
• Journal: Indian journal of medical and paediatric oncology : official journal of Indian Society of Medical & Paediatric Oncology (Indian J Med Paediatr Oncol) Vol. 34 Issue 1 Pg. 16-20 (Jan 2013) ISSN: 0971-5851 [Print] Germany
• PMID: 23878481 (Publication Type: Journal Article)