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CC BY-NC-ND 4.0 · Indian J Med Paediatr Oncol 2013; 34(01): 16-20
DOI: 10.4103/0971-5851.113407
ORIGINAL ARTICLE

Deferasirox in Indian children with thalassemia major: 3 years experience

Mayank Dhamija
Department of Pediatric Hematology and Oncology, Rajiv Gandhi Cancer Institute and Research Centre, Rohini, New Delhi, India
,
Amita Mahajan
Department of Pediatric Hematology Oncology, Indraprastha Apollo Hospitals, Sarita Vihar, New Delhi, India
,
Manas Kalra
Department of Oncology and Bone Marrow Transplant, The Children′s Hospital at Westmead, Sydney, NSW 2145, India
,
Anju Virmani
Department of Pediatric Endocrinology, Indraprastha Apollo Hospitals, Sarita Vihar, New Delhi, India
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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


Publication History

Article published online:
20 July 2021

© 2013. Indian Society of Medical and Paediatric Oncology. This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial-License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/.)

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