Title: A study on safety and efficacy of Deferasirox, an oral iron chelator in the treatment of transfusion haemosiderosis in children with β-Thalassemia Major in a tertiary care rural hospital

Authors: Srinivas Mathur, Dr Sanjay Shukla, Dr Rachna Sabale, Dr Sayed Asif Umar

 DOI:  https://dx.doi.org/10.18535/jmscr/v6i8.08

Abstract

Background: β-Thalassemia major is a hereditary hemolytic anaemia (and the commonest transfusion dependent anaemia) which continues to be a major cause of morbidity and mortality in developing countries like India. Deferasirox is a tridentate, once-daily, oral iron chelator that is widely used in the management of patients with transfusion haemosiderosis. Chelation therapy is usually started after 10-20 transfusions or when serum ferritin level reaches 1000 μg/L [Deferasirox has demonstrated consistent dose-dependent efficacy, producing sustained reductions in serum ferritin 6].

Aims and objective

Aim: To study the safety and efficacy of Deferasirox in transfusion haemosiderosis in children with β-Thalassemia.

Objectives 1. To assess the serum ferritin levels in multi-transfused β-Thalassemia patients who are on treatment with Deferasirox. 2. To study the efficacy of Deferasirox in patients admitted with transfusion haemosiderosis in β-Thalassemia. 3. To estimate iron overload in the patients admitted with transfusion haemosiderosis in β-Thalassemia. 4. To study the adverse effect profile andfinal outcome of administration of Deferasirox in patients with transfusion haemosiderosis in β-Thalassemia.

Study Period: 6 months (After the approval from IEC- RMC)

Sample Size: 100Results and Observation there was adecrease in serum ferritin from 4049.87ng/ml to 3647.4ng/ml this was a significant change with a p value 0f 0.0001. Furthermore, a significant negative correlation was observed between the mean dose of deferasirox prescribed and the mean serum ferritin levels. %). Diarrhoea was reported as the most common) in our study it was found that the mean change in the serum creatinine values increased from 0.475 to0.487(table7) in our study this change was not significant(table6)the findings for sodium and serum potassium were not considered significant (table 7 and 8) in our study .awareness among prescribers about ADRs of deferasirox and frequent monitoring of serum ferritin and serum iron levels as a guide to determine its dose is required to improve tolerability of this important and necessary drug.further multi-centered studies with more sample size are needed.

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