A longitudinal study, published in the American Journal of Hematology, demonstrated that a new oral chelator, deferasirox, can improve cardiac iron overload and effectively maintain cardiac iron levels within the normal range in patients with beta thalassemia major (BTM) in a real world setting. Furthermore, deferasirox appears to normalize cardiac function in patients with abnormal heart function.

Researchers retrospectively evaluated the long-term effects of deferasirox on cardiac magnetic resonance T2*, cardiac function parameters, and cardiovascular clinical events. All transfusion-dependent patients with BTM who received deferasirox at 4 treatment centers in Italy between June 15, 2003, and June 15, 2015, were included in the analysis.

The study cohort totaled 98 patients with BTM. The mean patient age was 27.3 years (range, 4.0-56.5; 35% male), and pediatric patients were included (26%). The mean duration of deferasirox treatment was 6.9 years (range, 1.8-11.6). The average cardiac magnetic resonance T2* significantly increased by 2.6 ± 11.9 milliseconds (=.035) from 30.9 ± 10.4 milliseconds at baseline to 33.4 ± 10.9 milliseconds at the last follow-up, and an even greater increase (11.6 ± 15.5 milliseconds; =.019) occurred in patients with cardiac iron overload at baseline (T2* <20 milliseconds).

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Within a subset of patients who had a left ventricular ejection fraction (LVEF) below 56% at baseline, 11 of 13 (85%) had normalized cardiac function with a significant increase in LVEF from 50.6% ± 6.0% to 60.2% ± 5.0% (=.001).

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The most frequent cardiac adverse event was arrhythmia; however, it never resulted in discontinuation of the drug. During 12 years of follow-up, no cardiac deaths or incident heart failure occurred.

The authors concluded that deferasirox confers long-term benefits to patients with BTM and suggested that the LVEF improvement in patients with abnormal cardiac function, who are typically excluded from clinical trials because of a high risk of death, warrants further evaluation in clinical trials.


1. Casale M, Filosa A, Ragozzino A, et al. Long‐term improvement in cardiac magnetic resonance in β‐thalassemia major patients treated with deferasirox extends to patients with abnormal baseline cardiac function [published online November 29, 2018]. Am J Hematol. doi: 10.1002/ajh.25370