Cardiac Function Continues to Improve in Beta-Thalassemia Patients After 2 Years on Iron Chelation With Deferasirox

Courtesy by: docguide.com

NEW ORLEANS — December 8, 2009 — Patients diagnosed with cardiac iron overload in Beta-Thalassemia continued to show functional improvement after 2 years of treatment with the iron chelator deferasirox, researchers stated here at the American Society of Hematology (ASH) 51st Annual Meeting and Exposition.

“This is the first large prospective study to report 2-year data on cardiac iron removal for any iron chelator,” said Dudley J. Pennell, MD, Royal Brompton Hospital, London, United Kingdom, during his poster presentation on December 7.

The effect of deferasirox on cardiac function was measured using magnetic cardiac imaging, focusing on the relaxation parameter T2*. Cardiac T2* values <20 ms indicate cardiac iron overload. Values <10 on the T2* scale indicate patients have a likelihood of experiencing heart failure and arrhythmia, according to Dr. Pennell.

He reported 2-year results of the Evaluation of Patients’ Iron Chelation With Exjade (EPIC). Deferasirox is a once daily oral iron chelator. He reported that favourable changes in heart function as measured by T2* assessment that were seen after the first 12 months of treatment were continued out to 24 months.

Among the 39 Beta-Thalassemia patients whose baseline T2* scores were <10 ms, the T2* score rose from 7.3 ms at baseline to 8.1 ms at 1 year (P < .001) and reached a mean of 9.5 ms for the 29 patients who were evaluable after 2 years (P < .001).

Among the 62 patients in the study who had baseline T2* scores between 10 and 20 ms, their mean baseline value of 14.6 ms improved to 17.5 ms at 12 months (P < .001) and reached 20.4 ms after 24 months (P < .001).

The patients also showed improvement in right ventricle function. Dr. Pennell suggested that improvement “is best explained by improved left ventricle and right ventricle compliance associated with reduced cardiac iron, and may be an early marker of functional improvement.”

Right ventricle ejection fraction increased from 66% at baseline to 68.8% at 1 year and 69.9% after 2 years (P < .001 of both scores compared with baseline values).

No drug-related serious adverse events were reported in the 1-year extension. Two serious adverse events seen in the first year of treatment — an episode of renal failure and renal tubular disorder — resolved following drug discontinuation.

About 30% of the participants in the study were aged under 16 years; the rest were aged less than 50 years.

“Results show that continued therapy with deferasirox for up to 2 years at doses of 30 to 40 mg/kg/day was effective in removing iron from the heart in Thalassemia major patients with mild, moderate and severe cardiac siderosis,” Dr. Pennell said.

Funding for this study was provided by Novartis Pharma AG.