Cardiac morbidity and mortality in deferoximine or deferiprone treated patients with thalassemia major

Authors
Caterina Borgna-Pignatti, Maria Domenica Cappellini, Piero De Stefano, Giovanni Carlo Del Vecchio, Gian Luca Forni, Maria Rita Gamberini, Roberta Ghilardi, Antonio Piga, Maria Antonietta Romeo, Huaqing Zhao, and Avital Cnaan

Journal
Blood. 2006; 107:3733-3737.

Background
The life expectancy of patients with thalassaemia major has significantly increased in recent years. However, iron overload of the heart remains the main cause of morbidity and mortality, being responsible for more than half of all deaths.

A recent study from the UK found that 50 percent of patients died before the age of 35. A long-term Italian study found that 65 percent of patients were still alive at that age.

Methods
An epidemiological study was initiated to compare the occurrence of cardiac disease in patients treated only with deferoxamine and in those whose therapy was switched to deferiprone during the observation period. All patients from 7 major thalassemia centres in Italy that were born between 1970 and 1993 who had not experienced a cardiac event were included in the study. 359 patients received deferoxamine only, and 157 patients received deferiprone. The patients were evaluated from January 31, 1995 to December 31, 2003 for cardiac events and disease.

Summary
Fifty-two cardiac events, including 10 cardiac deaths, occurred during therapy with deferoxamine. No cardiac events occurred during deferiprone therapy or within 20 months (or more) after therapy ceased. In contrast to patients treated with deferoxamine, the patients in this study treated with deferiprone did not have any cardiac events. Overall, within the setting of a natural history study, deferiprone therapy was associated with significantly greater cardiac protection than deferoxamine in patients with thalassemia major.

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