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Blood, 1 May 2006, Vol. 107, No. 9, pp. 3733-3737.
Prepublished online as a Blood First Edition Paper on December 22, 2005; DOI 10.1182/blood-2005-07-2933.


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Submitted July 21, 2005
Accepted November 28, 2005

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

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

Clinica Pediatrica, University of Ferrara, Ferrara, Italy
Centro Anemie Congenite, Ospedale Maggiore Policlinico, IRCCS, Milano, Italy
Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy
Clinica Pediatrica, University of Bari, Bari, Italy
Centro Microcitemie, Ospedali Galliera, Genova, Italy
Divisione Pediatrica, Arcispedale Sant'Anna, Ferrara, Italy
Clinica Pediatrica I, University of Milano, Milano, Italy
Centro Microcitemie, University of Torino, Torino, Italy
Clinica Pediatrica, University of Catania, Catania, Italy
Division of Biostatistics and Epidemiology at The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, USA

* Corresponding author; email: c.borgna{at}unife.it.

Deferoxamine (DFO) therapy has been associated with improved survival of thalassemia patients. However, cardiac disease remains the main cause of death in those patients. In 1995 the oral chelator deferiprone became available for clinical use. We compared the occurrence of cardiac disease in patients treated only with DFO and in those whose therapy was switched to deferiprone during the period of observation, from January 31, 1995 to December 31, 2003. All patients with thalassemia major, treated in seven Italian centers, born between 1970 and 1993 and who had not experienced a cardiac event prior to January 1995 were included. DFO only was given to 359 patients and 157 patients received deferiprone for part of the time. A total of 3610 patient years were observed on DFO and 750 on deferiprone. At baseline, the two groups were comparable for age and sex, while ferritin levels were significantly higher in patients switched to deferiprone. Fifty-two cardiac events, including 10 cardiac deaths, occurred during therapy with DFO. No cardiac events occurred during deferiprone therapy or within at least eighteen months after the end of it. In 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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