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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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RED CELLS
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
From the Clinica Pediatrica of the University of Ferrara, Italy; Clinica Pediatrica of the University of Torino, Italy; Clinica Pediatrica of the University of Catania, Italy; Clinica Pediatrica of the University of Milano, Italy; Clinica Pediatrica of the University of Bari, Italy; Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy; Divisione Pediatrica, Ferrara Hospital, Italy; Ospedali Galliera, Genova, Italy; Centro Anemie Congenite Ospedale Maggiore Policlinico IRCCS, Milan, Italy; and Division of Biostatistics and Epidemiology, The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia.
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 7 Italian centers who were 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 2 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 18 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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