title: Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major.
abstract: 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 3,610 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.
authors.affiliation: Clinica Pediatrica Università di Ferrara, Dipartimento di Medicina Clinica e Sperimentale, Via Fossato di Mortara 17/19, 44100 Ferrara, Italy. c.borgna@unife.it
authors.forename: Caterina
authors.initials: C
authors.lastname: Borgna-Pignatti
authors.forename: Maria Domenica
authors.initials: MD
authors.lastname: Cappellini
authors.forename: Piero
authors.initials: P
authors.lastname: De Stefano
authors.forename: Giovanni Carlo
authors.initials: GC
authors.lastname: Del Vecchio
authors.forename: Gian Luca
authors.initials: GL
authors.lastname: Forni
authors.forename: Maria Rita
authors.initials: MR
authors.lastname: Gamberini
authors.forename: Roberta
authors.initials: R
authors.lastname: Ghilardi
authors.forename: Antonio
authors.initials: A
authors.lastname: Piga
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: Romeo
authors.forename: Huaqing
authors.initials: H
authors.lastname: Zhao
authors.forename: Avital
authors.initials: A
authors.lastname: Cnaan
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D000077543:Deferiprone; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D005293:Ferritins / Q000097:blood; D006331:Heart Diseases / Q000209:etiology* / Q000401:mortality / Q000517:prevention & control; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007558:Italy / Q000453:epidemiology; D008297:Male; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000097:blood / Q000150:complications* / Q000188:drug therapy*
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine