Cardiac morbidity and mortality in deferoxamine- or deferiprone-treated patients with thalassemia major.
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.
Borgna-Pignatti
Caterina
C
Clinica Pediatrica Università di Ferrara, Dipartimento di Medicina Clinica e Sperimentale, Via Fossato di Mortara 17/19, 44100 Ferrara, Italy. c.borgna@unife.it
Cappellini
Maria Domenica
MD
De Stefano
Piero
P
Del Vecchio
Giovanni Carlo
GC
Forni
Gian Luca
GL
Gamberini
Maria Rita
MR
Ghilardi
Roberta
R
Piga
Antonio
A
Romeo
Maria Antonietta
MA
Zhao
Huaqing
H
Cnaan
Avital
A
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*
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine