title: Long-term treatment with deferiprone enhances left ventricular ejection function when compared to deferoxamine in patients with thalassemia major.
abstract: Transfusion and iron chelation treatment have significantly reduced morbidity and improved survival of patients with thalassemia major. However, cardiac disease continues to be the most common cause of death. We report the left-ventricular ejection fraction, determined by echocardiography, in one hundred sixty-eight patients with thalassemia major followed for at least 5years who received continuous monotherapy with deferoxamine (N=108) or deferiprone (N=60). The statistical analysis, using the generalized estimating equations model, indicated that the group treated with deferiprone had a significantly better left-ventricular ejection fraction than did those treated with deferoxamine (coefficient 0.97; 95% CI 0.37; 1.6, p=0.002). The heart may be particularly sensitive to iron-induced mitochondrial damage because of the large number of mitochondria and its low level of antioxidants. Deferiprone, because of its lower molecular weight, might cross into heart mitochondria more efficiently, improving their activity and, thereby, myocardial cell function. Our findings indicate that the long-term administration of deferiprone significantly enhances left-ventricular function over time in comparison with deferoxamine treatment. However, because of limitations related to the design of this study, these findings should be confirmed in a prospective, randomized clinical trial.
authors.affiliation: U.O.D. Centro per le Microcitemie, A.O.R.N. A. Cardarelli, Napoli, Italy. aldo.filosa@aocardarelli.it
authors.forename: Aldo
authors.initials: A
authors.lastname: Filosa
authors.forename: Angela
authors.initials: A
authors.lastname: Vitrano
authors.forename: Paolo
authors.initials: P
authors.lastname: Rigano
authors.forename: Giuseppina
authors.initials: G
authors.lastname: Calvaruso
authors.forename: Rita
authors.initials: R
authors.lastname: Barone
authors.forename: Marcello
authors.initials: M
authors.lastname: Capra
authors.forename: Liana
authors.initials: L
authors.lastname: Cuccia
authors.forename: Francesco
authors.initials: F
authors.lastname: Gagliardotto
authors.forename: Lorella
authors.initials: L
authors.lastname: Pitrolo
authors.forename: Luciano
authors.initials: L
authors.lastname: Prossomariti
authors.forename: Maddalena
authors.initials: M
authors.lastname: Casale
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Caruso
authors.forename: Calogera
authors.initials: C
authors.lastname: Gerardi
authors.forename: Saveria
authors.initials: S
authors.lastname: Campisi
authors.forename: Paolo
authors.initials: P
authors.lastname: Cianciulli
authors.forename: Michele
authors.initials: M
authors.lastname: Rizzo
authors.forename: Giuseppe
authors.initials: G
authors.lastname: D'Ascola
authors.forename: Angela
authors.initials: A
authors.lastname: Ciancio
authors.forename: Aurelio
authors.initials: A
authors.lastname: Maggio
mesh_terms: D000328:Adult; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use*; D005260:Female; D006331:Heart Diseases / Q000188:drug therapy / Q000209:etiology* / Q000503:physiopathology*; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology*; D008297:Male; D011728:Pyridones / Q000627:therapeutic use*; D012189:Retrospective Studies; D013318:Stroke Volume / Q000187:drug effects*; D016896:Treatment Outcome; D016277:Ventricular Function, Left / Q000187:drug effects; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications*
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D003676:Deferoxamine