title: Increased survival and reversion of iron-induced cardiac disease in patients with thalassemia major receiving intensive combined chelation therapy as compared to desferoxamine alone.
abstract: Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major. An intensification monotherapy with deferoxamine (DFO) as well as a combination therapy with DFO and deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for thalassemia major patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight thalassemia major patients with cardiac disease were enrolled in a prospective study lasting 42+/-6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40-50mg/kg over 8-12h at night 5-7 days/week), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in thalassemia major patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.
authors.affiliation: Ospedale Regionale per le le Microcitemie, Cagliari, Italy. laie@pacs.unica.it
authors.forename: Maria Eliana
authors.initials: ME
authors.lastname: Lai
authors.forename: Robert W
authors.initials: RW
authors.lastname: Grady
authors.forename: Stefania
authors.initials: S
authors.lastname: Vacquer
authors.forename: Alessia
authors.initials: A
authors.lastname: Pepe
authors.forename: Maria Paola
authors.initials: MP
authors.lastname: Carta
authors.forename: Patrizio
authors.initials: P
authors.lastname: Bina
authors.forename: Franco
authors.initials: F
authors.lastname: Sau
authors.forename: Paolo
authors.initials: P
authors.lastname: Cianciulli
authors.forename: Aurelio
authors.initials: A
authors.lastname: Maggio
authors.forename: Renzo
authors.initials: R
authors.lastname: Galanello
authors.forename: Patrizia
authors.initials: P
authors.lastname: Farci
mesh_terms: D000328:Adult; D009202:Cardiomyopathies / Q000139:chemically induced / Q000188:drug therapy*; D015913:Chelation Therapy; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007501:Iron / Q000097:blood; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy; D008297:Male; D011446:Prospective Studies; D011728:Pyridones / Q000627:therapeutic use*; D015996:Survival Rate; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000401:mortality
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron; D003676:Deferoxamine