title: Cardiac and hepatic iron and ejection fraction in thalassemia major: multicentre prospective comparison of combined deferiprone and deferoxamine therapy against deferiprone or deferoxamine monotherapy.
abstract: BACKGROUND
  Due to the limited data available in literature, the aim of this multi-centre study was to prospectively compare in thalassemia major (TM) patients the efficacy of combined deferiprone (DFP) and deferoxamine (DFO) regimen versus either DFP and DFO in monotherapy by cardiovascular magnetic resonance (CMR) over a follow up of 18 months.
  
  
  METHODS
  Among the first 1135 TM patients in the MIOT (Myocardial Iron Overload in Thalassemia) network, we evaluated those who had received either combined regimen (DFO + DFP, N=51) or DFP (N=39) and DFO (N=74) monotherapies between the two CMR scans. Iron overload was measured by T2* multiecho technique. Biventricular function parameters were quantitatively evaluated by cine images.
  
  
  RESULTS
  The percentage of patients that maintained a normal global heart T2* value was comparable between DFP+DFO versus both monotherapy groups. Among the patients with myocardial iron overload at baseline, the changes in the global heart T2* and in biventricular function were not significantly different in DFP+DFO compared with the DFP group. The improvement in the global heart T2* was significantly higher in the DFP+DFO than the DFO group, without a difference in biventricular function. Among the patients with hepatic iron at baseline, the decrease in liver iron concentration values was significantly higher with combination therapy than with either monotherapy group.
  
  
  CONCLUSIONS
  In TM patients at the dosages used in the real world, the combined DFP+DFO regimen was more effective in removing cardiac iron than DFO, and was superior in clearing hepatic iron than either DFO or DFP monotherapy. Combined therapy did not show an additional effect on heart function over DFP.
authors: Cardiovascular MR Unit, Fondazione G, Monasterio CNR-Regione Toscana and Institute of Clinical Physiology, Pisa, Italy. alessia.pepe@ftgm.it
authors: Alessia
authors: A
authors: Pepe
authors: Antonella
authors: A
authors: Meloni
authors: Giuseppe
authors: G
authors: Rossi
authors: Liana
authors: L
authors: Cuccia
authors: Giuseppe Domenico
authors: GD
authors: D'Ascola
authors: Michele
authors: M
authors: Santodirocco
authors: Paolo
authors: P
authors: Cianciulli
authors: Vincenzo
authors: V
authors: Caruso
authors: Maria Antonietta
authors: MA
authors: Romeo
authors: Aldo
authors: A
authors: Filosa
authors: Lorella
authors: L
authors: Pitrolo
authors: Maria Caterina
authors: MC
authors: Putti
authors: Angelo
authors: A
authors: Peluso
authors: Saveria
authors: S
authors: Campisi
authors: Massimiliano
authors: M
authors: Missere
authors: Massimo
authors: M
authors: Midiri
authors: Letizia
authors: L
authors: Gulino
authors: Vincenzo
authors: V
authors: Positano
authors: Massimo
authors: M
authors: Lombardi
authors: Paolo
authors: P
authors: Ricchi
mesh_terms: D000328:Adult; D000704:Analysis of Variance; D009202:Cardiomyopathies / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology / Q000503:physiopathology; D016009:Chi-Square Distribution; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D007558:Italy; D008099:Liver / Q000187:drug effects* / Q000378:metabolism / Q000473:pathology; D019028:Magnetic Resonance Imaging, Cine; D008297:Male; D009206:Myocardium / Q000378:metabolism* / Q000473:pathology; D011237:Predictive Value of Tests; D011446:Prospective Studies; D011728:Pyridones / Q000627:therapeutic use*; D013318:Stroke Volume / Q000187:drug effects*; D013997:Time Factors; D016896:Treatment Outcome; D016277:Ventricular Function, Left / Q000187:drug effects*; D016278:Ventricular Function, Right / Q000187:drug effects*; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D003676:Deferoxamine