Improving survival with deferiprone treatment in patients with thalassemia major: A prospective multicenter randomised clinical trial under the auspices of the Italian Society for Thalassemia and Hemoglobinopathies

Angela Vitrano, Paolo Rigano, Luciano Prossomariti, Gaetano Giuffrida, Calogera Gerardi, Carmelo Fidone, Francesco Gagliardotto, Crocetta Argento, Angela Vitrano, Pietro Violi, Francesco Cantella, Francesca Commendatore, Rocca Cingari, Gaetano Roccamo, Turi Lombardo, Giovanni Giugno, Giovanni Giugno, Michele Rizzo, Gaetano Giuffrida, Gaetano GiuffridaDomenico Giuseppe D'Ascola, Liana Cuccia, Carmelo Magnano, Roberto Malizia, Paolo Cianciulli, Saveria Campisi, Angela Ciancio, Anna Meo, Maria Concetta Galati, Aldo Filosa, Vincenzo Caruso, Marcello Capra, Aurelio Maggio, Maria Antonietta Romeo

Risultato della ricerca: Articlepeer review

77 Citazioni (Scopus)

Abstract

The prognosis for thalassemia major has dramatically improved in the last two decades. However, manytransfusion-dependent patients continue to develop progressive accumulation of iron. This can lead to tissuedamage and eventually death, particularly from cardiac disease. Previous studies that investigated ironchelation treatments, including retrospective and prospective non-randomised clinical trials, suggested thatmortality, due mainly to cardiac damage, was reduced or completely absent in patients treated with deferiprone (DFP) alone or a combined deferiprone–deferoxamine (DFP–DFO) chelation treatment. However, no survival analysis has been reported for a long-term randomised control trial. Here, weperformed a multicenter, long-term, randomised control trial that compared deferoxamine (DFO) versus DFPalone, sequential DFP–DFO, or combined DFP–DFO iron chelation treatments. The trial included 265 patients with thalassemia major, with 128 (48.3%) females and 137 (51.7%) males. No deaths occurred with the DFP-alone or the combined DFP–DFO treatments. One death occurred due to graft versus host disease (GVHD) in a patient that had undergone bone marrow transplantation; this patientwas censored at the time of transplant. Only one death occurred with the DFP–DFO sequential treatment in a patient that had experienced an episode of heart failure one year earlier. Ten deaths occurred with thedeferoxamine treatment.The main factors that correlated with an increase in the hazard ratio for death were: cirrhosis, arrhythmia,previous episode of heart failure, diabetes, hypogonadism, and hypothyroidism. In a Cox regression model, the interaction effect of sex and age was statistically significant (p-value <0.013). For each increasing year ofage, the hazard ratio for males was 1.03 higher than that for females (p-value<0.013).In conclusion, the results of this study show that the risk factors for predicting mortality in patients withthalassemia major are deferoxamine-treatment, complications, and the interaction effect of sex and age
Lingua originaleEnglish
pagine (da-a)247-251
Numero di pagine5
RivistaBLOOD CELLS, MOLECULES, &amp; DISEASES
Volume42
Stato di pubblicazionePublished - 2009

All Science Journal Classification (ASJC) codes

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  • ???subjectarea.asjc.1300.1312???
  • ???subjectarea.asjc.2700.2720???
  • ???subjectarea.asjc.1300.1307???

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