Sequential alternating deferiprone and deferoxamine treatment compared to deferiprone monotherapy: main findings and clinical follow-up of a large multicenter randomized clinical trial in thalassemia major patients

Angela Vitrano, Paolo Rigano, Luciano Prossomariti, Calogera Gerardi, Alessia Pepe, Carmelo Fidone, Francesco Gagliardotto, Crocetta Argento, Angela Vitrano, Pietro Violi, Alessandra Quota, Gaetano Restivo Pantalone, Domenico Giuseppe D''Ascola, Michele Rizzo, Liana Cuccia, Carmelo Magnano, Roberto Malizia, Paolo Cianciulli, Saveria Campisi, Aldo FilosaVincenzo Caruso, Christian Gluud, Alberto Morabito, Marcello Capra, Aurelio Maggio, Maria Antonietta Romeo, Gennaro D'Amico

Risultato della ricerca: Articlepeer review

15 Citazioni (Scopus)

Abstract

In β-thalassemia major (β-TM) patients, iron chelation therapy is mandatory to reduce ironoverload secondary to transfusions. Recommended first line treatment is deferoxamine (DFO) fromthe age of 2 and second line treatment after the age of 6 is deferiprone (L1). A multicenterrandomized open-label trial was designed to assess the effectiveness of long-term alternatingsequential L1-DFO versus L1 alone iron chelation therapy in β-TM patients. Deferiprone75 mg/kg 4 days/week and DFO 50 mg/kg/day for 3 days/week was compared with L1 alone75 mg/kg 7 days/week during 5-year follow-up. A total of 213 thalassemia patients wererandomized and underwent intention-to-treat analysis. Statistically, a decrease of serum ferritinlevels was significantly higher in alternating sequential L1-DFO patients compared with L1alone patients (p = 0.005). Kaplan-Meier survival analysis for the two chelation treatmentsdid not show statistically significant differences (log-rank test, p = 0.3145). Adverse events andcosts were comparable between the groups. Alternating sequential L1-DFO treatment decreasedserum ferritin concentration during a 5-year treatment by comparison to L1 alone, withoutsignificant differences of survival, adverse events or costs. These findings were confirmed in afurther 21-month follow-up. These data suggest that alternating sequential L1-DFO treatmentmay be useful for some β-TM patients who may not be able to receive other forms of chelationtreatment.
Lingua originaleEnglish
pagine (da-a)206-216
Numero di pagine11
RivistaHemoglobin
Volume35
Stato di pubblicazionePublished - 2011

All Science Journal Classification (ASJC) codes

  • Hematology
  • Genetics(clinical)
  • Clinical Biochemistry
  • Biochemistry, medical

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