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

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

18 Citationer (Scopus)

Abstract

In β-thalassemia major (β-TM) patients, iron chelation therapy is mandatory to reduce iron overload secondary to transfusions. Recommended first line treatment is deferoxamine (DFO) from the age of 2 and second line treatment after the age of 6 is deferiprone (L1). A multicenter randomized open-label trial was designed to assess the effectiveness of long-term alternating sequential L1-DFO vs. L1 alone iron chelation therapy in β-TM patients. Deferiprone 75 mg/kg 4 days/week and DFO 50 mg/kg/day for 3 days/week was compared with L1 alone 75 mg/kg 7 days/week during a 5-year follow-up. A total of 213 thalassemia patients were randomized and underwent intention-to-treat analysis. Statistically, a decrease of serum ferritin level was significantly higher in alternating sequential L1-DFO patients compared with L1 alone patients (p = 0.005). Kaplan-Meier survival analysis for the two chelation treatments did not show statistically significant differences (log-rank test, p = 0.3145). Adverse events and costs were comparable between the groups. Alternating sequential L1-DFO treatment decreased serum ferritin concentration during a 5-year treatment by comparison to L1 alone, without significant differences of survival, adverse events or costs. These findings were confirmed in a further 21-month follow-up. These data suggest that alternating sequential L1-DFO treatment may be useful for some β-TM patients who may not be able to receive other forms of chelation treatment.
OriginalsprogEngelsk
TidsskriftHemoglobin
Vol/bind35
Udgave nummer3
Sider (fra-til)206-16
Antal sider11
ISSN0363-0269
DOI
StatusUdgivet - 2011

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