Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial
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 Prossomariti, Carmelo Fidone, Paolo Rigano, Alessia Pepe, Gennaro D'Amico, Alberto Morabito, Christian Gluud, 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 Prossomariti, Carmelo Fidone, Paolo Rigano, Alessia Pepe, Gennaro D'Amico, Alberto Morabito, Christian Gluud
Abstract
A multicentre randomized open-label trial was designed to assess the effectiveness of long-term sequential deferiprone-deferoxamine (DFO-DFP) versus DFP alone to treat thalassaemia major (TM). DFP at 75 mg/kg, divided into three oral daily doses, for 4 d/week and DFO by subcutaneous infusion (8-12 h) at 50 mg/kg per day for the remaining 3 d/week was compared with DFP alone at 75 mg/kg, administered 7 d/week during a 5-year follow-up. The main outcome measures were differences between multiple observations of serum ferritin concentrations. Secondary outcomes were survival analysis, adverse events, and costs. Consecutive thalassaemia patients (275) were assessed for eligibility; 213 of these were randomized and underwent intention-to-treat analysis. The decrease of serum ferritin levels during the treatment period was statistically significant higher in sequential DFP-DFO patients compared with DFP-alone patients (P = 0.005). Kaplan-Meier survival analysis for the two chelation treatments did not show any statistically significant differences (long-rank test, P = 0.3145). Adverse events and costs were comparable between the groups. The trial results show that sequential DFP-DFO treatment compared with DFP alone significantly decreased serum ferritin concentration during treatment for 5 years without significant differences regarding survival, adverse events, or costs.
Trial registration: ClinicalTrials.gov NCT00733811.
Source: PubMed