Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomised clinical trial

A multicentre randomized open-label trial was designed to assess theeffectiveness 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 subcutaneousinfusion (8–12 h) at 50 mg/kg per day for the remaining 3 d/week wascompared with DFP alone at 75 mg/kg, administered 7 d/week during a5-year follow-up. The main outcome measures were differences betweenmultiple observations of serum ferritin concentrations. Secondary outcomeswere survival analysis, adverse events, and costs. Consecutive thalassaemiapatients (275) were assessed for eligibility; 213 of these were randomized andunderwent intention-to-treat analysis. The decrease of serum ferritin levelsduring the treatment period was statistically significant higher in sequentialDFP–DFO patients compared with DFP-alone patients (P = 0.005). Kaplan–Meier survival analysis for the two chelation treatments did not show anystatistically significant differences (log-rank test, P = 0.3145). Adverseevents and costs were comparable between the groups. The trial resultsshow that sequential DFP–DFO treatment compared with DFP alonesignificantly decreased serum ferritin concentration during treatment for5 years without significant differences regarding survival, adverse events,or costs.