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.
|Numero di pagine||11|
|Stato di pubblicazione||Published - 2011|