Children with transfusion‐dependent thalassemia (TDT) require regular blood transfusions that, without iron‐chelation therapy, lead to iron‐overload toxicities. Current practice delays chelation therapy (late‐start) until reaching iron overload (serum ferritin ≥1000 μg/L) to minimize risks of iron‐depletion.
Deferiprone’s distinct pharmacological properties, including iron‐shuttling to transferrin, may reduce risks of iron depletion during mild‐to‐moderate iron loads and iron overload/toxicity in children with TDT. The early‐start deferiprone (START) study evaluated the efficacy/safety of early‐start deferiprone in infants/young children with TDT.
Sixty‐four infants/children recently diagnosed with beta‐thalassemia and serum ferritin (SF) between 200 and 600 μg/L were randomly assigned 1:1 to receive deferiprone or placebo for 12 months or until reaching SF‐threshold (≥1000 μg/L at two consecutive visits). Deferiprone was initiated at 25 mg/kg/day and increased to 50 mg/kg/day; some recipients’ dosages increased to 75 mg/kg/day based on iron levels.
The primary endpoint was the proportion of patients ≥SF‐threshold by month 12. Monthly transferrin saturation (TSAT) assessment evaluated iron‐shuttling.
At baseline, there was no significant difference in mean age (deferiprone: 3.03 years, placebo: 2.63 years), SF (deferiprone: 513.8 μg/L, placebo: 451.7 μg/L), or TSAT (deferiprone: 47.98%, placebo: 43.43%) between groups. At month 12, there was no significant difference in growth or adverse event (AE) rates between groups. No deferiprone‐treated patients were iron‐depleted. At month 12, 66% of patients receiving deferiprone remained below SF threshold versus 39% of placebo (p = .045).
Deferiprone‐treated patients showed higher TSAT levels and reached ≥60% TSAT threshold faster. Early‐start deferiprone was well‐tolerated, not associated with iron depletion, and efficacious in reducing iron overload in infants/children with TDT. TSAT results provide the first clinical evidence of deferiprone shuttling iron to transferrin.Read Publication