01Aug 2023
Efficacy and safety of early-start deferirone in infants and young children with transfusion-dependent beta-thalassemia: Evidence for iron shuttling to transferrin in a randomized, double-blind, placebo-controlled, clinical trial (START)

Efficacy and safety of early-start deferirone in infants and young children with transfusion-dependent beta-thalassemia: Evidence for iron shuttling to transferrin in a randomized, double-blind, placebo-controlled, clinical trial (START)

The START study investigated early-start deferiprone in children with transfusion-dependent thalassemia (TDT) to assess its effectiveness and safety in reducing iron overload. Deferiprone, known for its iron-shuttling properties, was administered to 64 children with mild-to-moderate iron loads. After 12 months, 66% of deferiprone recipients remained below the serum ferritin (SF) threshold, compared to 39% of the placebo group. Deferiprone treatment did not lead to iron depletion and was well-tolerated, with no significant differences in adverse events between groups. Monthly transferrin saturation (TSAT) assessments confirmed deferiprone's iron-shuttling abilities to transferrin, indicating promising clinical evidence for its use.

  • #pediatrics

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