Deferiprone Reduces Iron Overload in Young Patients With Transfusion-Dependent Beta-Thalassaemia
In young patients with transfusion-dependent beta-thalassaemia (TDT), the use of early-start deferiprone appeared to be well tolerated and was associated with efficacy in reducing iron overload, according to the results of a recent study. Study findings were reported in the American Journal of Hematology.
Iron overload is the main risk factor for morbidity and mortality in patients with transfusion-dependent thalassaemia unless treated with life-long iron chelation therapies such as deferiprone, deferasirox, or deferoxamine. Current clinical practice delays chelation therapy in newly diagnosed patients until they reach the iron overload criterion of serum ferritin (SF) levels ≥1000 μg/L or > 10 transfusions.
This late-start strategy was thought to minimize toxicity risks of iron depletion, observed during early-start studies with deferoxamine. Unfortunately, iron accumulation in organs (e.g., heart or liver) during late-start therapy commonly leads to iron overload in transfusion-dependent children and potentially increased toxicities later in life.
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-thalassaemia 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.
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 efficacious at maintaining low iron levels; it was generally well-tolerated with a safety profile comparable to deferiprone treatment in older patients,” the study investigators wrote in their report.
These data suggest that early-start deferiprone may reduce the increased risk of iron overload toxicities later in life.
