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Diagnosis and Screening

Many NTDT patients have elevated LIC despite SF <1000 µg/l

SF can underestimate iron overload in NTDT patients1
NTDT Overview_SF Underestimate Iron Overload

Data from THALASSA, a 1-year, prospective, randomized, double-blind, placebo-controlled phase 2 study in 166 patients with β-thalassemia intermedia, α-thalassemia, or HbE/β-thalassemia with liver iron concentration (LIC) ≥5 mg Fe/g dw and serum ferritin (SF) >300 µg/l. Patients were randomized to EXJADE® 5 mg/kg/day (n=55) or placebo (n=28) and EXJADE 10 mg/kg/day (n=55) or placebo (n=28).1

  • Iron distribution is different in NTDT1,2
  • In NTDT patients SF may be a poor diagnostic marker of iron overload—in THALASSA, which only included patients with baseline LIC >5 mg Fe/g dw, more than half had baseline SF ≤1000 µg/l1
  • LIC is a more accurate diagnostic indicator of iron overload in these patients—liver MRI should be the preferred diagnostic tool in NTDT2