Exjade: first oral treatment for non-transfusion-dependent thalassaemia

Patients with iron overload in non-transfusion-dependent thalassaemia syndromes (NTDT) can now benefit from the oral chelating agent deferasirox (Exjade).

Thalassaemia is caused by mutations in haemoglobin genes. | SCIENCE PHOTO LIBRARY
Thalassaemia is caused by mutations in haemoglobin genes. | SCIENCE PHOTO LIBRARY

A double-blind study (THALASSA) compared the efficacy of two different starting doses of deferasirox (5mg/kg/day and 10mg/kg/day) against placebo over 1 year in patients with iron overload associated with NTDT.

Of the 166 study participants, 145 were adults and 21 were children aged 10 years or older. Doses were doubled at 24 weeks for patients with a liver iron concentration (LIC) >7mg Fe/g dry weight (dw) and LIC reduction <15% from baseline. Doses were also adjusted based on continuous safety assessments. The primary efficacy endpoint was absolute change in LIC from baseline at 52 weeks.

At 1 year, an average reduction of 3.80mg Fe/g dw was observed in patients treated with deferasirox (starting dose 10mg/kg) compared with an average increase of 0.38mg Fe/g dw in patients receiving placebo (p<0.001).

The most common drug-related adverse events were nausea, rash and diarrhoea.

View Exjade drug record

Further information: Novartis Pharmaceuticals UK Ltd

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