Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
It has been widely shown that an appropriate chelating therapy in chronic anemias
transfusion dependent can prevent the overstock of iron and can reduce the already existing
overstock reducing, then, the co-morbidity and improving survival.
In particular, some authors have shown in MDS affected patients undergoing intensive
chelating therapy with deferoxamine haematological recovery with a reduction of the need of
With the present study, we plan to evaluate the safety and efficacy of a therapy with the
new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis.
This is an open label, single arm study on Deferasirox treatment in MDS patients with
chronic transfusional hemosiderosis.
Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of
iron administered during transfusions and, if needed, to reduce the overload of already
After an screening phase in which patients are evaluated according to eligibility criteria,
a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
On a monthly basis thereafter from baseline assessment.
Emanuele ANGELUCCI, Pr.
Ospedale "A. Businco", Cagliari
Italy: The Italian Medicines Agency