Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
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This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload [ Time Frame: monthly during the therapy and at the end of the treatment (aftr 9 months therapy) ]
Secondary Outcome Measures
patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events [ Time Frame: during the treatment (9 months) ]
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Ages Eligible for Study:
18 Years to 80 Years (Adult, Senior)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion
Serum ferritin> 1800 µg/L
Age: 18-80 years
men and women
Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells
Deferoxamin therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason
Eastern Cooperative Oncology Group (ECOG) performance status score between 0-2
written informed consent
beta thalassaemia minor,
haemosiderosis caused by other than chronic transfusional iron overload,
patients with impaired renal function (Creatinin clearance< 60 ml/ min),
patient of childbearing potential unwilling to use contraceptive precautions
known hypersensitivity to deferasirox or any ingredients,
impaired hepatic function (SGOT,SGPT 5x above UNL).
Patients severely ill due to underlying disease progression or other severe concomitant disease.
Patients with poor prognosis of karyotype
patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition
History of nephrotic syndrome
Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
Patients with positive test to HIV
Other protocol-defined inclusion/exclusion criteria may apply