This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
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changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade [ Time Frame: Baseline assessment is followed by monthly assessments for up to 1 year ]
Secondary Outcome Measures :
changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination [ Time Frame: Baseline assessment is followed by monthly assessments for up to 1 year. ]
changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade [ Time Frame: at baseline and 1 at year (at the end of study). ]
Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination. [ Time Frame: From the start of study up to 1 year ]
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Layout table for eligibility information
Ages Eligible for Study:
2 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Age ≥ 2 years
Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia)
ECOG Performance Status ≤ 2
Transfusion overload confirmed with ferritin level >1000 µg/l.
No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
Serum creatine level > ULN
Liver enzymes level < 5 ULN.
No pregnancy or lactation
Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.
Age < 2 years
No iron overload (Ferritin level <1000 µg/l).
Primary iron overload (hereditary hemochromatosis)
Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)