Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis

Inclusion criteria

(Core):

• Patients with transfusional iron overload due to:
• low or intermediate (INT-1) risk Myelodysplastic Syndrome (MDS)determined via International Prognosis Scoring System (IPSS) criteria
• other congenital or acquired anemias excluding B-thalassemia and sickle cell disease
• Lifetime transfusion history of ≥20 unit (approximately 100 mL/kg) of packed red blood cells or showing evidence of iron overload (serum ferritin >1000 µg/L).
• Able to provide written informed consent
• Life expectancy ≥ 12 months If patient was previously treated with deferiprone, a washout period of one month should occur before the first dose of deferasirox

Inclusion criteria

(Extension):

• Patients completing the planned 12-month core study (CICL670A2204).
• Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with national legislation.

Exclusion criteria

(Core and Extension):

• Patients with β-thalassemia, sickle cell disease or myelodysplastic syndrome with an IPSS score being Intermediate-2 or High.
• Patients with serum creatinine > ULN
• Patients with ALT(SGPT) levels > 5 x ULN
• Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg in a non-first void urine sample on two assessments during the screening period.
• History of HIV positive test result , or of clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal range)
• Patients on investigational MDS therapies, including lenalidomide, thalidomide, azacitidine and arsenic trioxide, must have a ≥ 4 week washout period prior to the first dose of study drug.
• Patients with systemic uncontrolled hypertension
• Patients with unstable cardiac disease not controlled by standard medical therapy
• Systemic disease (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
• Pregnancy (as documented in required screening laboratory test) or breast feeding.
• Patients treated with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
• Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
• Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the study protocol
• History of hypersensitivity to any of the study drug or excipients
• Sexually active pre-menopausal female patients without adequate contraception. Female patients must use effective contraception or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Other protocol defined inclusion/exclusion criteria may apply.