An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-risk and INT-1, Myelodysplastic Patients Using Serum Ferritin Monitoring

Inclusion Criteria:

- Male or female patients with low or intermediate (INT-1) risk MDS

- Patients can be EITHER naïve to iron chelation OR have had prior treatment with
deferoxamine (DFO).

- Age greater than or equal to 18 years

- Availability of transfusion records for the 12 weeks prior to registration

- A lifetime minimum of 30 previous packed red blood cell transfusions

- Availability of at least three CBC values (pretransfusion) during the 12 weeks prior
to registration

- Serum Ferritin:

For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two
occasions, at least two weeks apart, during the prior year.

Serum ferritin ≥ 1000 ng/mL at screening via the central lab.

- Life expectancy ≥ 6 months

- Sexually active women must use an effective method of contraception, or must have
undergone clinically documented total hysterectomy and/or oophorectomy, or tubal
ligation or be postmenopausal (defined as amenorrhea for at least 12 months)

- Able to provide written informed consent

Exclusion Criteria:

- Serum creatinine above the upper limit of normal

- ALT > 500 U/L during screening

- Clinical or laboratory evidence of active Hepatitis B or C

- Urinary protein/creatinine ratio > 0.5 mg/mg

- History of HIV positive test result (ELISA or Western blot)

- ECOG Performance Status > 2

- Patients with uncontrolled systemic hypertension

- Unstable cardiac disease not controlled by standard medical therapy

- Patients with a diagnosis of or history of clinically relevant ocular toxicity
related to iron chelation

- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study
treatment

- Pregnancy or breast feeding

- Treatment 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

- History of non-compliance to medical regimens or patients who are considered
potentially unreliable and/or not cooperative