Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies

PRIMARY OBJECTIVES: I. To determine the effects of the iron-chelating agent deferasirox on
changes in: neutrophil function; macrophage function; lymphocyte function.

SECONDARY OBJECTIVES: I. To determine the effect of chelation on the incidence of bacterial,
viral and fungal infections documented by clinical, microbiologically-proven versus
radiologically-proven criteria. II. To determine the effect of iron chelation on mortality
and morbidity with incidence of the following parameters: Need for hospitalization; Duration
of hospitalization; Need for ventilatory support; Need for exchange transfusion/apheresis;
Need for treatment with antifungals or antibiotics for documented infections.

OUTLINE: Patients receive oral deferasirox once daily for up to 6 months or until blood
counts recover in the absence of disease progression or unacceptable toxicity.

Inclusion Criteria:

- Patients must have a pathology confirmed diagnosis of one of the following:
myelodysplastic syndrome (MDS); acute leukemia; multiple myeloma; myelofibrosis;
lymphoma; chronic anemia; sickle cell anemia

- Iron score >= 2

- Absolute Neutrophil Count (ANC) >= 1,000

- Platelets >= 50,000

- Albumin >= 2 g/dL

- Alkaline phosphatase =< 5X Upper Limit of Normal (ULN)

- Total bilirubin =< 1.5

- Creatinine =< 2X age-appropriate Upper Limit of Normal (ULN) OR creatinine clearance
>= 40 ml/min

- Serum Glutamic Oxaloacetic Transaminase (SGOT) [AST] and Serum Glutamic Pyruvic
Transaminase (SGPT) [ALT] =< 5X Upper Limit of Normal (ULN)

- Eastern Cooperative Oncology Group(ECOG) performance status of 0 or 1

- Women of child-bearing potential and men must agree to use adequate contraception
(hormonal or barrier method of birth control; abstinence) prior to study entry and for
the duration of study participation; should a woman become pregnant or suspect she is
pregnant while participating in this study, she should inform her treating physician
immediately

- Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

- Patients with active disease undergoing chemotherapy treatment

- Patient who have been treated with rituximab or immunomodulating drugs =< 1 month
prior to enrollment

- HIV-positive patients

- Hepatitis-C positive patients

- Women who are pregnant or breastfeeding

- Patients on hemodialysis/patients with renal failure

- Patients with sepsis or acute illness

- Known hypersensitivity to deferasirox

- Patients with moderate or severe hearing loss as defined by audiogram