This phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions.
PRIMARY OBJECTIVES: I. To assess the activity of iron chelation therapy (ICT) with deferasirox, in patients with anemia due to myelodysplastic syndrome (MDS). SECONDARY OBJECTIVES: I. Reduction in red blood cell (RBC) transfusion requirements. II. Hematologic improvement. III. Change in serum ferritin levels from baseline to the end of the study as measured on a monthly basis. IV. Safety and tolerability of deferasirox. EXPLORATORY OBJECTIVES: I. Blood and marrow samples will be taken to study erythropoiesis and the impact of iron overload on erythropoiesis. OUTLINE: Patients receive deferasirox orally (PO) once daily (QD). Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 30 days.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
2
Given PO
Correlative studies
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Proportion of Patients That Achieve Erythroid Hematologic Improvement.
As defined by the modified International Working Group (IWG) response criteria: Erythroid response (pretreatment, \<11 g/dL): 1. Hgb increase by ≥ 1.5 g/dL 2. Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation. Platelet response (pretreatment, \< 100 x 10\^9/L) 1. Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L platelets 2. Increase from \< 20 x 10\^9/L to \> 20 x 10\^9/L and by at least 100% Neutrophil response (pretreatment, \< 1.0 x 10\^9/L) 1\) At least 100% increase and an absolute increase \> 0.5 x 10\^9/L
Time frame: At 6 months
Change in Red Blood Cell (RBC) Transfusion Requirements
Assessed monthly for up to twelve months.
Time frame: Baseline up to 12 months
Change in Serum Ferritin Levels
Assessed monthly for up to twelve months.
Time frame: Baseline up to 12 months
Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement
As defined by the modified International Working Group (IWG) response criteria: Erythroid response (pretreatment, \<11 g/dL): 1. Hgb increase by ≥ 1.5 g/dL 2. Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation. Platelet response (pretreatment, \< 100 x 10\^9/L) 1. Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L platelets 2. Increase from \< 20 x 10\^9/L to \> 20 x 10\^9/L and by at least 100% Neutrophil response (pretreatment, \< 1.0 x 10\^9/L) 1\) At least 100% increase and an absolute increase \> 0.5 x 10\^9/L
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Time frame: At 6 months