Anemia Response Of the 68 patients who were transfusion dependent at baseline, to date 54% have become transfusion independent for a minimum of 12 weeks. The median duration of the transfusion-free period has not yet been reached (range: 82-506 days, ongoing). More than 25% of subjects who were not receiving transfusions while on study experienced at least a 1 g/dL increase in hemoglobin lasting for more than eight weeks.
Of the 26 patients who were dosed at 300mg QD and were transfusion dependent at baseline, to date 65% have become transfusion independent for a minimum of 12 weeks
CYT387 is well tolerated in myelofibrosis patients for dosing periods up to and exceeding two years. Reported adverse effects include thrombocytopenia; transient, mild dizziness; mild peripheral neuropathy; and abnormalities in liver/pancreas-related laboratory tests. Treatment emergent anemia and neutropenia were rarely reported.
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