Journal
BLOOD
Volume 123, Issue 25, Pages 3906-3908Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2014-01-549360
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Immune thrombocytopenia (ITP) results from platelet destruction and production suppression. Eltrombopag belongs to a new class of thrombopoietin-mimetic drugs that raise platelet counts in ITP patients. We performed a single-arm study to assess the response to a single course of dexamethasone (40 mg by mouth, days 1-4) in combination with eltrombopag (50 mg, days 5-32) in 12 adults with newly diagnosed ITP in an outpatient setting. Median follow-up was 12.5 months. After therapy (day 33), 100% of patients achieved at least >= 30 x 10(9)/L platelets. Four patients relapsed. Complete response at 6 months (platelets >= 100 x 10(9)/L) was achieved in 50% of patients and response at 6 months (platelets >= 30 <100 x 10(9)/L) was achieved in another 25%; relapse-free survival was 66.7% at 12 months (median response duration of 8.3 months). In conclusion, eltrombopag/dexamethasone is a feasible frontline therapy for ITP. This trial is registered at www.clinicaltrials.gov as NCT01652599.
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