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  • Eltrombopag and high-dose d...
    Gómez-Almaguer, David; Herrera-Rojas, Miguel A.; Jaime-Pérez, José C.; Gómez-De León, Andrés; Cantú-Rodríguez, Olga G.; Gutiérrez-Aguirre, César H.; Tarín-Arzaga, Luz; Hernández-Reyes, Jesús; Ruiz-Arguelles, Guillermo J.

    Blood, 06/2014, Letnik: 123, Številka: 25
    Journal Article

    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 × 109/L platelets. Four patients relapsed. Complete response at 6 months (platelets ≥100 × 109/L) was achieved in 50% of patients and response at 6 months (platelets ≥30 <100 × 109/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. •Eltrombopag/dexamethasone is a safe and effective combination for treating newly diagnosed ITP patients.•This treatment may prove useful in achieving lasting responses without additional immunosuppression in some patients.