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Acquired aplastic anemia, usually due to immune destruction of bone marrow stem and progenitor cells, often responds to treatment with immunosuppressive agents. Eltrombopag, which stimulates the thrombopoietin receptor, has been shown to have activity in aplastic anemia patients failing immunosuppression (NEJM JW Oncol Hematol Feb 2014 and Blood 2014; 123:1818; NEJM JW Oncol Hematol Sep 2012 and N Engl J Med 2012; 367:11).
To examine whether adding eltrombopag to immunosuppressive therapy with antithymocyte globulin and cyclosporine would increase response rates, investigators conducted a nonrandomized, phase I-II trial in 92 treatment-naive patients with aplastic anemia. Sequential cohorts were given daily eltrombopag from day 14 to 6 month…