Eltrombopag at doses up to 300 mg daily had an acceptable safety profile for the treatment of thrombocytopenia in patients with advanced myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML), a recent study published in the journal The Lancet Hematology has shown.1

Because platelet transfusions provide limited benefit for thrombocytopenic patients with MDS or AML, researchers sought to evaluate the safety and tolerability of eltrombopag, an oral thrombopoietin receptor antagonist that increases platelet counts, for the treatment of adult patients with advanced MDS, secondary AML after MDS, or de novo AML.

For the international, placebo-controlled, double-blind, phase 1/2 trial, researchers  enrolled 98 patients who had relapsed or refractory disease or were ineligible for standard therapy, had platelet counts <30 × 109 platelets/L, had 10% to 50% bone marrow blasts, or were dependent on platelet transfusions. Participants were randomly assigned to receive eltrombopag once daily at a dose ranging from 50 mg to 300 mg or placebo.

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Results showed that 98% of patients in the eltrombopag group experienced adverse events compared with 94% in the placebo group.

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The most common adverse events in the eltrombopag treatment arm were pyrexia, nausea, diarrhea, fatigue, decreased appetite, and pneumonia. Grade 3 or higher drug-related adverse events were higher in the placebo group and grade 3 or higher hemorrhage occurred in 26% of patients who received placebo compared with 16% given eltrombopag.

Researchers found no significant difference between the increase in the proportion of peripheral blasts or median bone marrow blast counts between the 2 treatment arms.

“The role of eltrombopag in these patients warrants further investigation,” the authors concluded.


  1. Platzbecker U, Wong RSM, Verma A, et al. Safety and tolerability of eltrombopag versus placebo for treatment of thrombocytopenia in patients with advanced myelodysplastic syndromes or acute myeloid leukaemia: a multicentre, randomised, placebo-controlled, double-blind, phase 1/2 trial. Lancet Hematol. 2015; 2(10):417-426.