Eltrombopag was well-tolerated, significantly improved platelet counts, and reduced bleeding events among patients with lower-risk myelodysplastic syndromes (MDS) and severe thrombocytopenia, according to a study published in The Lancet Haematology.1

Among patients with MDS, the presence of thrombocytopenia increases the risk of death, but treatments in this setting are limited. Researchers evaluated whether eltrombopag, a thrombopoietin receptor agonist, improves thrombocytopenia among patients with lower-risk disease.

For the first phase of the single-blind, phase 2 superiority trial (EQoL-MDS; EudraCT Identifier: 2010-022890-33), investigators enrolled 90 adults patients with low-risk or International Prognostic Scoring System intermediate-1-risk MDS and severe thrombocytopenia.

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Participants were randomly assigned 2:1 to receive eltrombopag at varying doses or placebo for at least 24 weeks until disease progression or unacceptable toxicity.

An interim analysis showed that 47% of the 59 patients in the eltrombopag arm achieved platelet response compared with 3% of the 31 patients in the placebo arm.

In the eltrombopag group, 14% had bleeding events vs 42% in the placebo group. There was no significant difference in the rate of progression to acute myeloid leukemia or disease progression between the 2 treatment arms.

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Nearly half (46%) of patients who received eltrombopag had grade 3 to 4 adverse events compared with 16% of those in the placebo group.

Additional analyses assessing the impact of eltrombopag on survival and its long-term safety in this population are ongoing.


  1. Oliva EN, Alati C, Santini V, et al. Eltrombopag versus placebo for low-risk myelodysplastic syndromes with thrombocytopenia (EQoL-MDS): phase 1 results of a single-blind, randomised, controlled, phase 2 superiority trial. Lancet Haematol. 2017 Feb 2. doi: 10.1016/S2352-3026(17)30012-1 [Epub ahead of print]