Randomized phase 2 trial of eltrombopag versus placebo for low-risk myelodysplastic syndromes with thrombocytopenia

Treatment of severe thrombocytopenia in patients with low-risk myelodysplastic syndromes (MDS) is a serious unmet need. The multicenter randomized phase 2 EQOL-MDS trial in Italy evaluated the thrombopoietin receptor agonist eltrombopag versus placebo for low-risk MDS with severe thrombocytopenia. Eltrombopag induced high rate of durable response and had an acceptable toxicity profile in the long term. Improvement in other cytopenias was also observed. The authors conclude that these results support administration of eltrombopag in patients with low-riskMDS and severe thrombocyopenia.