Use of eltrombopag for treatment of myelodysplastic syndromes

The prevalence of thrombocytopenia among patients with myelodysplastic syndromes ranges from 40% to 65% and is more often encountered in higher risk disease. The estimate of severe thrombocytopenia (<20 × 109 platelets per L) in lower risk myelodysplastic syndromes is 12%. Thrombocytopenia and its resultant bleeding events remain a major cause of morbidity and mortality. 1 Kantarjian H Giles F List A et al. The incidence and impact of thrombocytopenia in myelodysplastic syndromes. Cancer. 2007; 109: 1705-1714 Crossref PubMed Scopus (196) Google Scholar Eltrombopag versus placebo for low-risk myelodysplastic syndromes with thrombocytopenia (EQoL-MDS): phase 1 results of a single-blind, randomised, controlled, phase 2 superiority trialEltrombopag is well-tolerated in patients with lower-risk myelodysplastic syndromes and severe thrombocytopenia and is clinically effective in raising platelet counts and reducing bleeding events. The assessment of long-term safety and efficacy of eltrombopag and its effect on survival (phase 2 part of study) is still ongoing. Full-Text PDF