Eltrombopag in patients with high-risk myelodysplastic syndrome or acute myeloid leukaemia: cautious optimism

Moderate thrombocytopenia (platelet count <50 × 109/L) is a common (affecting roughly a fifth of patients) and important manifestation of myelodysplastic syndromes, with prognostic importance 1 Greenberg PL Tuechler H Schanz J et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012; 120: 2454-2465 Crossref PubMed Scopus (2013) Google Scholar that can affect the clinical outcomes of patients. 2 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 , 3 Dayyani F Conley AP Strom SS et al. Cause of death in patients with lower-risk myelodysplastic syndrome. Cancer. 2010; 116: 2174-2179 Crossref PubMed Scopus (131) Google Scholar Although thrombocytopenia is rarely the sole cause of death, nuisance and more clinically significant bleeding can occur in up to 26% of patients. This bleeding can be very distressing for patients and can precipitate the administration of platelet transfusions with short-lived action and their associated risks of infection and alloimmunisation. Hence, there is good rationale for targeting of moderate to severe thrombocytopenia in patients with myelodysplastic syndrome. 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 trialEltrombopag doses up to 300 mg daily had an acceptable safety profile in patients with advanced myelodysplastic syndrome or acute myeloid leukaemia. The role of eltrombopag in these patients warrants further investigation. Full-Text PDF