Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study

ASPIRE, a three-part, international, phase 2 trial (ClinicalTrials.gov identifier: NCT01440374), investigated eltrombopag efficacy and safety in patients with advanced myelodysplastic syndrome or acute myeloid leukemia and grade 4 thrombocytopenia (<25 x 10(9) platelets/L). Approximately 30-65% of patients in this open-label extension phase experienced clinically relevant thrombocytopenic events; no conclusions could be made regarding long-term efficacy (non-randomized design, no placebo control), and survival rates may simply reflect advanced disease. Long-term safety was consistent with the double-blind phase and contrasted with earlier SUPPORT study findings in higher-risk patients, suggesting that eltrombopag may have a role in treating thrombocytopenia in patients with low-/intermediate-risk myelodysplastic syndrome.

Automated summary

ASPIRE is a three-part international phase 2 trial that investigated the efficacy and safety of eltrombopag in patients with advanced myelodysplastic syndrome or acute myeloid leukemia and severe thrombocytopenia. The study found that approximately 30-65% of patients in the open-label extension phase experienced clinically relevant thrombocytopenic events. Long-term efficacy could not be determined due to the study design, and survival rates may be influenced by advanced disease. However, eltrombopag showed potential in treating thrombocytopenia in low-/intermediate-risk myelodysplastic syndrome patients, with consistent long-term safety results.