Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study.
Mittelman, Moshe a; Platzbecker, Uwe b; Grosicki, Sebastian c; Lawniczek, Tomasz d; Zhu, Zewen e; Selleslag, Dominik f
[Report]
Acta Haematologica.
146(5):373-378, 2023.
(Format: HTML, PDF)
: 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 109 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.
(C) 2023 S. Karger AG, Basel
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