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

Moshe Mittelman*, Uwe Platzbecker, Sebastian Grosicki, Tomasz Lawniczek, Zewen Zhu, Dominik Selleslag

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

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 × 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.

Original languageEnglish
Pages (from-to)373-378
Number of pages6
JournalActa Haematologica
Volume146
Issue number5
DOIs
StatePublished - 19 May 2023

Funding

FundersFunder number
Novartis Pharmaceuticals Corporation

    Keywords

    • Acute myeloid leukemia
    • Advanced myelodysplastic syndrome
    • Eltrombopag
    • Thrombocytopenia

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