@article{5fa749abd2844b489bc7a78c4f691476,
title = "Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study",
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.",
keywords = "Acute myeloid leukemia, Advanced myelodysplastic syndrome, Eltrombopag, Thrombocytopenia",
author = "Moshe Mittelman and Uwe Platzbecker and Sebastian Grosicki and Tomasz Lawniczek and Zewen Zhu and Dominik Selleslag",
note = "Publisher Copyright: {\textcopyright} 2023 The Author(s). Published by S. Karger AG, Basel. This article is licensed under the Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC). Usage and distribution for commercial purposes requires written permission.",
year = "2023",
month = may,
day = "19",
doi = "10.1159/000531146",
language = "אנגלית",
volume = "146",
pages = "373--378",
journal = "Acta Haematologica",
issn = "0001-5792",
publisher = "S. Karger AG",
number = "5",
}