Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study.
Acta Haematol
; 146(5): 373-378, 2023.
Article
em En
| MEDLINE
| ID: mdl-37231838
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.
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Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Trombocitopenia
/
Síndromes Mielodisplásicas
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Leucemia Mieloide Aguda
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Leucopenia
Limite:
Humans
Idioma:
En
Ano de publicação:
2023