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

被引：0

作者：

Mittelman, Moshe ^{[1
,7
]}

Platzbecker, Uwe ^{[2
]}

Grosicki, Sebastian ^{[3
]}

Lawniczek, Tomasz ^{[4
]}

Zhu, Zewen ^{[5
]}

Selleslag, Dominik ^{[6
]}

机构：

[1] Tel Aviv Univ, Tel Aviv Sourasky Med Ctr, Dept Hematol, Tel Aviv, Israel

[2] Univ Hosp Leipzig, Med Clin & Polyclin 1, Hematol & Cellular Therapy, Leipzig, Germany

[3] Med Univ Silesia, Dept Hematol & Canc Prevent, Katowice, Poland

[4] Novartis Pharm AG, Basel, Switzerland

[5] Novartis Pharmaceut, E Hanover, NJ USA

[6] AZ Sint Jan Brugge Oostende AV, Dept Hematol, Brugge, Belgium

[7] Tel Aviv Sourasky Med Ctr, Dept Hematol, IL-6423906 Tel Aviv, Israel

来源：

ACTA HAEMATOLOGICA | 2023年 / 146卷 / 05期

关键词：

Advanced myelodysplastic syndrome; Acute myeloid leukemia; Eltrombopag; Thrombocytopenia; PLACEBO; BLIND; RISK; INTERMEDIATE; ROMIPLOSTIM; MDS;

D O I：

10.1159/000531146

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：373 / 378

页数：6

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