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索拉非尼治疗 FMS 样酪氨酸激酶 3 内部串联重复突变复发性急性髓系白血病。

Sorafenib in Relapsed AML With FMS-Like Receptor Tyrosine Kinase-3 Internal Tandem Duplication Mutation.

机构信息

From the Department of Medicine, University of Tennessee Health Science Center, Memphis, Tennessee; and Department of Internal Medicine, Creighton University Medical Center, and Department of Internal Medicine, Division of Hematology and Oncology, University of Nebraska Medical Center, Omaha, Nebraska.

出版信息

J Natl Compr Canc Netw. 2015 May;13(5):508-14. doi: 10.6004/jnccn.2015.0070.

DOI:10.6004/jnccn.2015.0070
PMID:25964636
Abstract

Old age (≤65 years), relapsed or refractory disease, and the presence of FMS-like receptor tyrosine kinase-3 (FLT3) internal tandem duplication (ITD) mutation are poor prognostic factors in acute myeloid leukemia (AML). FLT3 inhibitors such as sorafenib have been shown to have a potential role in treating relapsed or refractory AML with FLT3 mutations. In the present report, the use of sorafenib in combination with cytarabine and idarubicin resulted in disease control for 7 months in an older patient with relapsed FLT3-positive AML. This case report and the existing literature indicate that sorafenib has disease activity against relapsed AML with the FLT3-ITD mutation in older patients. Larger multicenter studies should be conducted to confirm these findings, which have the potential to improve outcomes in this high-risk AML subgroup.

摘要

老年(≤65 岁)、疾病复发或难治以及存在 FMS 样酪氨酸激酶-3(FLT3)内部串联重复(ITD)突变是急性髓系白血病(AML)的不良预后因素。已经证实,FLT3 抑制剂如索拉非尼在治疗具有 FLT3 突变的复发或难治性 AML 方面具有潜在作用。在本报告中,索拉非尼联合阿糖胞苷和伊达比星治疗复发性 FLT3 阳性 AML 的老年患者,疾病得到了 7 个月的控制。该病例报告和现有文献表明,索拉非尼对老年具有 FLT3-ITD 突变的复发 AML 具有疾病活性。应开展更大规模的多中心研究来证实这些发现,这有可能改善该高危 AML 亚组的治疗结局。

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引用本文的文献

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Sci Rep. 2019 Dec 9;9(1):18630. doi: 10.1038/s41598-019-54901-9.
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Advances in the drug therapies of acute myeloid leukemia (except acute wpromyelocytic leukemia).急性髓系白血病(急性早幼粒细胞白血病除外)药物治疗的进展
Drug Des Devel Ther. 2018 Apr 30;12:1009-1017. doi: 10.2147/DDDT.S161199. eCollection 2018.
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PLoS Biol. 2017 Feb 3;15(2):e2000487. doi: 10.1371/journal.pbio.2000487. eCollection 2017 Feb.
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J Blood Med. 2016 Apr 19;7:73-83. doi: 10.2147/JBM.S100283. eCollection 2016.
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World J Clin Oncol. 2016 Apr 10;7(2):253-7. doi: 10.5306/wjco.v7.i2.253.
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