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索拉非尼联合小剂量高三尖杉酯碱作为原发性难治性FLT3-ITD阳性急性髓系白血病的挽救治疗:一例病例报告及文献复习

Sorafenib in combination with low-dose-homoharringtonine as a salvage therapy in primary refractory FLT3-ITD-positive AML: a case report and review of literature.

作者信息

Xu Gaixiang, Mao Liping, Liu Hui, Yang Min, Jin Jie, Qian Wenbin

机构信息

Department of Hematology, The First Affiliated Hospital, College of Medicine, Zhejiang University Hangzhou Zhejiang Province, People's Republic of China.

出版信息

Int J Clin Exp Med. 2015 Nov 15;8(11):19891-4. eCollection 2015.

Abstract

The presence of internal tandem duplications (ITD) in the Fms-related tyrosine kinase 3 receptor (FLT3) has been associated with a poor prognosis in acute myeloid leukemia (AML). Over the past decade, FLT3 is a promising target in FLT3-ITD-positive AML. Sorafenib which is one of the commonly focused FLT3 inhibitors may improve outcome, but only few patients display long-term responses in previously reported cases, prompting the search for underlying resistance mechanisms and therapeutic strategies to overcome them. To the best of our knowledge, this is the first case report about sorafenib in combination with low-dose-homoharringtonine as a salvage therapy successfully administrated and got complete remission (CR) in primary refractory FLT3-ITD-positive AML. Our result demonstrates the combination of this two drugs may be a good choice for the primary refractory FLT3-ITD-positive AML patient, although cooperative studies of large numbers of these patients are needed to evaluate and optimize this combination.

摘要

Fms 相关酪氨酸激酶 3 受体(FLT3)内部串联重复(ITD)的存在与急性髓系白血病(AML)的不良预后相关。在过去十年中,FLT3 是 FLT3-ITD 阳性 AML 中一个有前景的靶点。索拉非尼是常用的 FLT3 抑制剂之一,可能改善预后,但在先前报道的病例中只有少数患者表现出长期反应,这促使人们寻找潜在的耐药机制和克服这些机制的治疗策略。据我们所知,这是首例关于索拉非尼联合小剂量高三尖杉酯碱作为挽救疗法成功应用于原发性难治性 FLT3-ITD 阳性 AML 并获得完全缓解(CR)的病例报告。我们的结果表明,这两种药物联合使用可能是原发性难治性 FLT3-ITD 阳性 AML 患者的一个不错选择,尽管需要大量此类患者的合作研究来评估和优化这种联合用药方案。

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