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芦可替尼联合地西他滨通过降低……的变异等位基因频率有效治疗无法分类的骨髓增生异常综合征/骨髓增殖性肿瘤

Ruxolitinib Plus Decitabine Effectively Treats Myelodysplastic Syndrome/Myeloproliferative Neoplasm, Unclassifiable, by Decreasing the Variant Allele Frequency of .

作者信息

Luo Shuna, Xu Xiaofei, Ye Xingnong, Zhu Xiaoqiong, Wu Cai, Chen Dan, Jin Jingxia, Zheng Yan, Zheng Mengli, Huang Jian

机构信息

Department of Hematology, The Fourth Affiliated Hospital of Zhejiang University School of Medicine, Yiwu, Zhejiang, People's Republic of China.

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

出版信息

Onco Targets Ther. 2020 Oct 9;13:10143-10148. doi: 10.2147/OTT.S272207. eCollection 2020.

Abstract

Myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable (MDS/MPN-U) is a subtype of MDS/MPN that exhibits a combination of the features of both MDS and MPN. To date, no curative treatment is available for MDS/MPN-U; however, previous studies have suggested a potential survival advantage for ruxolitinib and hypomethylating agents. We reported a case of a -negative but -positive MDS/MPN-U patient treated with ruxolitinib plus decitabine. After treatment, the patient's clinical symptoms were moderated, and the size of the spleen and the peripheral blood cell counts were reduced. These effects might be due to the regimen's ability to reduce STAT5 activation and upregulate microRNA-181c to downregulate the variant allele frequency (VAF) of .

摘要

骨髓增生异常综合征/骨髓增殖性肿瘤,无法分类(MDS/MPN-U)是MDS/MPN的一种亚型,兼具MDS和MPN的特征。迄今为止,尚无针对MDS/MPN-U的治愈性治疗方法;然而,既往研究提示鲁索替尼和去甲基化药物可能具有生存优势。我们报告了1例JAK2阴性但CALR阳性的MDS/MPN-U患者,接受鲁索替尼加地西他滨治疗。治疗后,患者的临床症状得到缓解,脾脏大小及外周血细胞计数降低。这些效果可能归因于该治疗方案降低STAT5激活及上调微小RNA-181c以下调某基因变异等位基因频率(VAF)的能力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5da/7553600/ce0ee79f738e/OTT-13-10143-g0001.jpg

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