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索拉非尼联合化疗作为FLT3-ITD阳性急性髓系白血病的一线治疗方案

[Sorafenib in combination with chemotherapy as first-line therapy for FLT3-ITD positive acute myeloid leukemia].

作者信息

Zhang Q Y, Wei X D, Yin Q S, Mi R H, Yuan F F, Chen L

机构信息

Department of Hematology, the Affiliated Cancer Hospital of Zhengzhou University; Henan Cancer Hospital, Zhengzhou 450008, China.

出版信息

Zhonghua Xue Ye Xue Za Zhi. 2017 May 14;38(5):415-420. doi: 10.3760/cma.j.issn.0253-2727.2017.05.012.

DOI:10.3760/cma.j.issn.0253-2727.2017.05.012
PMID:28565742
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7354192/
Abstract

To analyze the clinical features of acute myeloid leukemia patients with Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutation and the therapeutic effect of sorafenib in combination with chemotherapy as first-line therapy for these patients. Clinical features and therapeutic effect were retrospectively analyzed in 53 AML patients with FLT3-ITD mutation diagnosed in Henan Cancer Hospital from January 2013 to August 2016. The biological characteristics and clinical efficacy of chemotherapy in combination with or without Sorafeinb were analyzed. FLT3-ITD mutation was identified in 53 AML patients, 22 cases (41.5%) were M(5) subtype. The median of the peripheral WBC was 61.00 (0.98-920.00) ×10(9)/L, and there were 50 (94.3%) patients with WBC>10×10(9)/L. The median of blast cell in bone marrow was 0.730 (0.234-0.966) . The total remission rate of all these 53 patients was 56.6% (30/53) . The complete remission (CR) rates in patients treated with chemotherapy in combination with sorafenib and patients with chemotherapy alone were 86.4% (19/22) and 35.5% (11/31) , respectively. The 1-year overall survival rates of the two groups were 78.3%% and 50.0% (=0.041) , and 1-year progression free survival rates were 75.9% and 42.4% (=0.044) , respectively. AML patients with FLT3-ITD mutation have the characteristics of high peripheral WBC, high blast cells in bone marrow and accompanying with M(5) subtype. Sorafeinb combined with chemotherapy can significantly improve CR rate and short term survival.

摘要

分析伴有Fms样酪氨酸激酶3内部串联重复(FLT3-ITD)突变的急性髓系白血病患者的临床特征,以及索拉非尼联合化疗作为这些患者一线治疗的疗效。回顾性分析2013年1月至2016年8月在河南省肿瘤医院确诊的53例伴有FLT3-ITD突变的急性髓系白血病患者的临床特征和治疗效果。分析联合或不联合索拉非尼化疗的生物学特征和临床疗效。53例急性髓系白血病患者检测到FLT3-ITD突变,22例(41.5%)为M(5)亚型。外周血白细胞中位数为61.00(0.98 - 920.00)×10⁹/L,50例(94.3%)患者白细胞>10×10⁹/L。骨髓原始细胞中位数为0.730(0.234 - 0.966)。这53例患者的总缓解率为56.6%(30/53)。联合索拉非尼化疗患者和单纯化疗患者的完全缓解(CR)率分别为86.4%(19/22)和35.5%(11/31)。两组的1年总生存率分别为78.3%和50.0%(P = 0.041),1年无进展生存率分别为75.9%和42.4%(P = 0.044)。伴有FLT3-ITD突变的急性髓系白血病患者具有外周血白细胞高、骨髓原始细胞高并伴有M(5)亚型的特点。索拉非尼联合化疗可显著提高CR率和短期生存率。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fb/7354192/a33763522d0e/cjh-38-05-415-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fb/7354192/a33763522d0e/cjh-38-05-415-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74fb/7354192/a33763522d0e/cjh-38-05-415-g001.jpg

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本文引用的文献

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Addition of sorafenib versus placebo to standard therapy in patients aged 60 years or younger with newly diagnosed acute myeloid leukaemia (SORAML): a multicentre, phase 2, randomised controlled trial.索拉非尼联合标准疗法与安慰剂联合标准疗法治疗 60 岁及以下初诊急性髓系白血病患者(SORAML):一项多中心、2 期、随机对照试验。
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索拉非尼维持治疗在异基因造血细胞移植后的FLT3-ITD急性髓系白血病中似乎安全且能改善临床结局。
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