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. 2016 Dec 14;37(12):1022-1026.
doi: 10.3760/cma.j.issn.0253-2727.2016.12.002.

[Efficacy and safety of Sorafenib as monotherapy to FLT3-ITD positive acute myeloid leukemia]

[Article in Chinese]
J S Jia  1 , H H Zhu, H X Fu, L Z Gong, J Kong, X J Huang, H Jiang
Affiliations

[Efficacy and safety of Sorafenib as monotherapy to FLT3-ITD positive acute myeloid leukemia]

[Article in Chinese]
J S Jia et al. Zhonghua Xue Ye Xue Za Zhi. .

Abstract
in English, Chinese

Objective: To explore the efficacy and safety of Sorafenib as monotherapy to FLT3 positive acute myeloid leukemia (AML). Methods: From April 2014 to December 2015, fourteen AML patients with FLT3 positive, 7 males and 7 females with a median age of 42 (range: 14-81) years old, were enrolled in this study. Of the 14 cases, 4 were de novo cases, 9 refractory cases and 1 relapsed case, including 78.6% patients with severe complications and 57.1% patients with KPS score less than 60 [the median KPS score was 45 (20-70) ]. The administration of Sorafenib was 400 mg twice daily and Sorafenib was continued if tolerated. The treatment response was evaluated by MICM and the data were analyzed by paired samples t test before and after Sorafenib treatment. Results: The peripheral blood WBC count [4.2 (0.9-11.8) ×109/L vs 39.6 (2.3-209.5) ×109/L, P<0.001 ], the percentage of peripheral blast cell [0.07 (0-0.54) vs 0.53 (0-0.94), P<0.001] and the percentage of bone marrow blast cell [0.266 (0.020-0.880) vs 0.604 (0.180-0.900), P=0.003] were significantly decreased after Sorafenib monotherapy compared with before. The overall response rate was 57.1% (8/14), including 5 cases (35.7%) with complete remission (CR). Of 4 de novo cases, 2 achieved CR, 1 with PR, 1 with NR; 3 of 10 refractory and relapsed patients achieved CR and 2 cases achieved PR, 5 cases NR. The median duration of achieving molecular remission (FLT3-ITD negative) after Sorafenib was 46(33-72) days, and the median progression free survival (PFS) was 53 (28-175) days. Conclusion: Sorafenib shows activity in FLT3-ITD mutation positive AML patients. Sorafenib monotherapy could be used as a treatment option for elderly patients or patients with severe complications, and refractory and relapsed patients with not suitable for intensive chemotherapy.

目的: 探讨索拉非尼单药治疗FLT3-ITD突变阳性急性髓系白血病（AML）患者的有效性与安全性。

方法: 选取2014年4月至2015年12月使用索拉非尼单药治疗的FLT3-ITD突变阳性AML患者14例，其中男7例，女7例，中位年龄42(14~81）岁。4例为初治患者，9例为难治性患者，1例为复发患者。其中78.6%（11/14）的患者存在严重合并症；57.1%（8/14）的患者KPS评分<60分，中位评分45(20~70）分。索拉非尼起始用量为400 mg每日2次，如患者耐受持续使用。通过MICM检查对患者的治疗效果进行评价，通过配对t检验对索拉非尼治疗前后的临床指标进行分析。

结果: 索拉非尼单药治疗后与治疗前相比较，外周血WBC [4.2(0.9~11.8）×109/L对39.6(2.3~209.5）×109/L，P<0.001]、幼稚细胞比例[0.07(0~0.54）对0.53(0~0.94），P<0.001]以及骨髓幼稚细胞比例[0.266(0.020~0.880）对0.604(0.180~0.900），P=0.003]均明显下降。治疗总反应率为57.1%（8/14），5例（35.7%）达到完全缓解（CR）；4例初治患者中2例达到CR, 1例达到部分缓解（PR），1例未缓解（NR）；10例难治复发患者中3例达到CR, 2例达到PR，5例NR。有效患者达分子生物学缓解（FLT3-ITD转阴）的中位时间为46(33~72）d，中位无进展生存时间为53(28~175）d。治疗相关不良反应可耐受。

结论: 索拉非尼对FLT3-ITD突变阳性AML患者具有一定疗效。索拉非尼单药治疗方案可作为老年或伴有严重合并症、暂时不适宜行强化疗和难治复发FLT3-ITD突变阳性AML患者的一个治疗选择。

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基金项目：首都市民健康项目培育（Z131100006813026）