Jia J S, Zhu H H, Fu H X, Gong L Z, Kong J, Huang X J, Jiang H
Peking University People's Hospital, Peking University Institute of Hematology, Beijing 100044, China.
Zhonghua Xue Ye Xue Za Zhi. 2016 Dec 14;37(12):1022-1026. doi: 10.3760/cma.j.issn.0253-2727.2016.12.002.
To explore the efficacy and safety of Sorafenib as monotherapy to FLT3 positive acute myeloid leukemia (AML). 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 test before and after Sorafenib treatment. The peripheral blood WBC count [4.2 (0.9-11.8) ×10/L 39.6 (2.3-209.5) ×10/L, <0.001 ], the percentage of peripheral blast cell [0.07 (0-0.54) 0.53 (0-0.94), <0.001] and the percentage of bone marrow blast cell [0.266 (0.020-0.880) 0.604 (0.180-0.900), =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. 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.
探讨索拉非尼单药治疗FLT3阳性急性髓系白血病（AML）的疗效和安全性。2014年4月至2015年12月，14例FLT3阳性AML患者入组本研究，其中男性7例，女性7例，中位年龄42岁（范围：14 - 81岁）。14例患者中，初诊病例4例，难治性病例9例，复发病例1例，78.6%的患者有严重并发症，57.1%的患者KPS评分低于60分[KPS评分中位数为45（20 - 70）]。索拉非尼给药剂量为每日2次，每次400mg，若耐受则持续使用。通过MICM评估治疗反应，并对索拉非尼治疗前后的数据进行配对样本检验。索拉非尼单药治疗后外周血白细胞计数[4.2（0.9 - 11.8）×10⁹/L对39.6（2.3 - 209.5）×10⁹/L，P<0.001]、外周血原始细胞百分比[0.07（0 - 0.54）对0.53（0 - 0.94），P<0.001]和骨髓原始细胞百分比[0.266（0.020 - 0.880）对0.604（0.180 - 0.900），P = 0.003]均较治疗前显著降低。总缓解率为57.1%（8/14），其中完全缓解（CR）5例（35.7%）。4例初诊病例中，2例达到CR，1例部分缓解（PR），1例未缓解（NR）；10例难治性和复发病例中，3例达到CR，2例达到PR，5例NR。索拉非尼治疗后达到分子缓解（FLT3 - ITD阴性）的中位持续时间为46（33 - 72）天，无进展生存期（PFS）中位数为53（28 - 175）天。索拉非尼在FLT3 - ITD突变阳性AML患者中显示出活性。索拉非尼单药治疗可作为老年患者或有严重并发症患者以及不适合强化化疗的难治性和复发病例的一种治疗选择。
