摘要
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400mg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）×10^9/L对39.6 （2.3～209.5）×10^9/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.
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） × 10^9/L vs 39.6 （2.3-209.5） × 10^9/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.
出处
《中华血液学杂志》
CAS
CSCD
北大核心
2016年第12期1022-1026,共5页
Chinese Journal of Hematology
基金
首都市民健康项目培育（Z131100006813026）
