索拉非尼单药治疗FLT3-ITD突变阳性急性髓系白血病14例疗效及安全性分析
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...
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| Published in | Zhōnghuá xuèyèxué zázhì Vol. 37; no. 12; pp. 1022 - 1026 |
|---|---|
| Main Authors | , , , , , , |
| Format | Journal Article |
| Language | Chinese English |
| Published |
No. 288, Nanjing road, Heping district, Tianjin
100044,北京大学人民医院、北京大学血液病研究所
01.12.2016
Editorial office of Chinese Journal of Hematology |
| Subjects | |
| Online Access | Get full text |
| ISSN | 0253-2727 2707-9740 |
| DOI | 10.3760/cma.j.issn.0253-2727.2016.12.002 |
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| Abstract | 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. |
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| AbstractList | 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)×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. 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. |
| Abstract_FL | 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. |
| Author | 宫立众 江浩 主鸿鹄 付海霞 孔军 黄晓军 贾晋松 |
| AuthorAffiliation | 100044,北京大学人民医院、北京大学血液病研究所 |
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| Author_FL | Jiang Hao Gong Lizhong Jia Jinsong Zhu Honghu Huang Xiaojun Kong Jun Fu Haixia |
| Author_FL_xml | – sequence: 1 fullname: Jia Jinsong – sequence: 2 fullname: Zhu Honghu – sequence: 3 fullname: Fu Haixia – sequence: 4 fullname: Gong Lizhong – sequence: 5 fullname: Kong Jun – sequence: 6 fullname: Huang Xiaojun – sequence: 7 fullname: Jiang Hao |
| Author_xml | – sequence: 1 fullname: 贾晋松 – sequence: 2 fullname: 主鸿鹄 – sequence: 3 fullname: 付海霞 – sequence: 4 fullname: 宫立众 – sequence: 5 fullname: 孔军 – sequence: 6 fullname: 黄晓军 – sequence: 7 fullname: 江浩 |
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| Copyright | Copyright © Wanfang Data Co. Ltd. All Rights Reserved. 2016年版权归中华医学会所有 Copyright © 2016 by Chinese Medical Association 2016 |
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| DOI | 10.3760/cma.j.issn.0253-2727.2016.12.002 |
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| Keywords | 索拉非尼 DNA突变分析 基因,FLT3 DNA mutational analysis Leukemia,myeloid,acute 白血病,髓样,急性 Gene,FLT3 Sorafinbe |
| Language | Chinese English |
| License | This work is licensed under a Creative Commons Attribution 3.0 License (CC-BY-NC). The Copyright own by Publisher. Without authorization, shall not reprint, except this publication article, shall not use this publication format design. Unless otherwise stated, all articles published in this journal do not represent the views of the Chinese Medical Association or the editorial board of this journal. |
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| Notes | 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 |
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| Publisher | 100044,北京大学人民医院、北京大学血液病研究所 Editorial office of Chinese Journal of Hematology |
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| Snippet | 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... 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... |
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| SubjectTerms | DNA突变分析 基因，FLT3 白血病，髓样，急性 索拉非尼 论著 |
| Title | 索拉非尼单药治疗FLT3-ITD突变阳性急性髓系白血病14例疗效及安全性分析 |
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