[1]李振波,陆 诚.阿扎胞苷治疗骨髓增生异常综合征合并额外性梳样蛋白1基因突变疗效及安全性分析[J].陕西医学杂志,2021,50(9):1135-1138,1142.[doi:DOI:10.3969/j.issn.1000-7377.2021.09.023]
　LI Zhenbo,LU Cheng.Efficacy and safety of azacitidine in treatment of MDS with ASXL1 gene mutation[J].,2021,50(9):1135-1138,1142.[doi:DOI:10.3969/j.issn.1000-7377.2021.09.023]
点击复制

阿扎胞苷治疗骨髓增生异常综合征合并额外性梳样蛋白1基因突变疗效及安全性分析
分享到：

《陕西医学杂志》[ISSN:1000-7377/CN:61-1281/TN]

卷:
50
期数:
2021年9期
页码:
1135-1138,1142
栏目:
药物与临床
出版日期:
2021-09-05

文章信息/Info

Title:
Efficacy and safety of azacitidine in treatment of MDS with ASXL1 gene mutation
作者:
李振波1; 陆 诚2
(1.宝鸡市高新医院血液科,陕西 宝鸡 721000; 2.宝鸡市中医医院肿瘤血液科,陕西 宝鸡 721001)
Author(s):
LI Zhenbo; LU Cheng
(Department of Hematology,Baoji High-Tech Hospital,Baoji 721000,China)
关键词:
骨髓增生异常综合征;  基因突变;  阿扎胞苷;  额外性梳样蛋白1;  疗效;  安全性
Keywords:
Myelodysplastic syndrome;  Gene mutation;  Azacitidine;  Additional sex combs-like 1;  Efficacy;  Safety
分类号:
R 551.3
DOI:
DOI:10.3969/j.issn.1000-7377.2021.09.023
文献标志码:
A
摘要:
目的:探究阿扎胞苷(AZA)治疗骨髓增生异常综合征(MDS)合并额外性梳样蛋白1(ASXL1)基因突变的疗效及安全性。方法:选取MDS合并ASXL1基因突变患者40例,依据采用的治疗方案不同分为观察组(17例)和对照组(23例)。观察组单用AZA治疗。对照组使用减量CAG方案[阿糖胞苷(Ara-C)+阿克拉霉素(ACR)+粒细胞集落刺激因子(G-CSF)]联合地西他滨治疗。比较两组疗效、不良反应、预后危险度及生存情况。结果:治疗1、3、6个疗程后,观察组总有效率(ORR)均略低于对照组,但组间比较差异无统计学意义(均P>0.05)。观察组Ⅲ-Ⅳ级血红蛋白减少、血小板减少、中性粒细胞绝对值减少发生率低于对照组(均P<0.05)。两组预后危险度构成比较差异无统计学意义(P>0.05)。观察组总生存时间(OS)较对照组缩短(P<0.05)。结论:AZA治疗MDS合并ASXL1基因突变有一定疗效及安全性,但在改善患者预后方面不及减量CAG联合地西他滨方案。
Abstract:
Objective:To explore the efficacy and safety of azacitidine(AZA)in the treatment of myelodysplastic syndrome(MDS)with additional sex combs-like 1(ASXL1)gene mutation.Methods:Forty MDS patients with ASXL1 gene mutation were selected and divided into observation group(17 cases)and control group(23 cases)according to the different treatment schemes adopted.The observation group was treated with AZA alone.The control group was treated with decrease CAG regimen combined with decitabine.The efficacy,adverse reactions,prognostic risk and survival were compared between the two groups.Results:After 1,3,and 6 courses of treatment,the total response rate(ORR)of the observation group was slightly lower than that of the control group,but the difference between the two groups was not statistically significant(all P>0.05).The incidence of grade Ⅲ-Ⅳ hemoglobin reduction,thrombocytopenia,and absolute reduction of neutrophils in the observation group was lower than that in the control group(all P<0.05).There was no significant difference in the composition of the prognostic risk between the two groups(P>0.05).The overall survival time(OS)of the observation group was shorter than that of the control group(P<0.05).Conclusion:AZA has certain efficacy and safety in the treatment of MDS patients with ASXL1 gene mutation.However,it is inferior to decrease CAG regimen combined with decitabine in improving the prognosis.

参考文献/References:

[1] 聂泽强,李晓云,马爻芳,等.WT1基因在骨髓增生异常综合征患者外同血中的表达及临床意义[J].陕西医学杂志,2019,48(8):977-980.
[2] 胡通林,郑智茵,沈建平,等.地西他滨治疗中高危骨髓增生异常综合征的疗效分析[J].中华全科医学,2020,18(4):552-554,689.
[3] 尹 钊,马保根,王怡斐,等.117例骨髓增生异常综合征患者ASXL1基因突变的临床研究[J].中华实用诊断与治疗杂志,2019,33(4):363-365.
[4] Cedena MT,Rapado I,Santos-Lozano A,et al.Correction:Mutations in the DNA methylation pathway and number of driver mutations predict response to azacitidine in myelodysplastic syndromes[J].Oncotarget,2018,9(56):30936.
[5] Liapis K,Papadopoulos V,Vrachiolias G,et al.Refinement of prognosis and the effect of azacitidine in intermediate-risk myelodysplastic syndromes[J].Blood Cancer J,2021,11(2):30.
[6] 高 露,王晓燕,鲍 立,等.阿扎胞苷治疗15例中高危骨髓增生异常综合征的疗效观察[J].临床血液学杂志,2018,31(11):854-858.
[7] Greenberg PL,Stone RM,Alkali A,et al.Myelodysplastic syndromes,version 2.2017,NCCN clinical practice guidelines in oncology[J].J Natl Compr Canc Netw,2017,15(1):60-87.
[8] Cheson BD,Greenberg PL,Bennett JM,et al.Clinical application and proposal for modification of the international working group(IWG)response criteria in myelodysplasia[J].Blood,2006,108(2):419-425.
[9] 周玉才,孙长勇,王茂生,等.参芪扶正注射液对MDS患者血清叶酸、VB12及EPO的影响[J].陕西中医,2017,38(5):605-606.
[10] 陈梅玉,刘 洁,晁红颖,等.骨髓增生异常综合征患者的ASXL1基因变异分析[J].中华医学遗传学杂志,2020,37(2):110-115.
[11] Sekeres MA,Othus M,List AF,et al.Randomized phase Ⅱ study of azacitidine alone or in combination with lenalidomide or with vorinostat in higher-risk myelodysplastic syndromes and chronic myelomonocytic leukemia:North American Intergroup Study SWOG S1117[J].J Clin Oncol,2017,35(24):2745-2753.
[12] Sanchez-Garcia J,Falantes J,Medina PA,et al.Prospective randomized trial of 5 days azacitidine versus supportive care in patients with lower-risk myelodysplastic syndromes without 5q deletion and transfusion-dependent an emia[J].Leuk Lymphoma,2018,59(5):1095-1104.
[13] 赵洪国,刘 锋,秦铁军,等.国产阿扎胞苷治疗较高危骨髓增生异常综合征患者的疗效和安全性分析:多中心、前瞻性、单臂研究[J].中华血液学杂志,2020,41(10):811-817.
[14] 崔 灿.国产阿扎胞苷治疗骨髓增生异常综合征的疗效及安全性评价[D].青岛:青岛大学,2020.
[15] Kharfan-Dabaja MA,Komrokji RS,Zhang Q,et al.TP53 and IDH2 somatic mutations are associated with inferior overall survival after allogeneic hematopoietic cell transplantation for myelodysplastic syndrome[J].Clin Lymphoma Myeloma Leuk,2017,17(11):753-758.
[16] 杜 欣.两种去甲基化药物在骨髓增生异常综合征中的回顾性评价[J].临床血液学杂志,2019,32(11):847-850.
[17] Cedena MT,Rapado I,Santos-Lozano A,et al.Mutations in the DNA methylation pathway and number of driver mutations predict response to azacitidine in myelodysplastic syndromes[J].Oncotarget,2017,8(63):106948-106961.
[18] 姚 伟,李 静,刘桂玲.阿扎胞苷对中高危骨髓增生异常综合征的临床疗效观察[J].东南大学学报:医学版,2020,39(2):193-196.
[19] Kuendgen A,Müller-Thomas C,Lauseker M,et al.Efficacy of azacitidine is independent of molecular and clinical characteristics-an analysis of 128 patients with myelodysplastic syndromes or acute myeloid leukemia and a review of the literature[J].Oncotarget,2018,9(45):27882-27894.
[20] 秦玉婷,王 蕾,郝建萍.MTHFR和TS基因多态性与接受阿扎胞苷治疗的骨髓增生异常综合征患者预后的相关性[J].临床血液学杂志,2020,33(5):334-337.
[21] 杜 欣.阿扎胞苷治疗骨髓增生异常综合征疗效评价[J].中国实用内科杂志,2015,35(2):123-125.
[22] 李 丹.去甲基化药物和异基因造血干细胞移植治疗骨髓增生异常综合征的meta分析[D].石家庄:河北医科大学,2018.
[23] 杨娅平.伴ASXL1基因突变骨髓增生异常综合征的临床特征及预后分析[D].承德:承德医学院,2019.

相似文献/References:

[1]聂泽强,李晓云,马爻芳,等.WT1基因在骨髓增生异常综合征患者外同血中的表达及临床意义[J].陕西医学杂志,2019,(8):977.
　NIE Zeqiang,LI Xiaoyun,MA Yaofang,et al.Expression of WT1 gene in patients with Myelodysplastic syndrome[J].,2019,(9):977.
[2]董 曼,王月莉.不同剂量地西他滨对骨髓增生异常综合征小鼠Beclin1蛋白表达的影响[J].陕西医学杂志,2021,50(6):657.[doi:DOI:10.3969/j.issn.1000-7377.2021.06.004]
　DONG Man,WANG Yueli.Effects of different doses of decitabine on Beclin1 protein expression in mice with myelodysplastic syndrome[J].,2021,50(9):657.[doi:DOI:10.3969/j.issn.1000-7377.2021.06.004]
[3]李荣芳,金 龙,赵立丹.外周血T细胞免疫球蛋白黏蛋白-3和血清乳酸脱氢酶联合检测在骨髓增生异常综合征诊断中的应用价值[J].陕西医学杂志,2021,50(9):1143.[doi:DOI:10.3969/j.issn.1000-7377.2021.09.025]
　LI Rongfang,JIN Long,ZHAO Lidan.Application value of combined detection of peripheral blood TIM-3 and serum LDH in diagnosis of MDS[J].,2021,50(9):1143.[doi:DOI:10.3969/j.issn.1000-7377.2021.09.025]
[4]王美佳,刘虹伶,曾依伶.地西他滨联合HA方案治疗骨髓增生异常综合征疗效及安全性研究[J].陕西医学杂志,2023,52(12):1731.[doi:DOI:10.3969/j.issn.1000-7377.2023.12.023]
　WANG Meijia,LIU Hongling,ZENG Yiling.Efficacy and safety of decitabine combined with harringtonine and cytarabine regimen in treatment of myelodysplastic syndrome[J].,2023,52(9):1731.[doi:DOI:10.3969/j.issn.1000-7377.2023.12.023]
[5]马丽丽,付长江.存活素和细胞周期素依赖性蛋白激酶抑制因子2B在骨髓增生异常综合征诊断及患者预后评估中的临床价值研究[J].陕西医学杂志,2024,(3):348.[doi:DOI:10.3969/j.issn.1000-7377.2024.03.013]
　MA Lili,FU Changjiang.Clinical value of Survivin and P15INK4B in diagnosis of myelodysplastic syndrome and prognosis evaluation of patients[J].,2024,(9):348.[doi:DOI:10.3969/j.issn.1000-7377.2024.03.013]

备注/Memo

备注/Memo:
基金项目:陕西省重点研发计划项目(2018SF-054)
更新日期/Last Update: 2021-09-06