首页|去甲基化药物对骨髓增生异常综合征伴骨髓纤维化患者的疗效分析

去甲基化药物对骨髓增生异常综合征伴骨髓纤维化患者的疗效分析

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目的:探究去甲基化药物(hypomethylating agent,HMA)在骨髓增生异常综合征(myelodysplastic syndrome,MDS)伴或不伴骨髓纤维化(MF)患者治疗中的临床效果.方法:收集山西白求恩医院2017年1月-2023年9月收治的147例MDS患者,其中MF0级患者50例,MF 1~3级患者97例.根据骨髓纤维化的严重程度将接受HMA单药治疗的患者分为2组,非纤维化组(MF0级)27例,纤维化期组(MF 1~3级)59例;根据是否接受HMA单药治疗将MF 1~3级患者分为2组,支持治疗组30例,HMA单药治疗组59例.分别比较2组患者的年龄、性别、血液学指标(白细胞计数、中性粒细胞计数、血红蛋白、血小板计数)、骨髓原始细胞比例、根据 IPSS-R 与 IPSS-M 的危险分层、WHO 2016 分型、基因突变(TP53、ASXL1、U2AF1、DNMT3A、SF3B1、TET2、JAK2、RUNX1)以及治疗疗效.结果:非纤维化组与纤维化期组年龄、白细胞计数、中性粒细胞计数、血红蛋白、骨髓原始细胞比例、根据IPSS-R与IPSS-M的危险分层以及基因突变比较,差异无统计学意义(P>0.05),性别、血小板计数、中位总生存期(overall survival,OS)与中位无进展生存期(progression-free survival,PFS),差异有统计学意义(P<0.05),对其中74例患者进行了疗效分析,差异无统计学意义(P>0.05);支持治疗组与HMA单药治疗组的年龄、性别、白细胞计数、中性粒细胞计数、血红蛋白以及中位OS、中位PFS比较,差异无统计学意义(P均>0.05),血小板计数、骨髓原始细胞比例、根据IPSS-R及IPSS-M的危险分层、WHO 2016分型、基因突变(ASXL1、TET2)差异有统计学意义(P<0.05).结论:MDS中骨髓纤维化与预后不良有关.接受HMA单药治疗并不能改善MDS伴骨髓纤维化患者的预后,并且接受HMA单药治疗并不能延长MDS伴骨髓纤维化患者的OS和PFS.
Effect of hypomethylating agent on myelodysplastic syndrome with myelofibrosis
Objective:To explore the clinical efficacy of hypomethylating agent(HMA)in the treatment of pa-tients with or without myelofibrosis(MF)in myelodysplastic syndrome(MDS).Methods:147 MDS patients ad-mitted to Shanxi Bethune Hospital from January 2017 to September 2023 were collected,including 50 patients with MF grade 0 and 97 patients with MF grade 1-3.Patients treated with HMA were divided into two groups ac-cording to the severity of myelofibrosis,27 cases in the non-fibrotic group(MF 0)and 59 cases in the fibrotic phase group(MF 1-3);patients with MF grade 1-3 were divided into two groups according to whether they re-ceived HMA,30 cases in the supportive treatment group and 59 cases in the HMA group.Age,sex,hematologi-cal parameters(white blood cell count,neutrophil count,hemoglobin,platelet count),proportion of bone marrow blasts,risk stratification according to IPSS-R and IPSS-M,WHO 2016 classification,gene mutations(TP53,ASXL1,U2AF1,DNMT3A,SF3B1,TET2,JAK2,RUNX1)and treatment efficacy were compared between the two groups.Results:There were no significant differences in age,white blood cell count,neutrophil count,hemoglobin,percentage of bone marrow blasts,risk stratification according to IPSS-R versus IPSS-M,and gene mutations between non-fibrotic and fibrotic groups(P>0.05),sex,platelet count,median overall survival and median progression-free survival,statistically significant(P<0.05),Seventy-four of these patients were analyzed for efficacy and there was no significant difference between subgroups(P>0.05);Supportive treatment vs HMA:There were no significant differences in age,sex,leukocyte count,neutrophil count,hemoglobin,and median o-verall survival versus median progression-free survival(P>0.05).Platelet count,proportion of bone marrow blasts,risk stratification according to IPSS-R and IPSS-M,WHO 2016 typing,and gene mutations(ASXL1,TET2)were statistically significant(P<0.05).Conclusion:Myelofibrosis in MDS is associated with poor prog-nosis.Treatment with HMA alone did not improve outcomes in patients with MDS and myelofibrosis,and treat-ment with HMA alone did not prolong overall survival and progression-free survival in patients with MDS and my-elofibrosis.

hypomethylating agentmyelodysplastic syndromemyelofibrosis

祝坤、张婕、贡蓉

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山西医科大学第三医院,山西白求恩医院,山西医学科学院,同济山西医院(太原,030032)

去甲基化药物 骨髓增生异常综合征 骨髓纤维化

2024

临床血液学杂志
华中科技大学同济医学院血液病研究所 北京医科大学血液病研究所

临床血液学杂志

CSTPCD
影响因子:1.063
ISSN:1004-2806
年,卷(期):2024.37(5)