非老年原发性血小板增多症患者临床特征及长效干扰素的疗效和安全性分析  

The clinical features of essential thrombocythemia in non-elderly patients and the evaluation of efficacy and safety in pegylated interferon therapy

作　　者：李雅[1] 游建华[1] 毛原飞[1] 高晓东[1] 李军民[1] 吴文[1] LI Ya;YOU Jianhua;MAO Yuanfei;GAO Xiaodong;LI Junmin;WU Wen(Department of Hematology,Shanghai Institute of Hematology,Ruijin Hospital,Shanghai Jiao Tong University School of Medicine,Shanghai 20025,China)

机构地区：[1]上海交通大学医学院附属瑞金医院血液科上海血液学研究所,上海200025

出　　处：《内科理论与实践》2020年第5期332-336,共5页Journal of Internal Medicine Concepts & Practice

摘　　要：目的:总结非老年原发性血小板增多症(essential thrombocythemia,ET)患者临床特征,并评估长效干扰素治疗非老年ET患者的疗效和安全性。方法:总结29例非老年ET患者的临床特征,采用聚乙二醇干扰素α-2b 180μg皮下注射,以每2周1次作为起始剂量,后续根据血常规调整治疗间歇,并评估其疗效及不良反应。结果:患者中位年龄34(2547)岁,其中5例(17%)存在血栓病史;所有患者Bcr/Abl、ASXL1突变均为阴性,其中11例(38%)存在Janus激酶2(Janus kinase 2,JAK2)V617F突变,8例(28%)存在钙网蛋白(calreticulin,CALR)基因外显子9突变,1例(3%)存在MPL外显子10突变,余9例(31%)患者的上述3种基因突变均为阴性。非老年ET患者应用长效干扰素治疗后血小板计数为(509±185)×109/L,较治疗前血小板计数(945±208)×109/L明显下降(P<0.05),治疗总反应率为82%。治疗后白细胞计数、红细胞计数及血红蛋白均较治疗前有明显下降(均P<0.05),但均在参考范围内。不良反应主要为流感样症状(34%、10例)、脱发(10%、3例)、皮疹(3%、1例)和注射局部红肿(17%、5例),均为Ⅰ/Ⅱ级不良反应,无患者因不良反应而终止治疗。结论:非老年ET患者基本为低中危患者,其中有血栓史者占17%;长效干扰素治疗非老年ET患者有效且安全。Objective To summarize the clinical features of essential thrombocythemia(ET) in non-elderly patients(<60 years) and assess the clinical efficacy and safety of pegylated interferon. Methods The clinical features in total 29 nonelderly ET patients were summarized. They got cytoreductive treatment through injecting pegylated interferon subcuta-neously, and the hematological response and the side effect were evaluated. The initial dose of pegylated interferon was180 μg, twice a week, and the regimen was adjusted based on the change of complete blood count. Results The median age of the patients was 34(25-47) years and five of them(17%) had a history of thrombosis. All patients had negative Bcr/Abl and ASXL1 mutations, 11 cases(38%) had Janus kinase 2(JAK2) V617 F mutations, 8(28%) had CALR exon9 mutation and only 1(3%) was found to have MPL exon 10 mutation. The three mutations mentioned above were negative in the remaining 9 cases(31%). The platelet count in non-elderly patients with ET after pegylated interferon treatment was(509±185)×109/L, which was significantly lower than before treatment(945 ±208)×109/L(P<0.05). The overall response rate for treatment was 82%. The white blood cell(WBC), red blood cell(RBC) and hemoglobin(Hb) were also lower( P<0.05) after pegylated interferon therapy, but they were all in normal range. The other common side effect of pegylated interferon in-cluded flu-like symptoms(10 patients, 34%), alopecia(3 patients, 10%), and rash(1 patient, 3%). The reactions in injec-tion site appeared in 5 patients(17%) and were grade Ⅰ/Ⅱ. No patients discontinued therapy because of the side effects of the drug. Conclusions Most of non-elderly patients with ET were low-and intermediate-risk cases, 17% have a history of thrombosis. Pegylated interferon was effective and safe in the treatment of non-elderly ET patients.

关 键 词：原发性血小板增多症 长效干扰素 骨髓增殖性肿瘤 降细胞治疗 

分 类 号：R588.3[医药卫生—内分泌]