中国华氏巨球蛋白血症诊断与治疗现状调查:一项横断面研究  

Diagnosis and treatment understanding of Waldenstrom macroglobulinemia in China:a crosssectional study

作　　者：易树华 熊文婕 曹欣欣[3] 孙春艳[4] 杜鹃 王慧涵 王黎[7] 牛挺[8] 姜中兴[9] 魏永强[10] 薛华[11] 褚红玲[12] 邱录贵 李剑[3] Yi Shuhua;Xiong Wenjie;Cao Xinxin;Sun Chunyan;Du Juan;Wang Huihan;Wang Li;Niu Ting;Jiang Zhongxing;Wei Yongqiang;Xue Hua;Chu Honging;Qiu Lugui;Li Jian(State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin 300020,China;Tianjin Institutes of Health Science,Tianjin 301600,China;Peking Union Medical College Hospital,Chinese Academy of Medical Sciences,Beijing 100730,China;Union Hospital,Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430022,China;The Second Afiliated Hospital of Naval Medical University(Shanghai Changzheng Hospital),Shanghai 200003,China;Shengjing Hospital of China Medical University,Shenyang 110004,China;Shanghai Institute of Hematology,State Key Laboratory of Medical Genomics,National Research Center for Translational Medicine at Shanghai,Rujin Hospital Afiliated to Shanghai Jiao Tong University School of Medicine,Shanghai 200025,China;West China Hospital of Sichuan University,Chengdu 610044,China;The First Afiliated Hospital of Zhengzhou University,Zhengzhou 450000,China;Nanfang Hospital,Southern Medical University,Guangzhou 510515,China;The Affiliated Hospital of Hebei University,Baoding 071030,China;Peking University Third Hospital,Beijing 100083,China)

机构地区：[1]中国医学科学院血液病医院(中国医学科学院血液学研究所),血液与健康全国重点实验室,国家血液系统疾病临床医学研究中心,细胞生态海河实验室,天津300020 [2]天津医学健康研究院,天津301600 [3]中国医学科学院北京协和医院,北京100730 [4]华中科技大学同济医学院附属协和医院,武汉430022 [5]海军军医大学第二附属医院(上海长征医院),上海200003 [6]中国医科大学附属盛京医院,沈阳110004 [7]上海交通大学医学院附属瑞金医院,上海200025 [8]四川大学华西医院,成都610044 [9]郑州大学第一附属医院,郑州450000 [10]南方医科大学南方医院,广州1510515 [11]河北大学附属医院,保定071030 [12]北京大学第三医院临床流行病学研究中心,北京100083

出　　处：《中华血液学杂志》2024年第2期148-155,共8页Chinese Journal of Hematology

基　　金：中国医学科学院医学与健康科技创新工程(2022-I2M-1-022、2021-I2M-C&T-B-081)。

摘　　要：目的深度了解我国临床医师对华氏巨球蛋白血症(WM)疾病的认知,临床诊疗行为和经验,为促进我国WM规范化诊疗,改善WM患者临床结局提供研究证据。方法开展面向全国多家三级以及二级医院内血液科、血液肿瘤科以及肿瘤内科医师的调研,自2022年2月至2022年7月招募有WM诊疗经验的临床医师,使用定性序贯定量调研的方法开展研究。结果来自于22个省级行政区内33个城市中219家医院的415位临床医师参加了调研。调研结果显示,在诊断方面,虽然医师为疑似WM患者开具的检查检验项目较为统一(实验室检查项目建议率92%~99%、病理检查79%~95%、基因检查96%、影像学检查63%~83%),但在临床实践中(医师认为)仍有22%的患者会被误诊为其他疾病,且非三甲医院的误诊率高于三甲医院(29%对21%,P<0.001),WM极易与其他疾病混淆以及医师经验不足无法做出准确判断是医师认为的最主要原因;96%的医师认为WM患者需接受MYD88和CXCR4为主的基因检测,因其有助于疾病确诊以及指导治疗方案的选择。在治疗方面,55%的医师认为缓解症状是主要治疗目标,另外检查指标的改善(54%)以及延长总生存期(51%)也是我国医师关注的治疗目标。在有治疗指征的患者中,医师认为21%左右的患者不会接受治疗,主要是经济因素以及患者对疾病认知不足造成的。在选择治疗药物时,63%的医师会把患者是否可以负担治疗药物作为主要的影响因素,其次是患者合并症(61%)、基因检测结果(55%)、疾病风险等级(54%)等。在治疗方案选择上无论是初诊患者还是复发/难治患者,94%医师认为布鲁顿酪氨酸激酶抑制剂(Bruton tyrosine kinase inhibitors,BTKi)是WM最主要的治疗药物(初治95%,复发75%),BTKi中伊布替尼推荐比例最高(84%)。对于接受治疗的患者中,医师的认为约23%的患者不能完成计划的治疗方案,主要原因与有治疗指征但未接受治疗的�Objective To conduct a nationwide physician survey to better understand clinicians disease awareness,treatment patterns,and experience of Waldenstrom macroglobulinemia(WM)in China.Methods This cross-sectional study was conducted from February 2022 to July 2022 by recruiting clinicians with wM treatment experience from hematology,hematology-oncology,and oncology departments throughout China.Quantitative surveys were designed based on the qualitative interviews.Results The study included 415 clinicians from 219 hospitals spread across thirty-three cities and twenty-two provinces.As for diagnosis,the laboratory tests prescribed by physicians for suspected WM patients were relatively consistent(92%-99%recommendation for laboratory,79%-95%recommendation for pathology,96%recommendation for gene testing,and 63%-83%recommendation for imaging examination).However,from a physician's perspective,there was 22%misdiagnosis occurred in clinical practice.The rate of misdiagnosis was higher in lower-level hospitals than in tertiary grade A hospitals(29%vs 21%,P<0.001).The main reasons for misdiagnosis were that WM was easily confused with other diseases,and physicians lacked the necessary knowledge to make an accurate diagnosis.In terms of gene testing in clinical practice,96% of participating physicians believed that WM patients would require gene testing for MYD88 and CXCR4 mutations because the results of gene testing would aid in confirming diagnosis and treatment options.In terms of treatment,55%of physicians thought that the most important goal was to achieve remission,while 54%and 51%of physicians wanted to improve laboratory and/or examination results and extend overall survival time,respectively.Among patients with treatment indications,physicians estimated that approximately 21% of them refused to receive treatment,mainly owing to a lack of affordable care and disease awareness.When selecting the most appropriate treatment regimens,physicians would consider patient affordability(63%),comorbidity(61%),and risk level(54%

关 键 词：华氏巨球蛋白血症 医师调研 横断面研究 诊疗现状 

分 类 号：R733.1[医药卫生—肿瘤]