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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 Waldenström macroglobulinemia in China: a cross-sectional study

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收录情况: ◇ 统计源期刊 ◇ 北大核心 ◇ CSCD-C ◇ 卓越:梯队期刊 ◇ 中华系列

机构: [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 510515 [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
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关键词: 华氏巨球蛋白血症 医师调研 横断面研究 诊疗现状

摘要:
Objective: To conduct a nationwide physician survey to better understand clinicians' disease awareness, treatment patterns, and experience of Waldenström 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% ). Regimens containing Bruton tyrosine kinase inhibitor (BTKi) were most widely recommended for both treatment-naïve and relapsed/refractory patients (94% for all patients, 95% for treatment-naïve patients, and 75% for relapsed/refractory patients), and most physicians recommended Ibrutinib (84% ). For those patients who received treatment, physicians reported that approximately 23% of patients did not comply with the treatment regimen due to a lack of affordability and disease awareness. Furthermore, 66% of physicians believe that in the future, increasing disease awareness and improving diagnosis rates is critical. Conclusions: This study is the first national physician survey of WM conducted in China. It systematically describes the issues that exist in WM diagnosis and treatment in China, such as a high rate of misdiagnosis, limited access to gene testing and new drugs, and poor patient adherence to treatment. Chinese doctors believe that improving doctors' and patients' understanding of WM is one of the most urgent issues that must be addressed right now.

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第一作者机构: [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
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