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.
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原因相同。针对WM的学科发展，66%的医师认为仍需加强临床医师和患者对疾病认知，提高WM的诊断率。 结论： 本研究是首项针对WM的全国范围医师调研，系统性地描述了我国医师在WM诊疗中存在的问题，包括疾病误诊率高、基因检测和治疗新药可及性低、患者对治疗依从性差，我国医师认为改善医师和患者对WM的认识是当前亟须解决的问题之一。.
Keywords: Cross-sectional study; Diagnosis and treatment understanding; Physician survey; Waldenström macroglobulinemia.