Rovó Alicia, Gavillet Mathilde, Drexler Beatrice, Keller Peter, Schneider Jenny Sarah, Colucci Giuseppe, Beauverd Yan, van Dorland Hendrika Anette, Pollak Matthias, Schmidt Adrian, De Gottardi Andrea, Bissig Marina, Lehmann Thomas, Duchosal Michel A, Zeerleder Sacha
Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
Service and Central Laboratory of Hematology, Department of Oncology, Lausanne University Hospital (CHUV), Lausanne, Switzerland.
Front Med (Lausanne). 2023 Jul 26;10:1200431. doi: 10.3389/fmed.2023.1200431. eCollection 2023.
This national survey investigated the current practice in Switzerland by collecting participants' opinions on paroxysmal nocturnal hemoglobinuria (PNH) clone assessment and clinical practice.
This study aimed to investigate clinical indications prompting PNH clones' assessment and physician's accessibility of a flow cytometry facility, and also to understand clinical attitudes on the follow-up (FU) of patients with PNH clones.
The survey includes 16 multiple-choice questions related to PNH and targets physicians with a definite level of experience in the topic using two screener questions. Opinion on clinical management was collected using hypothetical clinical situations. Each participant had the option of being contacted to further discuss the survey results. This was an online survey, and 264 physicians were contacted through email once a week for 5 weeks from September 2020.
In total, 64 physicians (24.2%) from 23 institutions participated (81.3% hematologists and 67.2% from university hospitals). All had access to flow cytometry for PNH clone testing, with 76.6% having access within their own institution. The main reasons to assess for PNH clones were unexplained thrombosis and/or hemolysis, and/or aplastic anemia (AA). Patients in FU for PNH clones were more likely to be aplastic anemia (AA) and symptomatic PNH. In total, 61% of the participants investigated PNH clones repetitively during FU in AA/myelodysplastic syndromes patients, even when there was no PNH clone found at diagnosis, and 75% of the participants tested at least once a year during FU. Opinions related to clinical management were scattered.
The need to adhere to guidelines for the assessment, interpretation, and reporting of PNH clones emerges as the most important finding, as well as consensus for the management of less well-defined clinical situations. Even though there are several international guidelines, clear information addressing specific topics such as the type of anticoagulant to use and its duration, as well as the indication for treatment with complement inhibitors in some borderline situations are needed. The analysis and the discussion of this survey provide the basis for understanding the unmet needs of PNH clone assessment and clinical practice in Switzerland.
这项全国性调查通过收集参与者对阵发性夜间血红蛋白尿（PNH）克隆评估和临床实践的意见，对瑞士目前的做法进行了调查。
本研究旨在调查促使进行PNH克隆评估的临床指征以及医生使用流式细胞术设备的可及性，同时了解对PNH克隆患者随访（FU）的临床态度。
该调查包括16个与PNH相关的多项选择题，并使用两个筛选问题针对在该主题上有一定经验水平的医生。通过假设临床情况收集对临床管理的意见。每个参与者都可以选择被联系以进一步讨论调查结果。这是一项在线调查，从2020年9月开始，连续5周每周通过电子邮件联系264名医生。
共有来自23个机构的64名医生（24.2%）参与（81.3%为血液科医生，67.2%来自大学医院）。所有人都可以使用流式细胞术进行PNH克隆检测，其中76.6%在自己的机构内可以进行检测。评估PNH克隆的主要原因是不明原因的血栓形成和/或溶血，和/或再生障碍性贫血（AA）。接受PNH克隆随访的患者更可能是再生障碍性贫血（AA）和有症状的PNH。总体而言，61%的参与者在AA/骨髓增生异常综合征患者的随访期间重复检测PNH克隆，即使在诊断时未发现PNH克隆，并且75%的参与者在随访期间每年至少检测一次。与临床管理相关的意见分散。
遵守PNH克隆评估、解释和报告指南的必要性是最重要的发现，同时对于不太明确的临床情况的管理也达成了共识。尽管有几项国际指南，但仍需要明确针对特定主题的信息，如使用的抗凝剂类型及其持续时间，以及在一些临界情况下使用补体抑制剂治疗的指征。本次调查的分析和讨论为了解瑞士PNH克隆评估和临床实践中未满足的需求提供了依据。
