Waldenström Macroglobulinemia diagnosis, risk assessment and treatment in Portugal – results from a Delphi-like Panel

R. Bergantim, S. Chacim, A. Monteiro, A. M. Macedo, G. Rodrigues, M. G. da Silva
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Abstract

Clinical features of Waldenström Macroglobulinemia (WM) are variable, often leading to heterogeneous decisions regarding patients’ diagnosis, risk stratification, and treatment. This study assessed the agreement rates on WM diagnosis, risk stratification, and active treatment strategies to capture how this heterogeneity may influence national practices among hematologists. A two-round Delphi-like Panel with 22 national hematologists experienced in WM was conducted online, where 33 statements were classified using a 4-point Likert scale. For each statement, the consensus level was set at 70% for “fully agree/disagree”; the majority level was defined as >70% in agreement or disagreement. After two rounds, no statements were categorized as consensus, and 15 out of 33 failed to obtain a qualified majority. Globally, the experts could not reach a qualified majority in approximately half of the sentences from each category (diagnosis, risk assessment, and therapeutic decision), indicating that contradictory opinions are transversal to all the topics involving WM. A lack of consensus in diagnosing and managing WM among Portuguese hematologists became evident. These results illustrate heterogeneity in clinical practices, and future research initiatives should be considered to improve and reinforce accepted guidelines for diagnosing, assessing, and treating WM patients.
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Waldenström葡萄牙巨球蛋白血症的诊断、风险评估和治疗——来自德尔菲样小组的结果
Waldenström巨球蛋白血症(WM)的临床特征是多变的,常常导致对患者的诊断、风险分层和治疗的不同决定。本研究评估了WM诊断、风险分层和积极治疗策略的一致性,以捕获这种异质性如何影响血液学家的国家实践。由22名在WM方面有经验的国家血液学家组成的两轮德尔菲式小组在线进行,其中33项陈述使用4点李克特量表进行分类。对于每个陈述,“完全同意/不同意”的共识水平设置为70%;多数水平定义为同意或不同意的比例大于70%。两轮之后,没有一项发言被归类为共识,33人中有15人未能获得特定多数。在全球范围内,专家们无法在每个类别(诊断、风险评估和治疗决策)的大约一半的句子中达到合格多数,这表明矛盾的观点是横向的,涉及WM的所有主题。葡萄牙血液学家在诊断和管理WM方面缺乏共识,这一点很明显。这些结果说明了临床实践的异质性,未来的研究应该考虑改进和加强诊断、评估和治疗WM患者的公认指南。
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