Polycythaemia vera is a challenging disease to study given its low prevalence and prolonged time-to-event for important clinical endpoints such as thrombosis, progression, and mortality. Although researchers in this space often rise to meet these challenges, there is considerable room for improvement in the analysis of retrospective data, the development of risk-stratification tools, and the design of randomised controlled trials. In this Viewpoint, we review the evidence behind the contemporary approach to risk stratification and treatment of polycythaemia vera. Frameworks for using data more efficiently, constructing more nuanced prognostic models, and overcoming challenges in clinical trial design are discussed.

中文翻译：

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改进真性红细胞增多症的调查方法：对当前证据和未来愿景的批判性评估。

真性红细胞增多症是一种具有挑战性的疾病，因为它的患病率低，重要临床终点（如血栓形成、进展和死亡率）的事件发生时间延长。尽管该领域的研究人员经常奋起迎接这些挑战，但在回顾性数据的分析、风险分层工具的开发和随机对照试验的设计方面仍有相当大的改进空间。在这个观点中，我们回顾了当代真性红细胞增多症风险分层和治疗方法背后的证据。讨论了更有效地使用数据、构建更细微的预后模型以及克服临床试验设计中的挑战的框架。