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追踪骨髓纤维化的决策过程:真实世界实践中芦可替尼治疗的诊断、分层和管理。

Tracing the decision-making process for myelofibrosis: diagnosis, stratification, and management of ruxolitinib therapy in real-word practice.

机构信息

Hematology, Department of Precision and Translational Medicine, Policlinico Umberto 1, Sapienza University, Rome, Italy.

Department of Clinical and Experimental Medicine, Section of Hematology, University of Pisa, Pisa, Italy.

出版信息

Ann Hematol. 2020 Jan;99(1):65-72. doi: 10.1007/s00277-019-03847-z. Epub 2019 Dec 12.

Abstract

The management of patients with myelofibrosis (MF) has dramatically changed since the introduction of ruxolitinib as a tailored treatment strategy. However, the perceptions about the use of this drug in clinical practice remain, at times, a matter of discussion. We conducted a survey about the diagnostic evaluation, prognostic assessment, and management of ruxolitinib in real-life clinical practice in 18 Italian hematology centers. At diagnosis, most hematologists do not use genetically or molecularly inspired score systems to assess prognosis, mainly due to scarce availability of next-generation sequencing (NGS) methodology, with NGS conversely reserved only for a subset of lower-risk MF patients with the aim of possibly improving the treatment strategy. Some common points in the management of ruxolitinib were 1) clinical triggers for ruxolitinib therapy, regardless of risk category; 2) evaluation of infectious risk before the starting of the drug; and 3) schedule of monitoring during the first 12 weeks with the need, in some instances, of supportive treatment. Further development of international recommendations and insights will allow the achievement of common criteria for the management of ruxolitinib in MF, before and after treatment, and for the definition of response and failure.

摘要

自鲁索利替尼作为一种针对性治疗策略引入以来,骨髓纤维化(MF)患者的管理发生了巨大变化。然而,在临床实践中,人们对这种药物的应用的看法,有时仍然存在争议。我们在 18 家意大利血液学中心进行了一项关于鲁索利替尼在真实临床实践中的诊断评估、预后评估和管理的调查。在诊断时,大多数血液学家并不使用基于遗传或分子的评分系统来评估预后,主要是由于缺乏下一代测序(NGS)方法,而 NGS 则相反仅保留给少数低危 MF 患者,目的是可能改善治疗策略。鲁索利替尼管理方面的一些共同点包括:1)鲁索利替尼治疗的临床触发因素,无论风险类别如何;2)在开始药物治疗前评估感染风险;3)在最初 12 周内监测计划,在某些情况下需要支持性治疗。进一步制定国际建议和深入了解将有助于在 MF 患者治疗前后实现鲁索利替尼管理的共同标准,并定义反应和失败。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4ec9/6944647/d2d9926d6584/277_2019_3847_Fig1_HTML.jpg

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