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最新综述:2019 年异基因造血干细胞移植治疗骨髓纤维化

State-of-the-art review: allogeneic stem cell transplantation for myelofibrosis in 2019.

机构信息

Guy's and St. Thomas' NHS Foundation Trust, Department of Haematology, Guy's Tower, Great Maze Pond, London, UK

Comprehensive Cancer Centre, King's College, London, UK.

出版信息

Haematologica. 2019 Apr;104(4):659-668. doi: 10.3324/haematol.2018.206151. Epub 2019 Mar 14.

Abstract

Advances in understanding the pathogenesis and molecular landscape of myelofibrosis have occurred over the last decade. Treating physicians now have access to an ever-evolving armamentarium of novel agents to treat patients, although allogeneic hematopoietic stem cell transplantation remains the only curative approach. Improvements in donor selection, conditioning regimens, disease monitoring and supportive care have led to augmented survival after transplantation. Nowadays, there are comprehensive guidelines concerning allogeneic hematopoietic stem cell transplantation for patients with myelofibrosis. However, it commonly remains difficult for both physicians and patients alike to weigh up the risk-benefit ratio of transplantation given the inherent heterogeneity regarding both clinical course and therapeutic response. In this timely review, we provide an up-to-date synopsis of current transplantation recommendations, discuss usage of JAK inhibitors before and after transplantation, examine donor selection and compare conditioning platforms. Moreover, we discuss emerging data concerning the impact of the myelofibrosis mutational landscape on transplantation outcome, peritransplant management of splenomegaly, poor graft function and prevention/management of relapse.

摘要

在过去的十年中,人们对骨髓纤维化的发病机制和分子特征有了更深入的了解。治疗医生现在可以使用越来越多的新型药物来治疗患者,尽管异基因造血干细胞移植仍然是唯一的治愈方法。供者选择、预处理方案、疾病监测和支持性护理的改善,导致移植后生存率提高。如今,针对骨髓纤维化患者的异基因造血干细胞移植有全面的指南。然而,鉴于临床病程和治疗反应的固有异质性,移植的风险效益比对于医生和患者来说都常常难以权衡。在本次及时的综述中,我们提供了当前移植建议的最新概述,讨论了 JAK 抑制剂在移植前后的使用,检查了供者选择并比较了预处理平台。此外,我们还讨论了关于骨髓纤维化突变特征对移植结果的影响、移植前和移植后巨脾的处理、移植物功能不良的预防/管理以及复发的预防/管理方面的新数据。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b8c7/6442950/5bd7c45b7526/104659.fig1.jpg

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