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原发性骨髓纤维化及由其他骨髓增殖性肿瘤演变而来的骨髓纤维化的异基因造血干细胞移植。

Allogeneic haematopoietic stem cell transplantation for primary myelofibrosis and myelofibrosis evolved from other myeloproliferative neoplasms.

作者信息

Tamari Roni, Castro-Malaspina Hugo

机构信息

aAdult Bone Marrow Transplant Service, Department of Medicine, Memorial Sloan Kettering Cancer Center bDepartment of Medicine, Weill Cornell Medical College, New York, New York, USA.

出版信息

Curr Opin Hematol. 2015 Mar;22(2):184-90. doi: 10.1097/MOH.0000000000000121.

Abstract

PURPOSE OF REVIEW

Allogeneic haematopoietic stem cell transplantation (allo-HSCT) is the only curative treatment for myelofibrosis. Major improvements in the field, such as the introduction of reduced intensity conditioning regimens, have made transplant a better tolerated treatment that can be offered to older patients and those with comorbidities. However, treatment-related toxicities, graft-versus-host disease, infectious complications and relapse remain major problems posttransplant. We reviewed here the recent published data and outline the criteria to select patients with myelofibrosis who can benefit the most from this curative treatment.

RECENT FINDINGS

Data regarding mutations in myelofibrosis have been useful to better define the prognosis of patients and have provided a tool to monitor minimal residual disease after transplantation. New data regarding the use of age and comorbidities has allowed a better selection of patients who can benefit from transplantation. Janus-activated kinase signal (JAK) 1/2 inhibitors pretransplant can improve patient's performance status and potentially improve transplant outcomes.

SUMMARY

Improvements in the field of allo-HSCT, the ability to improve patient's performance status prior to transplant with JAK1/2 inhibitors and a more accurate disease risk stratification based on molecular mutations to select patients who can benefit from allo-HSCT should result in better transplant outcomes. Efforts should be made to transplant patients with myelofibrosis on prospective studies to answer some unresolved questions.

摘要

综述目的

异基因造血干细胞移植(allo-HSCT)是骨髓纤维化唯一的治愈性治疗方法。该领域的重大进展,如引入减低强度预处理方案,使移植成为一种耐受性更好的治疗方法,可用于老年患者和合并症患者。然而,治疗相关毒性、移植物抗宿主病、感染并发症和复发仍然是移植后的主要问题。我们在此回顾了最近发表的数据,并概述了选择能从这种治愈性治疗中获益最大的骨髓纤维化患者的标准。

最新发现

关于骨髓纤维化突变的数据有助于更好地确定患者的预后,并提供了一种监测移植后微小残留病的工具。关于年龄和合并症使用的新数据使我们能够更好地选择能从移植中获益的患者。移植前使用Janus激活激酶信号(JAK)1/2抑制剂可改善患者的身体状况,并可能改善移植结果。

总结

allo-HSCT领域的进展、使用JAK1/2抑制剂在移植前改善患者身体状况的能力以及基于分子突变进行更准确的疾病风险分层以选择能从allo-HSCT中获益的患者,应能带来更好的移植结果。应努力在前瞻性研究中对骨髓纤维化患者进行移植,以回答一些未解决的问题。

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