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异基因干细胞移植治疗骨髓纤维化

Allogeneic stem-cell transplantation for myelofibrosis.

作者信息

Lavi Noa, Rowe Jacob M, Zuckerman Tsila

机构信息

aDepartment of Hematology and Bone Marrow Transplantation, Rambam Health Care Campus, Haifa bDepartment of Hematology, Shaare Zedek Medical Center, Jerusalem cRuth and Bruce Rappaport Faculty of Medicine, Technion, Haifa, Israel.

出版信息

Curr Opin Hematol. 2017 Nov;24(6):475-480. doi: 10.1097/MOH.0000000000000381.

Abstract

PURPOSE OF REVIEW

Allogeneic hematopoietic stem-cell transplantation (HSCT) remains the only curative therapy for myelofibrosis. The number of HSCTs performed for this indication has been steadily increasing over the past years, even after the approval of the Janus kinase (JAK) inhibitor, ruxolitinib. This increase may be attributed to improved patient selection based on new prognostic molecular markers, more frequent use of matched unrelated donors, secondary to better (high-resolution) human leukocyte antigen typing and supportive care. Ruxolitinib approval raises new questions regarding the role of JAK inhibitors in the transplant setting.

RECENT FINDINGS

The current review summarizes recent updates on HSCT in myelofibrosis. Predictors for transplant outcomes, and specific considerations related to myelofibrosis patient selection for HSCT (e.g. molecular risk stratification) are reviewed. In addition, this review will consider management of myelofibrosis patients in the peritransplant period, including the role of ruxolitinib in the pretransplant period, pre and posttransplant splenomegaly, transplant protocols, posttransplant follow-up of minimal residual disease and interventions in the event of poor engraftment.

SUMMARY

HSCT remains a highly relevant treatment option for myelofibrosis in the era of JAK inhibitors. Recent advances may contribute to a refined definition of HSCT eligibility and identification of the optimal transplantation time, conditioning protocols and posttransplant management.

摘要

综述目的

异基因造血干细胞移植(HSCT)仍然是骨髓纤维化唯一的治愈性疗法。在过去几年中,即使在Janus激酶(JAK)抑制剂芦可替尼获批之后,因该适应症进行的HSCT数量仍在稳步增加。这种增加可能归因于基于新的预后分子标志物改善了患者选择、由于更好的(高分辨率)人类白细胞抗原分型和支持治疗而更频繁地使用匹配的无关供体。芦可替尼的获批引发了关于JAK抑制剂在移植环境中作用的新问题。

最新发现

本综述总结了骨髓纤维化中HSCT的最新进展。回顾了移植结果的预测因素以及与骨髓纤维化患者HSCT选择相关的具体考虑因素(例如分子风险分层)。此外,本综述将考虑骨髓纤维化患者在移植前后时期的管理,包括芦可替尼在移植前期的作用、移植前后的脾肿大、移植方案、移植后微小残留病的随访以及植入不良情况下的干预措施。

总结

在JAK抑制剂时代,HSCT仍然是骨髓纤维化高度相关的治疗选择。最近的进展可能有助于更精确地定义HSCT的适应症,并确定最佳移植时间、预处理方案和移植后管理。

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