Zhang Lining, Yang Fan, Feng Sizhou
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China.
Aerospace Center Hospital, Beijing, China.
Ther Adv Hematol. 2020 Feb 13;11:2040620720906002. doi: 10.1177/2040620720906002. eCollection 2020.
Myelofibrosis is one of the Philadelphia chromosome (Ph)-negative myeloproliferative neoplasms with heterogeneous clinical course. Though many treatment options, including Janus kinase (JAK) inhibitors, have provided clinical benefits and improved survival, allogeneic hematopoietic stem-cell transplantation (AHSCT) remains the only potentially curative therapy. Considering the significant transplant-related morbidity and mortality, it is crucial to decide who to proceed to AHSCT, and when. In this review, we discuss recent updates in patient selection, prior splenectomy, conditioning regimen, donor type, molecular mutation, and other factors affecting AHSCT outcomes. Relapse is a major cause of treatment failure; we also describe recent data on minimal residual disease monitoring and management of relapse. In addition, emerging studies have reported pretransplant therapy with ruxolitinib for myelofibrosis showing favorable results, and further research is needed to explore its use in the post-transplant setting.
骨髓纤维化是费城染色体（Ph）阴性骨髓增殖性肿瘤之一，临床病程具有异质性。尽管包括Janus激酶（JAK）抑制剂在内的许多治疗选择已带来临床益处并改善了生存率，但异基因造血干细胞移植（AHSCT）仍然是唯一具有潜在治愈可能的疗法。鉴于与移植相关的显著发病率和死亡率，决定哪些患者适合进行AHSCT以及何时进行至关重要。在本综述中，我们讨论了患者选择、脾切除术、预处理方案、供体类型、分子突变以及其他影响AHSCT结果的因素的最新进展。复发是治疗失败的主要原因；我们还描述了关于微小残留病监测和复发管理的最新数据。此外，新兴研究报告了芦可替尼用于骨髓纤维化的移植前治疗显示出良好结果，需要进一步研究以探索其在移植后环境中的应用。
