Allogeneic hematopoietic cell transplantation for patients with myelofibrosis.
Allogeneic hematopoietic cell transplantation for patients with myelofibrosis.
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DOI：
10.1097/moh.0b013e3283257ab2
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发表时间：
2009-03
影响因子：
3.2
通讯作者：
Deeg HJ
中科院分区：
文献类型：
--
作者：
Zang DY;Deeg HJ
造血细胞移植（HCT）为骨髓纤维化患者提供了潜在的治愈性治疗。目前状况如何？移植策略的改变允许向由于年龄或合并症而在过去不被认为是移植候选者的患者提供HCT。省略高剂量全身照射，调整白消安剂量以达到规定的目标水平，使用氟达拉滨代替环磷酰胺作为免疫抑制剂，添加美法仑，以及加入抗胸腺细胞球蛋白，似乎都有助于提高新方案的耐受性。降低强度预处理方案与非复发死亡率降低相关，即使在60-70岁的患者中也可以成功进行HCT。约50-75%的患者通过HCT治愈。新出现的概念包括新的预后评分系统和新的分子标志物，如Janus激酶（JAK）2突变，这可能有助于做出治疗决定和评估缓解状态。移植预处理方案的修改降低了移植相关死亡率，并允许在年龄越来越大的患者中成功进行HCT。选择应该移植的患者，移植的最佳时机以及移植前和移植后的策略仍然是具有挑战性的问题。
Hematopoietic cell transplantation (HCT) offers potentially curative therapy for patients with myelofibrosis. What is the current status? Changes in transplant strategies allow offering HCT to patients who, because of age or comorbid conditions, were not considered transplant candidates in the past. The omission of high-dose total body irradiation, adjusting doses of busulfan to achieve defined target levels, using fludarabine instead of cyclophosphamide as an immunosuppressive agent, the addition of melphalan, and the incorporation of anti-thymocyte globulin, all appear to have contributed to better tolerability of new regimens. Reduced-intensity conditioning regimens are associated with a decrease in non-relapse mortality and allow for successful HCT even in patients 60–70 years of age. Some 50–75% of patients are cured by HCT. Emerging concepts include new prognostic scoring systems and novel molecular markers such as Janus kinase (JAK) 2 mutations, which may aid in making treatment decisions and assess remission status. Modifications of transplant conditioning regimens have reduced transplant-related mortality and allow carrying out successful HCT in increasingly older patients. The selection of patients who should be transplanted, the optimal timing for transplantation, and pre- and post-transplant strategies remain challenging problems.