使用临床、突变和移植特异性资料选择原发性骨髓纤维化患者进行干细胞移植

Nico Gagelmann, Nicolaus Kröger
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引用次数: 0

摘要

与其他血液系统恶性肿瘤一样,MF患者的治疗已经进入了分子时代。几种驱动和非驱动突变的预后影响现在已经确定;这与患者选择异体造血细胞移植和移植后的结果有明显的相关性。对于移植,目前的JAK2或三阴性驱动突变基因型以及目前的ASXL1突变可能为患者提供关于移植后结果的咨询,以及临床(表现状态、年龄、白细胞和血小板计数)和移植相关因素(供体关系)的最佳效用。不同的调节强度似乎对移植后的结果没有影响,但仍需要在分子时代进行比较。虽然ruxolitinib为移植前患者提供了临床益处,但需要对ruxolitinib在移植算法中的微调和整合进行更多的研究。总之,随着临床重要的风险分层已经在整个疾病过程中实现,新的靶向药物越来越多,需要一种协作和综合的方法来将新的生物学见解转化为骨髓纤维化患者的个性化/量身定制和定时治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Selecting patients with primary myelofibrosis for stem cell transplant using clinical, mutational, and transplant-specific profiles

As with other hematologic malignancies, the management of patients with MF has very much entered the molecular era. The prognostic impact of several driver and nondriver mutations is now well-established; this has obvious relevance to both patient selection for allogeneic hematopoietic cell transplantation and posttransplant outcomes. For transplant, present JAK2 or triple-negative driver mutation genotype together with present ASXL1 mutation may provide best utility for counseling patients with respect to posttransplant outcome, together with clinical (performance status, age, and leukocyte and platelet counts) and transplant-related factors (donor relation). Different conditioning intensities do not seem to play a role with regards to outcome posttransplant but still need to be compared in the molecular era. While ruxolitinib provides clinical benefits for patients before transplant, more studies on the finetuning and integration of ruxolitinib into the transplant algorithm are needed. In conclusion, as clinically important risk stratification has been achieved throughout the disease course and new targeted agents become increasingly available, a collaborative and integrative approach is needed to translate new biological insights to personalized/tailored and timed therapy for patients with myelofibrosis.

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