Shanavas Mohamed, Gupta Vikas
Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center and University of Toronto, 610 University Avenue, Toronto, Ontario, M5G 2M9, Canada.
Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Center and University of Toronto, 610 University Avenue, Toronto, Ontario, M5G 2M9, Canada.
Best Pract Res Clin Haematol. 2014 Jun;27(2):165-74. doi: 10.1016/j.beha.2014.07.007. Epub 2014 Jul 19.
JAK1/2 inhibitors have broadened the therapeutic options in myelofibrosis. Though not curative, they result in a meaningful clinical benefit with relatively fewer side effects. In contrast, allogeneic hematopoietic cell transplantation (HCT) is a potentially curative option, but is associated with significant morbidity and mortality. Hence, an important question is the optimal timing of HCT in the era of JAK inhibitors. Timing of HCT is a crucial decision, and need to be individualized based on the personal preferences and goals of therapy; in addition to patient, disease, and transplant related factors. Risk stratification by the currently established prognostic scoring systems need to be further refined by incorporation of prognostically significant mutations to guide the treatment choices better. Data on use of JAK inhibitors prior to HCT have just started to emerge. We discuss some of the current controversies and dilemmas in transplantation for myelofibrosis based on a few real life scenarios.
JAK1/2抑制剂拓宽了骨髓纤维化的治疗选择。虽然不能治愈,但它们能带来显著的临床益处,且副作用相对较少。相比之下,异基因造血细胞移植(HCT)是一种潜在的治愈选择,但与显著的发病率和死亡率相关。因此,一个重要的问题是在JAK抑制剂时代HCT的最佳时机。HCT的时机是一个关键决策,需要根据个人治疗偏好和目标进行个体化;此外还要考虑患者、疾病和移植相关因素。目前已建立的预后评分系统进行的风险分层需要通过纳入具有预后意义的突变来进一步完善,以更好地指导治疗选择。关于HCT前使用JAK抑制剂的数据刚刚开始出现。我们基于一些实际案例讨论目前骨髓纤维化移植中的一些争议和困境。