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Allogeneic stem cell transplant for myelofibrosis patients over age 60: likely impact of the JAK2 inhibitors
Leukemia Supplements Pub Date : 2012-05-09 , DOI: 10.1038/leusup.2012.2
V Fauble 1 , J Leis 1 , R A Mesa 1
Affiliation  

The myeloproliferative neoplasm, myelofibrosis (MF), has only one therapeutic intervention that is potentially curative in these individuals, specifically that of allogeneic stem cell transplantation (ASCT). ASCT has been utilized up to this juncture, primarily in younger individuals with higher risk disease. There is more limited data on outcomes in individuals over the age of 60 years. The choice of an individualized therapeutic intervention for a patient with MF is a very complex issue and is dependent on several factors. The first factor being their overall prognosis with their illness (which can vary from a median of 2 years in high-risk patients to over 10 years in low-risk patients) and the potential impact of a therapeutic intervention not only on survival but also on quality of life. Current available therapies have been strictly palliative for disease-associated anemia and/or splenomegaly. At present, we have a new generation of inhibitors of JAK2 (Ruxolitinib, CYT387, SB1518, TG101348, with others in development), which have been shown to improve splenomegaly, improve symptomatic burden of illness and improve quality of life. In addition, these inhibitors of JAK2 may have an impact on the natural history of MF, but confirmation of the presence and degree of this impact is still pending. Clinical availability of JAK2 inhibitors may alter the timing of transplant in marginal transplant candidates (that is, those over the age of 60), may have a role preceding ASCT to improve spleen size and performance status before transplant and might be frontline therapy in intermediate and high-risk patients who are not candidates for ASCT.



中文翻译:

60 岁以上骨髓纤维化患者的异基因干细胞移植:JAK2 抑制剂的可能影响

骨髓增生性肿瘤,骨髓纤维化 (MF),只有一种治疗干预对这些个体具有潜在的治愈作用,特别是同种异体干细胞移植 (ASCT)。到目前为止,ASCT 已被使用,主要用于患有高风险疾病的年轻人。关于 60 岁以上个体结果的数据更为有限。为 MF 患者选择个体化治疗干预是一个非常复杂的问题,并且取决于几个因素。第一个因素是他们对疾病的总体预后(可能从高危患者的中位 2 年到低危患者的 10 年以上)以及治疗干预不仅对生存而且对生活质量。目前可用的疗法已严格缓解疾病相关性贫血和/或脾肿大。目前,我们有新一代的 JAK2 抑制剂(鲁索替尼、CYT387、SB1518、TG101348,以及其他正在开发中),已被证明可改善脾肿大、改善症状负担并提高生活质量。此外,这些 JAK2 抑制剂可能对 MF 的自然病程有影响,但这种影响的存在和程度仍有待确认。JAK2 抑制剂的临床可用性可能会改变边缘移植候选者(即 60 岁以上)的移植时机,可能在 ASCT 之前发挥作用,以改善移植前的脾脏大小和体能状态,并且可能是中间和不适合进行 ASCT 的高危患者。

更新日期:2012-05-09
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