Janus kinase inhibitors and allogeneic stem cell transplantation for myelofibrosis.
Janus kinase inhibitors and allogeneic stem cell transplantation for myelofibrosis.
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Janus 激酶抑制剂和同种异体干细胞移植治疗骨髓纤维化。
DOI：
10.1016/j.bbmt.2014.03.017
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Deeg,HJoachim
中科院分区：
文献类型：
--
作者：
Gupta,Vikas;Gotlib,Jason;Radich,JeraldP;Kröger,NicolausM;Rondelli,Damiano;Verstovsek,Srdan;Deeg,HJoachim
骨髓纤维化（MF）是多种造血功能障碍的表现，统称为骨髓增殖性肿瘤。同种异体造血干细胞移植（ASCT）是唯一具有已证实具有治疗潜力的疗法。然而，大多数 MF 患者年龄在 6 岁或 7 岁，其中只有部分患者被认为是合适的移植候选者。毒性有限的低强度预处理方案的开发使得临床医生能够为越来越多的老年患者提供 ASCT。 Janus 激酶 (JAK) 1/2 抑制剂的出现使临床医生能够缓解 MF 患者的症状并提高生活质量。这些药物也可能影响有关 ASCT 时间的决定。未来的研究需要解决 JAK1/2 抑制剂在移植候选患者中的作用，并确定它们在移植前以及可能在移植后的作用。确定移植中使用 JAK1/2 抑制剂的适应症可能会为 MF 患者带来新的治疗策略。
Myelofibrosis (MF) is a manifestation of several disorders of hematopoiesis, collectively referred to as myeloproliferative neoplasms. Allogeneic hematopoietic stem cell transplantation (ASCT) is the only therapy with proven curative potential. However, most patients with MF are in their 6th or 7th decade of life, and only some of these patients have been considered suitable transplantation candidates. The development of reduced-intensity conditioning regimens with limited toxicity has allowed clinicians to offer ASCT to a growing number of older patients. The availability of Janus Kinase (JAK) 1/2 inhibitors allows clinicians to provide symptom relief and improved quality of life for MF patients. These drugs may also affect the decision regarding, in particular, the timing of ASCT. Future studies need to address the role of JAK1/2 inhibitors in patients who are transplantation candidates and determine their role before and, possibly, after transplantation. The identification of indications for the use of JAK1/2 inhibitors in the context of transplantation may lead to new therapeutic strategies for patients with MF.