Unrelated Cord Blood Transplantation for Patients with Primary or Secondary Myelofibrosis
Unrelated Cord Blood Transplantation for Patients with Primary or Secondary Myelofibrosis
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DOI：
10.1016/j.bbmt.2014.06.011
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发表时间：
2014-11-01
影响因子：
4.3
通讯作者：
Ruggeri, Annalisa
中科院分区：
文献类型：
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作者：
Robin, Marie;Giannotti, Federica;Ruggeri, Annalisa
为了确定脐带血移植（UCBT）是否是骨髓纤维化（MF）的替代治疗方法，我们评估了Eurocord报告的35例脐带血移植。7例患者在UCBT时患有继发性急性髓性白血病（AML）， UCBT时的中位年龄为54岁。24例患者接受降低强度调节（RIC）方案，35例患者中有17例接受全身照射（2至12 Gy）-氟达拉滨-环磷酰胺（TCF）调节。中位随访时间为24个月。60天中性粒细胞恢复的累积发生率为80%。15例患者术后复发。2年总生存率和无事件生存率（EFS）分别为44%和30%。所有给予TCF的患者均实现了中性粒细胞和血小板恢复，并且在RIC人群中，TCF的使用与较好的EFS相关（44%对0%，P = 0.001）。转化为急性髓性白血病的患者与非晚期患者的结果相似。总之，尽管移植物衰竭仍然是一个主要问题，但UCBT在MF管理中的作用，特别是使用基于RIC tcf的方案，值得进一步研究以改善结果。(C) 2014年美国血液和骨髓移植学会。
To determine whether umbilical cord blood transplantation (UCBT) is an alternative cure for myelofibrosis (MF), we evaluated 35 UCBTs reported to Eurocord. Seven patients had secondary acute myeloid leukemia (AML) at UCBT, and median age at UCBT was 54 years. Twenty-four patients received a reduced-intensity conditioning (RIC) regimen, and 17 of 35 patients received total body irradiation (2 to 12 Gy)-fludarabine-cyclophosphamide (TCF) conditioning. The median follow-up was 24 months. The cumulative incidence of neutrophil recovery at 60 days was 80%. Fifteen patients relapsed after UCBT. The 2-year overall survival and event-free-survival (EFS) rates were 44% and 30%, respectively. All patients given TCF achieved neutrophil and platelet recovery, and the use of TCF was associated with superior EFS in the RIC population (44% versus 0%, P =.001). Patients with transformation to AML had similar outcomes to patients with less advanced stages. In conclusion, despite graft failure remaining a major concern, the role of UCBT in the management of MF, especially using RIC TCF-based regimens, deserves further investigation to improve results. (C) 2014 American Society for Blood and Marrow Transplantation.