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异基因造血干细胞移植治疗白血病转化型骨髓纤维化:来自欧洲血液和骨髓移植协会细胞与基因治疗工作组骨髓增殖性肿瘤学组的研究。

Allogeneic stem cell transplantation for myelofibrosis with leukemic transformation: a study from the Myeloproliferative Neoplasm Subcommittee of the CMWP of the European Group for Blood and Marrow Transplantation.

机构信息

Department of Stem Cell Transplantation, University Hospital Hamburg, Hamburg, Germany.

EBMT Data Office, Leiden University Medical Center, Leiden, The Netherlands.

出版信息

Biol Blood Marrow Transplant. 2014 Feb;20(2):279-81. doi: 10.1016/j.bbmt.2013.10.027. Epub 2013 Nov 4.

Abstract

Transformed acute myeloid leukemia in myelofibrosis results in a median survival of less than 5 months. We identified 46 of 1048 myelofibrosis patients in the European Group for Blood and Marrow Transplantation registry who received allogeneic stem cell transplantation for acute leukemia evolving from myelofibrosis. The cumulative incidence of treatment-related mortality at 1 year was 28% (95% confidence interval, 14 to 42) and of relapse at 3 years was 47% (95% confidence interval, 31 to 63). The 3-year progression-free (PFS) and overall survival (OS) rates were 26% and 33%, respectively. The only significant factor for survival was complete remission versus no complete remission before transplantation (69% versus 22%, P = .008); however, complete remission was achieved only in 8 patients. Allogeneic stem cell transplantation can cure myelofibrosis patients transformed to leukemia.

摘要

骨髓纤维化继发的转化型急性髓系白血病中位生存时间不足 5 个月。我们在欧洲血液和骨髓移植学会注册中心的 1048 例骨髓纤维化患者中发现 46 例患者继发骨髓纤维化的急性白血病接受了异基因造血干细胞移植。1 年时治疗相关死亡率的累积发生率为 28%(95%置信区间,14 至 42),3 年时复发率为 47%(95%置信区间,31 至 63)。3 年无进展生存(PFS)率和总生存(OS)率分别为 26%和 33%。唯一对生存有显著影响的因素是移植前是否达到完全缓解(69%比 22%,P=.008);然而,仅有 8 例患者达到完全缓解。异基因造血干细胞移植可治愈转化为白血病的骨髓纤维化患者。

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