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噻替派-白消安-氟达拉滨作为异基因造血移植治疗骨髓纤维化患者的预处理方案:单中心经验。

Thiotepa-busulfan-fludarabine as a conditioning regimen for patients with myelofibrosis undergoing allogeneic hematopoietic transplantation: a single center experience.

机构信息

Department of Hematology and Cellular Therapy, Saint Antoine Hospital, AP-HP, Paris, France.

Department of Medicine and Surgery, Hematology and Hematopoietic Stem Cell Transplant Center, University of Naples Federico II, Naples, Italy.

出版信息

Leuk Lymphoma. 2021 Feb;62(2):419-427. doi: 10.1080/10428194.2020.1827246. Epub 2020 Oct 5.

Abstract

We assessed the outcomes associated with thiotepa, busulfan and fludarabine (TBF) conditioning regimen in a cohort of 29 consecutive patients allografted for myelofibrosis (MF). The median age was 56 (range 42-70) years. According to the refined Dynamic International Prognostic Scoring System (DIPSS-plus), 15 (52%) patients were classified as high risk. Graft source was peripheral blood stem cells in 27 patients. Donor type was HLA-matched related ( = 5), matched unrelated ( = 16), mismatched unrelated ( = 1), and haploidentical ( = 7). All but 2 patients engrafted. The cumulative incidence (CI) of grade II-IV acute graft-versus-host disease (GVHD) was 21% (95% CI, 10-42) at day 100. The CI of chronic GVHD was 39% (95% CI, 23-65) at 3 years. The median follow-up period was 39 (range 14-60) months. Overall survival was 69% (95% CI, 50-83) at 3 years. No relapse was observed. TBF is a valid conditioning strategy in patients with MF.

摘要

我们评估了 29 例接受硫替哌、白消安和氟达拉滨(TBF)预处理方案的骨髓纤维化(MF)患者的结局。这些患者均接受了异基因造血干细胞移植。中位年龄为 56 岁(范围 42-70 岁)。根据改良的动态国际预后评分系统(DIPSS-plus),15 例(52%)患者被归类为高危。27 例患者的移植物来源为外周血造血干细胞。供者类型为 HLA 匹配的亲缘( = 5)、匹配的无关( = 16)、不匹配的无关( = 1)和单倍体相合( = 7)。除 2 例患者外,其余患者均植入成功。100 天时,Ⅱ-Ⅳ级急性移植物抗宿主病(GVHD)的累积发生率(CI)为 21%(95%CI,10-42)。3 年时慢性 GVHD 的 CI 为 39%(95%CI,23-65)。中位随访时间为 39 个月(范围 14-60 个月)。3 年时的总生存率为 69%(95%CI,50-83)。未观察到疾病复发。TBF 是 MF 患者的一种有效预处理方案。

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