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异基因造血细胞移植治疗 70 岁及以上骨髓纤维化:来自德国干细胞移植登记处的一项研究。

Allogeneic Hematopoietic Cell Transplantation for Myelofibrosis Aged 70 Years or Older: A Study from the German Registry for Stem Cell Transplantation.

机构信息

Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

German Registry for Stem Cell Transplantation, DRST, Ulm, Germany.

出版信息

Transplant Cell Ther. 2024 Oct;30(10):1011.e1-1011.e13. doi: 10.1016/j.jtct.2024.07.026. Epub 2024 Aug 3.

DOI:10.1016/j.jtct.2024.07.026
PMID:39102984
Abstract

Current consensus recommends hematopoietic cell transplantation (HCT) for patients with myelofibrosis with intermediate or high-risk disease and age of less than 70 years. However, a higher chronological age should not be prohibitive for the eligibility decision in general, acknowledging that current life expectancy for the general population aged 70 years is ∼15 years, and current numbers of patients transplanted at 70 years or older is steadily increasing. The following study aimed to evaluate characteristics and outcomes of HCT in 115 myelofibrosis patients aged 70 years or older. This is a retrospective multicenter study, using the German Registry for Stem Cell Transplantation and Cellular Therapy (DRST). Adult myelofibrosis patients were included who received HCT up until 2021. Patients with secondary leukemia were excluded. Main endpoints were HCT demographics over time and outcomes after HCT (including overall survival, relapse incidence, non-relapse mortality, and graft-versus-host disease/relapse-free survival). Numbers of HCT increased over the past decade, with a significant spike since 2019. Comorbidity status of transplanted patients improved over time, while reduced-intensity conditioning was the preferred HCT platform, especially in most recent years. The 3-year overall survival was 55% (95% confidence interval [CI], 44%-65%). The 1-year cumulative incidence of relapse was 7% (95% CI, 3%-13%) and the 1-year cumulative incidence of non-relapse mortality was 22% (95% CI, 14%-31%). The 3-year graft-versus-host disease and relapse-free survival was 37% (95% CI, 27%-47%). Driver mutation genotype (in particular, non-CALR/MPL genotype) appeared to be the only variable that was significantly and independently associated with better survival in multivariable analysis, whereas neither comorbidity index nor dose intensity of pre-transplant conditioning appeared to influence outcome. This study demonstrated feasibility of curative treatment with HCT for myelofibrosis aged 70 or older, with significant increases in HCT numbers and improved fitness of older adults over recent years.

摘要

目前的共识建议对有中高危疾病且年龄小于 70 岁的骨髓纤维化患者进行造血细胞移植(HCT)。然而,一般来说,较高的年龄不应成为排除资格的原因,要认识到目前 70 岁人群的预期寿命约为 15 年,并且目前 70 岁或以上接受 HCT 的患者数量正在稳步增加。本研究旨在评估 115 例 70 岁或以上骨髓纤维化患者的 HCT 特征和结果。这是一项回顾性多中心研究,使用德国干细胞移植和细胞治疗登记处(DRST)。纳入接受 HCT 的成人骨髓纤维化患者,排除继发性白血病患者。主要终点是过去十年 HCT 的人口统计学数据以及 HCT 后的结果(包括总生存、复发发生率、非复发死亡率以及移植物抗宿主病/无复发生存)。在过去十年中,HCT 的数量有所增加,自 2019 年以来出现显著增长。移植患者的合并症状况随着时间的推移而改善,而减强度预处理是首选的 HCT 平台,尤其是在最近几年。3 年总生存率为 55%(95%置信区间 [CI],44%-65%)。1 年累积复发率为 7%(95%CI,3%-13%),1 年累积非复发死亡率为 22%(95%CI,14%-31%)。3 年移植物抗宿主病和无复发生存为 37%(95%CI,27%-47%)。驱动突变基因型(特别是非 CALR/MPL 基因型)似乎是多变量分析中唯一与生存显著相关的变量,而合并症指数或移植前预处理的剂量强度似乎都不影响结果。本研究表明,对于 70 岁或以上的骨髓纤维化患者,进行 HCT 治疗是可行的,近年来 HCT 的数量显著增加,老年患者的健康状况也得到改善。

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