首次完全缓解的老年急性髓系白血病患者的造血细胞移植：随机 III 期研究的结果。
Hematopoietic cell transplantation for older acute myeloid leukemia patients in first complete remission: results of a randomized phase III study.
发表日期：2024 Aug 08
作者：
Dietger Niederwieser, Dirk Hasenclever, Wolfgang E. Berdel, Bart J Biemond, Haifa Al-Ali, Yves Chalandon, Michel Van Gelder, Christian Junghanß, Gösta Gahrton, Mathias Hänel, Rüdiger Hehlmann, Thomas Heinicke, Andreas Hochhaus, Simona Iacobelli, Rien van Marwijk Kooy, Nicolaus Kröger, Jeroen Janssen, Madlen Jentzsch, Frank Breywisch, Mohamad Mohty, Stavroula Masouridi-Levrat, Gert Ossenkoppele, Jacob Passweg, Wolfram Pönisch, Johannes Schetelig, Christoph Schliemann, Sebastian Schwind, Matthias Stelljes, Leo F Verdonck, Vladan Vucinic, Bob Löwenberg, Jan Cornelissen
来源：
HAEMATOLOGICA
摘要：
鉴于选择首次完全缓解 (CR1) 的老年 AML 患者，同种异体造血细胞移植 (HCT) 巩固治疗相对于化疗的优势仍不清楚。对年龄为 60-75 岁的 CR1 期新诊断 AML 患者进行了登记，并启动了供体搜索。一个巩固周期后，有匹配供体的患者被随机分配接受氟达拉滨/低剂量全身照射和环孢素/吗替麦考酚酯免疫抑制的 HCT 或传统非 HCT。主要结局是最长五年的平均无白血病生存期（RM-LFS）。 2010 年至 2017 年间，CR1 登记了 245 名患者（中位年龄 67 岁）。一次巩固后，26.9% 的患者未达到纳入标准。在仍在接受研究的 179 名患者 (73%) 中，75.4% 的患者拥有相同的 HLA 供体。排除了 10 名不符合条件的患者，其中 125 名患者随机接受 HCT (n=83) 或非 HCT (n=42)。长达 5 年的主要结局 RM-LFS 在 HCT 组中为 24.5 个月（95%CI：18.9-30.1），在非 HCT 组中为 15.6 个月（95%CI：10.4-20.8）（p=0.022）。累积复发率从非 HCT 后的 91.1 (95%CI:80.7-100.0) 降低至 HCT 后的 37.8 (95%CI:27.2-48.4)% (p. 11)
Given the selection of elderly patients with AML in first complete remission (CR1) the advantage of consolidation with allogeneic hematopoietic cell transplantation (HCT) over chemotherapy is still unclear. Newly diagnosed AML patients in CR1 aged 60-75 years were registered and a donor search initiated. After one consolidation cycle, patients with a matched donor were randomized to HCT with fludarabine/lowdose total body irradiation and cyclosporine/mycophenolate mofetil immunosuppression or conventional non-HCT. Primary outcome was restricted mean leukemia-free survival (RM-LFS) up to five years. Between 2010 and 2017, 245 patients (median age 67 years) were registered at CR1. After one consolidation, 26.9% of patients failed inclusion criteria. Of the 179 (73%) patients still on study, 75.4% had an HLA identical donor. Ten ineligible patients were excluded, and 125 randomized to HCT (n=83) or non-HCT (n=42). The primary outcome RM-LFS up to 5 years was 24.5 months (95%CI:18.9-30.1) in the HCT and 15.6 months (95%CI:10.4-20.8) in the non-HCT arm (p=0.022) due to a decrease in cumulative relapse incidence from 91.1 (95%CI:80.7-100.0) after non-HCT to 37.8 (95%CI:27.2-48.4)% after HCT (p.