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比较自体、匹配同胞和替代供体干细胞移植治疗急性髓细胞白血病患者缓解后状态的效果:一项倾向评分匹配研究。

Comparison of autologous, matched sibling, and alternative donor stem cell transplant outcomes for acute myeloid leukemia patients in first remission: A propensity score matching study.

机构信息

State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.

Tianjin Institutes of Health Science, Tianjin, China.

出版信息

Hematol Oncol. 2024 Jan;42(1):e3230. doi: 10.1002/hon.3230. Epub 2023 Sep 26.

DOI:10.1002/hon.3230
PMID:37752767
Abstract

Autologous hematopoietic stem cell transplantation (auto-HSCT), matched sibling donor HSCT (MSD-HSCT), and alternative donor HSCT (AD-HSCT) are viable post-remission treatment options for acute myeloid leukemia (AML). A total of 283 de novo favorable- and intermediate-risk AML patients, based on the ELN 2022 criteria, in first complete remission were initially included for propensity score matching. Following the matching process, 126 patients were selected for further analysis, with 42 patients in each of the auto-HSCT, MSD-HSCT, and AD-HSCT groups. Among the AD-HSCT group, 38 of 42 (90.5%) patients received haploidentical HSCT. In patients with persistent undetectable measurable residual disease (uMRD) before transplant (n = 83), overall survival (OS) was similar across the groups. However, auto-HSCT showed a trend of increased disease-free survival (DFS) compared to AD-HSCT (HR 2.85, P = 0.09), resulting in a 3-year DFS and OS of 79.1% and 82.8%, respectively. In the non-persistent uMRD group (n = 38), auto-HSCT exhibited a tendency to increase the risk of relapse, particularly when compared to AD-HSCT (HR 0.24, P = 0.07), but this did not result in inferior OS. The monthly direct medical cost per patient within the first 2 years after HSCT was significantly lower in auto-HSCT compared to MSD-HSCT (P = 0.015) and AD-HSCT (P < 0.001). Our results provide evidence for the use of auto-HSCT as a viable therapeutic option for favorable- and intermediate-risk de novo AML patients in first complete remission with persistent uMRD. Additionally, our findings demonstrated a notable cost advantage associated with auto-HSCT compared to MSD-HSCT and AD-HSCT.

摘要

自体造血干细胞移植(auto-HSCT)、匹配同胞供体 HSCT(MSD-HSCT)和替代供体 HSCT(AD-HSCT)是急性髓系白血病(AML)缓解后可行的治疗选择。根据 2022 年 ELN 标准,共有 283 例新发低危和中危 AML 患者在首次完全缓解时被纳入倾向评分匹配。匹配过程后,选择了 126 例患者进行进一步分析,每组 42 例,分别为 auto-HSCT、MSD-HSCT 和 AD-HSCT 组。在 AD-HSCT 组中,42 例患者中有 38 例(90.5%)接受了半相合 HSCT。在移植前持续检测不到可测量残留疾病(uMRD)的患者(n=83)中,各组的总生存率(OS)相似。然而,与 AD-HSCT 相比,auto-HSCT 显示出改善无病生存率(DFS)的趋势(HR 2.85,P=0.09),导致 3 年 DFS 和 OS 分别为 79.1%和 82.8%。在非持续性 uMRD 组(n=38)中,与 AD-HSCT 相比,auto-HSCT 有增加复发风险的趋势(HR 0.24,P=0.07),但并未导致 OS 下降。HSCT 后前 2 年内,每个患者的每月直接医疗费用在 auto-HSCT 组显著低于 MSD-HSCT 组(P=0.015)和 AD-HSCT 组(P<0.001)。我们的结果为在首次完全缓解时持续存在 uMRD 的新发低危和中危 AML 患者使用 auto-HSCT 作为可行的治疗选择提供了证据。此外,我们的研究结果表明,与 MSD-HSCT 和 AD-HSCT 相比,auto-HSCT 具有显著的成本优势。

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