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更新的脐带血移植治疗慢性粒单核细胞白血病的可比疗效:一项全国性研究。

Updated comparable efficacy of cord blood transplantation for chronic myelomonocytic leukaemia: a nationwide study.

机构信息

Department of Hematology, Yokohama Municipal Citizen's Hospital, Yokohama, Japan.

Department of Hematology, Kobe City Hospital Organization Kobe City Medical Center General Hospital, Kobe, Japan.

出版信息

Bone Marrow Transplant. 2024 Jun;59(6):742-750. doi: 10.1038/s41409-024-02223-4. Epub 2024 Feb 8.

Abstract

Chronic myelomonocytic leukaemia (CMML) is a haematological malignancy with a poor prognosis. Allogeneic haematopoietic stem cell transplantation remains the only curative approach. Without human leucocyte antigen-matched related sibling donors, the optimal alternative donor has yet to be established. Although unrelated bone marrow transplantation (UBMT) has been extensively studied, cord blood transplantation (CBT) for CMML remains largely unexplored. This nationwide retrospective study compared the outcomes of UBMT and single-unit umbilical CBT in patients with CMML. This study included 118 patients who underwent their first allo-HSCT during 2013-2021. Of these, 50 received BMT (UBMT group), while 68 underwent CBT (CBT group). The primary endpoint was the 3-year overall survival (OS). There were comparable 3-year OS rates between the UBMT (51.0%, 95% confidence interval [CI]: 34.1-65.5%) and CBT (46.2%, 95% CI: 33.2-58.1%; P = 0.60) groups. In the inverse probability of treatment weighting analysis, CBT did not show significantly improved outcomes compared with UBMT regarding the 3-year OS rate (hazard ratio 0.97 [95% CI: 0.57-1.66], P = 0.91). Thus, CBT may serve as an alternative to UBMT for patients with CMML. Further research is necessary to optimise transplantation strategies and enhance outcomes in patients with CMML undergoing CBT.

摘要

慢性髓单核细胞白血病(CMML)是一种预后不良的血液系统恶性肿瘤。异基因造血干细胞移植仍然是唯一的治愈方法。在没有人类白细胞抗原匹配的相关亲属供体的情况下,尚未确定最佳的替代供体。虽然已经广泛研究了无关供体骨髓移植(UBMT),但 CMML 的脐带血移植(CBT)仍在很大程度上未被探索。这项全国性回顾性研究比较了 CMML 患者接受 UBMT 和单份脐带血 CBT 的结果。这项研究纳入了 118 名在 2013 年至 2021 年期间接受首次同种异体 HSCT 的患者。其中,50 名患者接受了 BMT(UBMT 组),而 68 名患者接受了 CBT(CBT 组)。主要终点是 3 年总生存率(OS)。UBMT 组(51.0%,95%置信区间 [CI]:34.1-65.5%)和 CBT 组(46.2%,95% CI:33.2-58.1%;P=0.60)的 3 年 OS 率相当。在逆概率治疗加权分析中,与 UBMT 相比,CBT 并没有显著改善 3 年 OS 率(风险比 0.97 [95% CI:0.57-1.66],P=0.91)。因此,CBT 可能是 CMML 患者替代 UBMT 的一种选择。需要进一步研究以优化移植策略并提高接受 CBT 的 CMML 患者的结果。

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