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缓解期急性白血病患者原发移植物失败后行第二次异基因造血干细胞移植(HSCT2)的长期疗效:一项代表欧洲血液和骨髓移植学会急性白血病工作组的研究。

Long-term outcome of second allogeneic hematopoietic stem cell transplantation (HSCT2) for primary graft failure in patients with acute leukemia in remission: A study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.

机构信息

Division of Hematology, Sheba Medical Center, Tel Hashomer, Israel.

EBMT Paris study office; Department of Hematology, Saint Antoine Hospital; INSERM UMR 938, Sorbonne University, Paris, France.

出版信息

Bone Marrow Transplant. 2023 Sep;58(9):1008-1016. doi: 10.1038/s41409-023-02012-5. Epub 2023 May 30.

Abstract

Second transplantation (HSCT2) is a potential treatment for primary graft failure (pGF). We assessed the outcome of HSCT2, performed between 2000 and 2021, for pGF in 243 patients with acute leukemia. Median age was 44.8 years. Conditioning at first HSCT (HSCT1) was myeloablative (MAC) in 58.4%. Median time from HSCT1 to HSCT2 was 48 days. Donors for HSCT2 were the same as for HSCT1 in 49%. Engraftment post HSCT2 was achieved by 73.7% of patients. The incidence of acute (a) graft versus host disease (GVHD) grades II-IV and III-IV was 23.2 and 8.1%. 5-year total and extensive chronic (c) GVHD was 22.3 and 10.1%. 5-year nonrelapse mortality (NRM), relapse incidence (RI), leukemia-free survival (LFS), overall survival (OS) and GVHD free, relapse-free survival (GRFS) was 51.6, 18.8, 29.6, 30.7 and 22.4%, respectively. Infections were the main cause of death. In multivariable analysis, being transplanted at second vs. first remission, lower Karnofsky performance status (KPS; <90) and receiving MAC at HSCT1 were adverse prognostic factors for NRM, LFS, OS, and GRFS, as was increased age for NRM, LFS, OS. We conclude that HSCT2 can rescue about a third of the patients who experienced pGF, but NRM is as high as 50%.

摘要

二次移植(HSCT2)是原发性移植物失败(pGF)的潜在治疗方法。我们评估了 243 例急性白血病患者在 2000 年至 2021 年间进行的 pGF 二次移植(HSCT2)的结果。中位年龄为 44.8 岁。首次 HSCT(HSCT1)的预处理为清髓性(MAC)占 58.4%。HSCT2 至 HSCT1 的中位时间为 48 天。HSCT2 的供者与 HSCT1 相同的占 49%。73.7%的患者在 HSCT2 后实现了植入。急性(a)移植物抗宿主病(GVHD)Ⅱ-Ⅳ级和Ⅲ-Ⅳ级的发生率分别为 23.2%和 8.1%。5 年总发病率和广泛慢性(c)GVHD 分别为 22.3%和 10.1%。5 年非复发死亡率(NRM)、复发率(RI)、无白血病生存率(LFS)、总生存率(OS)和 GVHD 无复发率(GRFS)分别为 51.6%、18.8%、29.6%、30.7%和 22.4%。感染是死亡的主要原因。在多变量分析中,与首次缓解相比,在第二次缓解时进行移植、Karnofsky 表现状态(KPS;<90)较低和在 HSCT1 时接受 MAC 预处理是 NRM、LFS、OS 和 GRFS 的不良预后因素,年龄增加也是 NRM、LFS、OS 的不良预后因素。我们的结论是,HSCT2 可以挽救约三分之一经历 pGF 的患者,但 NRM 高达 50%。

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