Suppr超能文献

比较重型再生障碍性贫血患者亲缘单倍体造血干细胞移植联合移植后环磷酰胺与脐带血移植的疗效

Comparison of Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide versus Umbilical Cord Blood Transplantation in Adult Patients with Aplastic Anemia.

机构信息

Department of Hematology, Tohoku University Hospital.

Department of Hematology, Tokyo Medical and Dental University.

出版信息

Transplant Cell Ther. 2023 Dec;29(12):766.e1-766.e8. doi: 10.1016/j.jtct.2023.09.009. Epub 2023 Sep 18.

Abstract

Aplastic anemia patients who are refractory to immunosuppressive therapy or with very low neutrophil counts require allogeneic hematopoietic stem cell transplantation (HSCT). Umbilical cord blood transplantation (UCBT) has been a treatment option when an HLA-matched donor is not available, and HSCT from a related haploidentical donor using post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis (PTCy-haplo) recently became another important approach. We aimed to compare the outcomes of PTCy-haplo and UCBT in adult patients with aplastic anemia to identify more effective and safer approaches for alternative donor transplantation. Data in a nationwide registry were analyzed retrospectively to assess the outcomes of aplastic anemia patients age ≥16 years who underwent PTCy-haplo or UCBT as their first HSCT between 2016 and 2020. The primary endpoint was 1-year overall survival (OS) after HSCT. Secondary endpoints included 1-year failure-free survival (FFS), neutrophil and platelet engraftment, and acute and chronic GVHD. Eighty-three patients who underwent PTCy-haplo (n = 24) or UCBT (n = 59) were eligible. The 1-year OS rate was 78.5% (95% confidence interval [CI], 55.7% to 90.5%) in the PTCy-haplo group and 77.5% (95% CI, 64.5% to 86.3%; P = .895) in the UCBT group. The 1-year FFS rate was 78.7% (95% CI, 56.1% to 90.6%) in the PTCy-haplo group and 62.2% (95% CI, 48.5% to 73.3%; P = .212) in the UCBT group. Among patients age <40 years, the PTCy-haplo group had a significantly higher FFS rate (92.9% [95% CI, 59.1% to 99.0%]) vs 63.9% [95% CI, 43.2% to 78.7%]; P = .047). Neutrophil engraftment and platelet engraftment rates were significantly higher in the PTCy-haplo group compared with the UCBT group: 95.8% (95% CI, 73.9% to 99.4%) vs 78.0% (95% CI, 65.1% to 86.6%, P < .001) and 83.3% (95% CI, 61.5% to 93.4%) vs 72.9% (95% CI, 59.6% to 82.4%; P = .025). No significant difference was observed in the cumulative incidence of grade II-IV acute GVHD and chronic GVHD between the 2 groups. Aplastic anemia patients achieved significantly higher neutrophil and platelet engraftment rates with PTCy-haplo than with UCBT. OS and the incidences of acute and chronic GVHD were similar between the 2 groups. In patients age <40 years, the FFS rate was higher in the PTCy-haplo group. PTCy-haplo is promising for alternative donor transplantation in adult patients with aplastic anemia.

摘要

再生障碍性贫血患者如果对免疫抑制治疗无反应或中性粒细胞计数非常低,则需要异体造血干细胞移植(HSCT)。当没有 HLA 匹配的供体时,脐带血移植(UCBT)是一种治疗选择,并且最近使用移植后环磷酰胺(PTCy)预防移植物抗宿主病(GVHD)的相关单倍体相合供体 HSCT(PTCy-haplo)也成为另一种重要方法。我们旨在比较 PTCy-haplo 和 UCBT 在再生障碍性贫血成年患者中的结果,以确定更有效和更安全的替代供体移植方法。回顾性分析全国性登记处的数据,以评估 2016 年至 2020 年间接受 PTCy-haplo 或 UCBT 作为其首次 HSCT 的年龄≥16 岁的再生障碍性贫血患者的结果。主要终点是 HSCT 后 1 年的总生存率(OS)。次要终点包括 1 年无失败生存率(FFS)、中性粒细胞和血小板植入、急性和慢性 GVHD。符合条件的 83 例患者接受了 PTCy-haplo(n=24)或 UCBT(n=59)。PTCy-haplo 组 1 年 OS 率为 78.5%(95%CI,55.7%至 90.5%),UCBT 组为 77.5%(95%CI,64.5%至 86.3%;P=0.895)。PTCy-haplo 组 1 年 FFS 率为 78.7%(95%CI,56.1%至 90.6%),UCBT 组为 62.2%(95%CI,48.5%至 73.3%;P=0.212)。在年龄<40 岁的患者中,PTCy-haplo 组的 FFS 率明显更高(92.9%[95%CI,59.1%至 99.0%] vs 63.9%[95%CI,43.2%至 78.7%];P=0.047)。与 UCBT 组相比,PTCy-haplo 组的中性粒细胞植入和血小板植入率显著更高:95.8%(95%CI,73.9%至 99.4%)vs 78.0%(95%CI,65.1%至 86.6%,P<0.001)和 83.3%(95%CI,61.5%至 93.4%)vs 72.9%(95%CI,59.6%至 82.4%;P=0.025)。两组之间 II-IV 级急性 GVHD 和慢性 GVHD 的累积发生率无显著差异。与 UCBT 相比,PTCy-haplo 可使再生障碍性贫血患者获得更高的中性粒细胞和血小板植入率。OS 和急性及慢性 GVHD 的发生率在两组间相似。在年龄<40 岁的患者中,PTCy-haplo 组的 FFS 率更高。PTCy-haplo 是再生障碍性贫血成年患者替代供体移植的有前途的方法。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验