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比较非亲缘单份脐带血与“体外”T 细胞去除的单倍体相合移植治疗血液系统恶性肿瘤患儿的临床结局。

Comparison of clinical outcomes between unrelated single umbilical cord blood and "ex-vivo" T-cell depleted haploidentical transplantation in children with hematological malignancies.

机构信息

Department of Pediatrics, Hematopoietic Stem Cell Transplantation Unit, Hospital Infantil Universitario "Niño Jesus", Menedez Pelayo 65, 28009, Madrid, Spain.

Hospital Infantil Universitario "La Paz" Madrid, Madrid, Spain.

出版信息

World J Pediatr. 2021 Dec;17(6):609-618. doi: 10.1007/s12519-021-00461-w. Epub 2021 Sep 30.

Abstract

BACKGROUND

Over the last two decades, umbilical cord blood (UCB) and haploidentical transplantation (HaploHSCT) have emerged as alternative sources of hematopoietic stem cell for allogeneic transplantation. There are few retrospective studies and no prospective studies comparing both types of alternative transplantation in pediatric patients.

RESULTS

We analyzed the data of 134 children with hematological malignancies who received a hematopoietic stem cell transplantation from a single umbilical cord blood (UCB) (n = 42) or an "ex-vivo" T-cell depleted transplant from a haploidentical-related donor (HaploHSCT) (n = 92) between 1996 and 2014. Hematological recovery was faster after HaploHSCT than the UCB transplant group (median times to neutrophil and platelet recovery: 13 vs. 16 days, 10 vs. 57 days, respectively) (P < 0.001). The HaploHSCT group had a significantly early immune reconstitution based on NK and CD8 + T cells compared with the UCB group. However, after the first year post-transplantation, HaploHSCT had a lower number of CD4 + T and B lymphocytes compared with the UCB transplant recipients. The cumulative incidence of TRM was 29±8% in the HaploHSCT group versus 40±5% in the UCB group. Relapse incidence was 21±7% in the HaploHSCT group and 19±8% in the UCB group. Probability of DFS was 58±8% in the HaploHSCT group versus 40±9% in the UCB group (P = 0.051).

CONCLUSIONS

TCD haploidentical transplant is associated with advantages in terms of engraftment and early immune reconstitution kinetics. TCD haploidentical transplant was associated with lower incidence of infectious and non-infectious complications, especially in the early phases of the transplant compared with UCB transplant recipients. However, there are no advantages in transplant outcomes compared with UCB transplant.

摘要

背景

在过去的二十年中,脐带血(UCB)和单倍体相合移植(HaploHSCT)已成为同种异体移植中造血干细胞的替代来源。很少有回顾性研究,也没有前瞻性研究比较儿科患者这两种替代移植类型。

结果

我们分析了 1996 年至 2014 年间 134 例患有血液系统恶性肿瘤的儿童的数据,他们接受了来自单个脐带血(UCB)(n=42)或来自单倍体相关供体的“体外”T 细胞耗竭移植(HaploHSCT)(n=92)的造血干细胞移植。HaploHSCT 组的血液学恢复速度快于 UCB 移植组(中性粒细胞和血小板恢复的中位数时间:13 天与 16 天,10 天与 57 天,分别)(P<0.001)。与 UCB 组相比,HaploHSCT 组具有基于 NK 和 CD8+T 细胞的更早的免疫重建。然而,在移植后第一年,HaploHSCT 组的 CD4+T 和 B 淋巴细胞数量低于 UCB 移植受者。HaploHSCT 组的 TRM 累积发生率为 29±8%,而 UCB 组为 40±5%。HaploHSCT 组的复发率为 21±7%,而 UCB 组为 19±8%。HaploHSCT 组的 DFS 概率为 58±8%,而 UCB 组为 40±9%(P=0.051)。

结论

TCD 单倍体相合移植在植入和早期免疫重建动力学方面具有优势。与 UCB 移植受者相比,TCD 单倍体相合移植与较低的感染和非感染性并发症发生率相关,特别是在移植的早期阶段。然而,与 UCB 移植相比,移植结果没有优势。

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