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在严重β地中海贫血儿童中,经体内 T 细胞耗竭、清髓性条件化造血干细胞移植后的免疫恢复。

Immune recovery after in vivo T-cell depletion myeloablative conditioning hematopoietic stem cell transplantation in severe beta-thalassemia children.

机构信息

Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China.

Department of Rheumatology and Immunology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China.

出版信息

Eur J Haematol. 2019 Oct;103(4):342-350. doi: 10.1111/ejh.13289. Epub 2019 Aug 6.

Abstract

BACKGROUND

The clinical outcome of hematopoietic stem cell transplantation (HSCT) in those with severe beta-thalassemia (β-TM) is closely related to post-transplantation immune reconstitution (IR). However, the data on the IR in these settings are scarce.

METHODS

A prospective analysis of the clinical outcome and IR in 47 children with severe β-TM who underwent in vivo T-cell depletion myeloablative conditioning and matched sibling donor HSCT was performed. Immune reconstitution, including immune cell subset counts, as well as the generation of new T and B cells assays after HSCT, was measured.

RESULTS

In the first year after HSCT, bacterial infections and cytomegalovirus (CMV) reactivation were observed in 70.2% and 36.2% of the patients, respectively. In the same period, poor CD4 T-cell recovery was observed. The B cells recovered within 6 months. Natural killer (NK) cells recovered as early as 1 month, but their function was defective. Cord blood and bone marrow (CB + BM) group had slower T-cell recovery, and higher B cells and NK cells in comparison with peripheral blood and bone marrow (PB + BM) group.

CONCLUSIONS

The high incidence of infection within 1 year after in vivo T-cell depletion myeloablative conditioning HSCT in severe β-TM was consistent with poor IR.

摘要

背景

造血干细胞移植(HSCT)治疗重型β-地中海贫血(β-TM)患者的临床结局与移植后免疫重建(IR)密切相关。然而,目前针对该人群免疫重建的数据十分有限。

方法

我们对 47 例接受了体内 T 细胞耗竭清髓预处理及同胞全相合 HSCT 的重型β-TM 患儿进行了前瞻性分析,评估了其临床结局和 IR。我们对免疫重建情况进行了检测,包括免疫细胞亚群计数以及移植后新 T 细胞和 B 细胞生成的检测。

结果

在 HSCT 后 1 年内,70.2%的患者发生了细菌感染,36.2%的患者发生了巨细胞病毒(CMV)再激活。同时,我们观察到 CD4+T 细胞恢复不良。B 细胞在 6 个月内恢复。自然杀伤(NK)细胞在 1 个月时即可恢复,但 NK 细胞功能存在缺陷。与外周血和骨髓(PB+BM)组相比,脐血和骨髓(CB+BM)组的 T 细胞恢复较慢,B 细胞和 NK 细胞水平较高。

结论

体内 T 细胞耗竭清髓预处理 HSCT 后 1 年内,重型β-TM 患者感染发生率较高,这与免疫重建不良相一致。

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