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