Combination of umbilical cord mesenchymal stem cells and standard immunosuppressive regimen for pediatric patients with severe aplastic anemia

被引：10

作者：

Lan, Yang ^{[1
]}

Liu, Fang ^{[1
]}

Chang, Lixian ^{[1
]}

Liu, Lipeng ^{[1
]}

Zhang, Yingchi ^{[1
]}

Yi, Meihui ^{[1
]}

Cai, Yuli ^{[1
]}

Feng, Jing ^{[1
]}

Han, Zhibo ^{[1
,2
]}

Han, Zhongchao ^{[1
,2
]}

Zhu, Xiaofan ^{[1
]}

机构：

[1] Chinese Acad Med Sci & Peking Union Med Coll, Natl Clin Res Ctr Blood Dis, State Key Lab Expt Hematol, Inst Hematol & Blood Dis Hosp, 288 Nanjing Rd, Tianjin 300020, Peoples R China

[2] Tianjin AmCellGene Engn Co Ltd, Natl Engn Res Ctr Cell Prod, Tianjin 300020, Peoples R China

来源：

BMC PEDIATRICS | 2021年 / 21卷 / 01期

关键词：

Child; Immunosuppressive therapy; Mesenchymal stem cell; Severe aplastic anemia; Umbilical cord;

D O I：

10.1186/s12887-021-02562-x

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Background: Defects of bone marrow mesenchymal stem cells (BM-MSCs) in proliferation and differentiation are involved in the pathophysiology of aplastic anemia (AA). Infusion of umbilical cord mesenchymal stem cells (UC-MSCs) may improve the efficacy of immunosuppressive therapy (IST) in childhood severe aplastic anemia (SAA). Methods: We conducted an investigator-initiated, open-label, and prospective phase IV trial to evaluate the safety and efficacy of combination of allogenic UC-MSCs and standard IST for pediatric patients with newly diagnosed SAA. In mesenchymal stem cells (MSC) group, UC-MSCs were injected intravenously at a dose of 1 x 10(6)/kg per week starting on the 14th day after administration of rabbit antithymocyte globulin (ATG), for a total of 3 weeks. The clinical outcomes and adverse events of patients with UC-MSCs infusion were assessed when compared with a concurrent control group in which patients received standard IST alone. Results: Nine patients with a median age of 4 years were enrolled as the group with MSC, while the data of another 9 childhood SAA were analysed as the controls. Four (44%) patients in MSC group developed anaphylactic reactions which were associated with rabbit ATG. When compared with the controls, neither the improvement of blood cell counts, nor the change of T-lymphocytes after IST reached statistical significance in MSC group (both p > 0.05) and there were one (11%) patient in MSC group and two (22%) patients in the controls achieved partial response (PR) at 90 days after IST. After a median follow-up of 48 months, there was no clone evolution occurring in both groups. The 4-year estimated overall survival (OS) rate in two groups were both 88.9% +/- 10.5%, while the 4-year estimated failure-free survival (FFS) rate in MSC group was lower than that in the controls (38.1% +/- 17.2% vs. 66.7% +/- 15.7%, p = 0.153). Conclusions: Concomitant use of IST and UC-MSCs in SAA children is safe but may not necessarily improve the early response rate and long-term outcomes.

引用

页数：8

共 32 条

[31] Direct Imaging of Immune Rejection and Memory Induction by Allogeneic Mesenchymal Stromal Cells [J].