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造血干细胞移植治疗儿童重型再生障碍性贫血植活失败的疗效分析。

Successful outcomes of second hematopoietic stem cell transplantation for graft failure in pediatric patients with severe aplastic anemia.

机构信息

Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, 450003, Henan, China.

The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450003, Henan, China.

出版信息

Sci Rep. 2022 Jun 22;12(1):10528. doi: 10.1038/s41598-022-14665-1.

Abstract

Severe aplastic anemia (SAA) is a life-threatening hematological disorder. The major therapies include matched sibling donor (MSD)- hematopoietic stem cell transplantation (HSCT), matched unrelated donor (MUD)-HSCT and immunosuppressive therapy (IST). However, there are many problems that can occur after HSCT, and graft failure (GF) is one of the most serious complications. To find an effective treatment, we analyzed 10 cases of second HSCT to treat SAA pediatric patients who suffered from GF and concluded that second haploidentical family donors HSCT is an effective treatment. Moreover, adding a small dose of busulfan or 2 ~ 3 Gy total body irradiation (TBI) in nonmyeloablative regimens (NMAs) can promote the engraftment. Although the study also showed that PBSCs, as a source of stem cells, can promote the implantation of neutrophil cells, due to small sample size, more research is still needed.

摘要

重型再生障碍性贫血(SAA)是一种危及生命的血液系统疾病。主要治疗方法包括同胞供者(MSD)-造血干细胞移植(HSCT)、非血缘供者(MUD)-HSCT 和免疫抑制治疗(IST)。然而,HSCT 后会出现许多问题,移植物失败(GF)是最严重的并发症之一。为了找到有效的治疗方法,我们分析了 10 例接受第二 HSCT 治疗 GF 的 SAA 儿科患者,得出结论,第二单倍体家族供者 HSCT 是一种有效的治疗方法。此外,在非清髓性方案(NMA)中添加小剂量白消安或 2~3Gy 全身照射(TBI)可促进植入。尽管该研究还表明,作为干细胞来源的 PBSCs 可促进中性粒细胞细胞的植入,但由于样本量小,仍需要更多的研究。

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