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X 连锁慢性肉芽肿病患者播散性卡介苗感染行成功无关供体脐带血干细胞移植

Successful Unrelated Cord Blood Stem Cell Transplantation in an X-linked Chronic Granulomatous Disease Patient with Disseminated BCG-induced Infection.

机构信息

Department of Pediatrics, Changhua Christian Children's Hospital, Changhua, Taiwan, ROC; School of Medicine, Chung Shan Medical University, Taichung, Taiwan, ROC.

Department of Pediatrics, Changhua Christian Children's Hospital, Changhua, Taiwan, ROC.

出版信息

Pediatr Neonatol. 2015 Oct;56(5):346-50. doi: 10.1016/j.pedneo.2013.04.001. Epub 2013 May 13.

DOI:10.1016/j.pedneo.2013.04.001
PMID:23680261
Abstract

A 19-month-old boy with chronic granulomatous disease (CGD) received umbilical cord blood transplantation (UCBT) from an unrelated donor after experiencing a life-threatening disseminated Bacillus Calmette-Guérin infection. After busulfan and cyclophosphamide conditioning, we performed a 5/6-matched UCBT. Engraftment and mixed chimerism was 100% in peripheral blood, and 100% of his neutrophils had normal oxidative burst activity on day 17. The patient is now 3 years old, free from infection, and growing well. To our knowledge, this is the second case of CGD treated with UCBT in Taiwan. His successful outcome illustrates that UCBT in a patient with CGD should be considered early if a human leukocyte antigen-matched donor is not available or the patient has just recovered from a severe infection.

摘要

一名 19 个月大的男孩患有慢性肉芽肿病(CGD),在经历了危及生命的卡介苗播散性感染后,他接受了来自无关供体的脐带血移植(UCBT)。在接受了白消安和环磷酰胺预处理后,我们进行了 5/6 配型的 UCBT。外周血中 100%发生了嵌合,并且他的中性粒细胞在第 17 天 100%具有正常的氧化爆发活性。该患者现在 3 岁,没有感染,生长良好。据我们所知,这是台湾用 UCBT 治疗 CGD 的第二例病例。他的成功结果表明,如果没有人类白细胞抗原匹配的供体,或者患者刚刚从严重感染中恢复过来,应考虑早期对 CGD 患者进行 UCBT。

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