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慢性肉芽肿病患者成功接受无关供者脐血移植。

Successful unrelated donor cord blood transplantation for chronic granulomatous disease.

机构信息

Division of Hematology and Oncology, Department of Pediatrics, Chang Gung Children's Hospital, Chang Gung University, Taoyuan, Taiwan.

出版信息

Int J Hematol. 2010 May;91(4):670-2. doi: 10.1007/s12185-010-0537-5. Epub 2010 Mar 12.

DOI:10.1007/s12185-010-0537-5
PMID:20224873
Abstract

This report exemplified a success of unrelated donor cord blood transplantation (CBT) in a 4-month-old boy with chronic granulomatous disease (CGD). Following Bu14/Cy200/ATG conditioning, the patient received an HLA 2-locus-mismatched cord blood unit, and the total number of infused nucleated cells was 11.52 x 10(7)/kg. Neutrophil engraftment was achieved on day +13, and a platelet count greater than 20 x 10(9)/L was achieved on day +50. The neutrophil oxidative burst was 100% normal with full donor lymphocyte reconstitution at 8 months post-transplant. This case highlights that unrelated donor CBT for CGD is potentially safe and feasible, even in early infancy, when an appropriately matched related or unrelated donor is unavailable.

摘要

本报告举例说明了一名 4 个月大的慢性肉芽肿病(CGD)男童进行无关供者脐血移植(CBT)的成功案例。在 Bu14/Cy200/ATG 预处理后,该患者接受了一个 HLA 2 位点不合的脐血单位,输注的有核细胞总数为 11.52 x 10(7)/kg。移植后第 13 天中性粒细胞植入,第 50 天血小板计数大于 20 x 10(9)/L。移植后 8 个月时,中性粒细胞氧化爆发完全正常,完全供者淋巴细胞重建。本案例强调了即使在早期婴儿期,当无法获得合适匹配的相关或无关供者时,无关供者 CBT 治疗 CGD 也是安全可行的。

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引用本文的文献

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Biol Blood Marrow Transplant. 2012 Sep;18(9):1368-77. doi: 10.1016/j.bbmt.2012.02.002. Epub 2012 Feb 10.
2
Chronic granulomatous disease: overview and hematopoietic stem cell transplantation.慢性肉芽肿病:概述与造血干细胞移植。
J Allergy Clin Immunol. 2011 Jun;127(6):1319-26; quiz 1327-8. doi: 10.1016/j.jaci.2011.03.028. Epub 2011 Apr 17.

本文引用的文献

1
Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth.非血缘供者及人类白细胞抗原(HLA)相合的同胞造血干细胞移植可治愈慢性肉芽肿病,长期预后及生长情况良好。
Br J Haematol. 2009 Apr;145(1):73-83. doi: 10.1111/j.1365-2141.2009.07614.x. Epub 2009 Feb 16.
2
Hematopoietic stem cell transplantation from matched unrelated donors in chronic granulomatous disease.慢性肉芽肿病中来自匹配无关供者的造血干细胞移植
Immunol Res. 2009;44(1-3):35-41. doi: 10.1007/s12026-008-8068-3.
3
Advances in treatment for chronic granulomatous disease.
慢性肉芽肿病的治疗进展
Immunol Res. 2009;43(1-3):77-84. doi: 10.1007/s12026-008-8051-z.
4
Hematopoietic cell transplantation for correction of primary immunodeficiencies.用于纠正原发性免疫缺陷的造血细胞移植。
Bone Marrow Transplant. 2008 Aug;42 Suppl 1:S49-S52. doi: 10.1038/bmt.2008.121.
5
Successful unrelated cord blood transplantation for chronic granulomatous disease: a case report and review of the literature.成功的非亲缘脐血移植治疗慢性肉芽肿病:一例报告并文献复习
Pediatr Transplant. 2009 May;13(3):384-9. doi: 10.1111/j.1399-3046.2008.00996.x. Epub 2008 Jul 9.
6
Modern management of chronic granulomatous disease.慢性肉芽肿病的现代管理
Br J Haematol. 2008 Feb;140(3):255-66. doi: 10.1111/j.1365-2141.2007.06880.x.
7
Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation.第二次非亲属供者脐带血移植后慢性肉芽肿病的纠正
Pediatr Blood Cancer. 2007 Dec;49(7):982-4. doi: 10.1002/pbc.21365.
8
Mutations of chronic granulomatous disease in Turkish families.土耳其家族中慢性肉芽肿病的突变
Eur J Clin Invest. 2007 Jul;37(7):589-95. doi: 10.1111/j.1365-2362.2007.01828.x.
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Transplantation of unrelated donor umbilical cord blood utilizing double-unit grafts for five teenagers with transfusion-dependent thalassemia.利用双单位移植物对五名输血依赖型地中海贫血青少年进行无关供者脐带血移植。
Bone Marrow Transplant. 2007 Aug;40(4):307-11. doi: 10.1038/sj.bmt.1705737. Epub 2007 Jun 18.
10
Treatment of McLeod phenotype chronic granulomatous disease with reduced-intensity conditioning and unrelated-donor umbilical cord blood transplantation.采用减低剂量预处理及非亲缘供者脐带血移植治疗麦克劳德表型慢性肉芽肿病。
Int J Hematol. 2007 Jan;85(1):70-2. doi: 10.1532/IJH9706129.