慢性肉芽肿病的骨髓移植：长期随访及文献综述

Bone marrow transplantation for chronic granulomatous disease: long-term follow-up and review of literature.

作者信息

Leung T, Chik K, Li C, Shing M, Yuen P

机构信息

Division of Haematology and Oncology, Department of Paediatrics, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong.

出版信息

Bone Marrow Transplant. 1999 Sep;24(5):567-70. doi: 10.1038/sj.bmt.1701932.

DOI:10.1038/sj.bmt.1701932

PMID:10482944

Abstract

Chronic granulomatous disease (CGD) is a heterogeneous group of disorders with defective respiratory burst activity in phagocytes which results in recurrent pyogenic infections. We report an 8-year-old boy with X-linked CGD who received an HLA-identical BMT from his sister. The nitroblue tetrazolium test returned to normal 3 months post transplant. Neutrophil engraftment has been stable for 7 years post BMT. Our patient was the eighth case of CGD successfully treated by BMT. Conditioning regimens in these patients have consisted mainly of BU and CY. We suggest that BMT is a safe and effective method of cure for patients with CGD. BMT should be considered for patients with HLA-identical siblings.

摘要

慢性肉芽肿病（CGD）是一组异质性疾病，其吞噬细胞的呼吸爆发活性存在缺陷，导致反复发生化脓性感染。我们报告一名患有X连锁CGD的8岁男孩，他接受了来自其姐姐的 HLA 相同的骨髓移植（BMT）。移植后3个月，硝基蓝四氮唑试验恢复正常。骨髓移植后7年中性粒细胞植入情况一直稳定。我们的患者是第8例通过骨髓移植成功治疗的CGD病例。这些患者的预处理方案主要由白消安（BU）和环磷酰胺（CY）组成。我们认为骨髓移植是治疗CGD患者的一种安全有效的方法。对于有 HLA 相同同胞的患者应考虑进行骨髓移植。