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慢性肉芽肿病的认识与治疗新进展

Recent advances in understanding and treating chronic granulomatous disease.

作者信息

Gennery Andrew

机构信息

Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Great North Childrens' Hospital, Newcastle upon Tyne, UK.

Primary Immunodeficiency Group, Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK.

出版信息

F1000Res. 2017 Aug 11;6:1427. doi: 10.12688/f1000research.11789.1. eCollection 2017.

Abstract

A number of recent advances have been made in the epidemiology and treatment of chronic granulomatous disease. Several reports from developing regions describe the presentations and progress of local populations, highlighting complications due to Bacillus Calmette-Guérin vaccination. A number of new reports describe complications of chronic granulomatous disease in adult patients, as more survivors reach adulthood. The complications experienced by X-linked carriers are particularly highlighted in three new reports, confirming that infection and inflammatory or autoimmune conditions are more common and severe than previously recognised. Finally, definitive treatment with haematopoietic stem cell transplantation and gene therapy is reviewed.

摘要

慢性肉芽肿病的流行病学和治疗方面最近取得了一些进展。来自发展中地区的几份报告描述了当地人群的临床表现和病情进展,突出了卡介苗接种引起的并发症。随着更多幸存者成年,一些新报告描述了成年慢性肉芽肿病患者的并发症。三份新报告特别强调了X连锁携带者所经历的并发症,证实感染以及炎症或自身免疫性疾病比以前认识到的更为常见和严重。最后,对造血干细胞移植和基因治疗的确定性治疗进行了综述。

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