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白细胞黏附缺陷症 II 型:长期随访及文献复习。

Leukocyte adhesion deficiency type II: long-term follow-up and review of the literature.

机构信息

Kipper Institute of Allergy and Immunology, Schneider Children's Medical Center of Israel, Petah Tiqva, Israel.

出版信息

J Clin Immunol. 2010 Mar;30(2):308-13. doi: 10.1007/s10875-009-9354-0. Epub 2010 Jan 23.

Abstract

INTRODUCTION

Leukocyte adhesion deficiency (LAD) is a group of rare inherited disorders characterized by immune deficiency and peripheral neutrophilia. There are only seven reported cases of LAD type II worldwide, and no long-term follow-up data.

CASE REPORT

We reviewed the medical file of a 20-year-old man with LAD II. Clinical characteristics included short stature, severe mental retardation, and autistic features. He had had no severe infections since infancy, and his current immunological status was stable. The last laboratory work-up revealed mild leukocytosis and neutrophilia. Genetic analysis of the Golgi GDP-fucose transporter (GFTP) sequence yielded a point mutation resulting in Y337C amino acid transition in the tenth transmembrane domain.

CONCLUSION

In conclusion, in LAD II, the main clinical countenance shifts from frequent infections due to immunodeficiency in the early years to the metabolic consequences of the defect in fucose metabolism, i.e., retarded growth and mental retardation, in the later years. A novel mutation in the GFTP loci associated with LAD II is described.

摘要

简介

白细胞黏附缺陷症(LAD)是一组罕见的遗传性疾病,其特征为免疫缺陷和外周性中性粒细胞增多。全世界仅有 7 例 LAD II 型的报告病例,且无长期随访数据。

病例报告

我们回顾了一位 20 岁男性 LAD II 患者的病历。临床特征包括身材矮小、严重智力障碍和自闭症特征。他自婴儿期以来就没有严重感染,目前免疫状况稳定。最近的实验室检查结果显示轻度白细胞增多和中性粒细胞增多。高尔基体 GDP-岩藻糖转运蛋白(GFTP)序列的基因突变分析导致第十个跨膜域中 Y337C 氨基酸转换。

结论

总之,在 LAD II 中,主要的临床特征从早年因免疫缺陷而频繁感染转变为岩藻糖代谢缺陷的代谢后果,即生长和智力发育迟缓。描述了与 LAD II 相关的 GFTP 基因座的新突变。

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