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本文引用的文献

1
Congenital generalized lipodystrophies--new insights into metabolic dysfunction.先天性全身脂肪营养不良——对代谢功能障碍的新见解
Nat Rev Endocrinol. 2015 Sep;11(9):522-34. doi: 10.1038/nrendo.2015.123. Epub 2015 Aug 4.
2
[Congenital generalized lipodystrophy: report of a case].[先天性全身性脂肪营养不良:一例报告]
Zhonghua Er Ke Za Zhi. 2004 Dec;42(12):959.
3
Acquired and inherited lipodystrophies.获得性和遗传性脂肪营养不良。
N Engl J Med. 2004 Jul 1;351(1):103-4; author reply 103-4.
4
Lipodystrophies: rare disorders causing metabolic syndrome.脂肪营养不良:导致代谢综合征的罕见疾病。
Endocrinol Metab Clin North Am. 2004 Jun;33(2):305-31. doi: 10.1016/j.ecl.2004.03.003.
5
Heterozygous missense mutations in BSCL2 are associated with distal hereditary motor neuropathy and Silver syndrome.BSCL2基因的杂合错义突变与远端遗传性运动神经病和西尔弗综合征相关。
Nat Genet. 2004 Mar;36(3):271-6. doi: 10.1038/ng1313. Epub 2004 Feb 22.
6
Lipodystrophy and gigantism with associated endocrine manifestations. A new diencephalic syndrome?脂肪营养不良和巨人症伴相关内分泌表现。一种新的间脑综合征?
Acta Paediatr (Stockh). 1959 Nov;48:555-74.
7
An undiagnosed endocrinometabolic syndrome: report of 2 cases.一种未确诊的内分泌代谢综合征:2例报告。
J Clin Endocrinol Metab. 1954 Feb;14(2):193-204. doi: 10.1210/jcem-14-2-193.
8
Serum adiponectin and leptin levels in patients with lipodystrophies.脂肪营养不良患者的血清脂联素和瘦素水平
J Clin Endocrinol Metab. 2002 May;87(5):2395. doi: 10.1210/jcem.87.5.8624.
9
Leptin-replacement therapy for lipodystrophy.用于脂肪营养不良的瘦素替代疗法。
N Engl J Med. 2002 Feb 21;346(8):570-8. doi: 10.1056/NEJMoa012437.

[一名女孩出现异常面容及甘油三酯反复升高超过一年]

[Unusual facies and recurrent high triglycerides for more than one year in a girl].

作者信息

Yin Ze-Xi, He Xiang-Ling, Zou Run-Ying

机构信息

Department of Pediatrics, First Affiliated Hospital of Hunan Normal University/Hunan Provincial People's Hospital, Changsha 410005, China.

出版信息

Zhongguo Dang Dai Er Ke Za Zhi. 2018 Dec;20(12):1050-1054. doi: 10.7499/j.issn.1008-8830.2018.12.014.

DOI:10.7499/j.issn.1008-8830.2018.12.014
PMID:30572997
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7389504/
Abstract

A girl, aged 1 year and 9 months, was found to have hypertriglyceridemia in the neonatal period, with unusual facies and signs of dark skin all over the body, disappearance of subcutaneous adipose, acanthosis nigricans of the neck, excessive and thick hair, empty cheeks, muscle hypertrophy of the extremities, hepatomegaly, and neutrophil deficiency. Whole exome sequencing of monogenic disorder revealed a homozygote mutation in the BSCL2 gene, c.974 (exon 7)_c.975 (exon 7) insG. Her parents were heterozygotes for this locus. The girl was diagnosed with congenital generalized lipodystrophy (CGL), but the association between CGL and neutrophil deficiency remained unclear. Triglyceride was maintained at a normal level after the treatment with a low-fat and high-carbohydrate diet, and there were no obvious changes in signs. CGL is a rare autosomal recessive systemic disease manifested as disappearance of systemic subcutaneous adipose, muscle hypertrophy of the extremities, and metabolic disorders in the neonatal period, such as high triglycerides, hyperinsulinemia, and hyperglycemia. About 95% of CGL cases are caused by mutations in the AGPAT2 or BSCL2 gene.

摘要

一名1岁9个月大的女童在新生儿期被发现患有高甘油三酯血症,伴有特殊面容、全身皮肤黝黑、皮下脂肪消失、颈部黑棘皮病、毛发过多且浓密、脸颊凹陷、四肢肌肉肥大、肝肿大以及中性粒细胞缺乏。单基因疾病的全外显子测序显示,BSCL2基因存在纯合突变,c.974(第7外显子)_c.975(第7外显子)插入G。她的父母是该位点的杂合子。该女童被诊断为先天性全身脂肪营养不良(CGL),但CGL与中性粒细胞缺乏之间的关联仍不明确。采用低脂高碳水化合物饮食治疗后,甘油三酯维持在正常水平,体征无明显变化。CGL是一种罕见的常染色体隐性全身性疾病,表现为新生儿期全身皮下脂肪消失、四肢肌肉肥大以及代谢紊乱,如高甘油三酯血症、高胰岛素血症和高血糖症。约95%的CGL病例由AGPAT2或BSCL2基因突变引起。