Yin Ze-Xi, He Xiang-Ling, Zou Run-Ying
Department of Pediatrics, First Affiliated Hospital of Hunan Normal University/Hunan Provincial People's Hospital, Changsha 410005, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2018 Dec;20(12):1050-1054. doi: 10.7499/j.issn.1008-8830.2018.12.014.
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.
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。