Mory Patricia B, Crispim Felipe, Kasamatsu Teresa, Gabbay Monica A L, Dib Sergio A, Moisés Regina S
Escola Paulista de Medicina, Universidade Federal de São Paulo, SP, Brasil.
Arq Bras Endocrinol Metabol. 2008 Nov;52(8):1252-6. doi: 10.1590/s0004-27302008000800008.
Lipodystrophies are a group of heterogeneous disorders characterized by the loss of adipose tissue and metabolic complications. The main familial forms of lipodystrophy are Congenital Generalized Lipodystrophy and Familial Partial Lipodystrophy (FPLD). FPLD may result from mutations in the LMNA gene. Besides FPLD, mutations in LMNA have been shown to be responsible for other inherited diseases called laminopathies. Here we describe the case of a 15-year-old girl who was referred to our service due to diabetes mellitus and severe hypertriglyceridemia. Physical examination revealed generalized loss of subcutaneous fat, confirmed by DEXA (total body fat 8.6%). As the patient presented with pubertal-onset of generalized lipodystrophy and insulin resistance, molecular analysis of the LMNA gene was performed. We identified a heterozygous substitution in exon 1 (c.29C>T) predicting a p.T10I mutation. In summary, we describe an atypical phenotype of lipodistrophy associated with a de novo appearance of the p.T10I mutation in LMNA gene.
脂肪营养不良是一组异质性疾病,其特征为脂肪组织丢失和代谢并发症。脂肪营养不良的主要家族性形式为先天性全身性脂肪营养不良和家族性部分性脂肪营养不良(FPLD)。FPLD可能由LMNA基因突变引起。除了FPLD,已证明LMNA基因突变还可导致其他遗传性疾病,即核纤层蛋白病。在此,我们描述了一名15岁女孩的病例,该女孩因糖尿病和严重高甘油三酯血症转诊至我们科室。体格检查发现皮下脂肪普遍缺失,双能X线吸收法(DEXA)证实(全身脂肪含量为8.6%)。由于该患者表现为青春期发病的全身性脂肪营养不良和胰岛素抵抗,因此对LMNA基因进行了分子分析。我们在第1外显子中鉴定出一个杂合性替代(c.29C>T),预测为p.T10I突变。总之,我们描述了一种与LMNA基因中p.T10I突变的新发出现相关的非典型脂肪营养不良表型。