Vendrell J, Ercilla G, Faundez A, Soler S, Gutierrez C, Gomis R, Vilardell E, Richart C
Department of Endocrinology and Nutrition, Hospital Clinic, Barcelona, Spain.
Diabetes Res Clin Pract. 1994 Jan;22(2-3):175-80. doi: 10.1016/0168-8227(94)90052-3.
The Wolfram syndrome (WS) is an autosomal recessive disorder beginning in childhood that consists of four clinical features: diabetes insipidus, diabetes mellitus, optic atrophy and deafness. Its pathogenesis remains unknown, although the tendency to develop this syndrome has been related to some class II antigens of the HLA system. We report six new cases in four families. A review of published data from the genetic features of this syndrome is performed, establishing the high frequency of the HLA-DR2 antigen in the WS (44.4%) compared with a control group (21.9%; relative risk, 2.8) and to patients with Type 1 insulin-dependent diabetes mellitus (Type 1 diabetes) (6.77%; relative risk, 9.7). We also comment the high frequency of the HLA-DQw1 antigen (85.5%) in this syndrome, without statistical significance. A familial segregation study of the HLA haplotypes has been carried out without finding correlation between the autosomal recessive pattern attributed to the WS, and the major histocompatibility complex. In conclusion, whereas HLA may increase susceptibility to the WS, as shown by the existence of an HLA-DR2 association, the major genetic influence on the inheritance of the WS must be at another locus.
沃夫勒姆综合征(WS)是一种始于儿童期的常染色体隐性疾病,具有四种临床特征:尿崩症、糖尿病、视神经萎缩和耳聋。尽管该综合征的发病倾向与HLA系统的某些II类抗原有关,但其发病机制仍不清楚。我们报告了四个家族中的六例新病例。对该综合征遗传特征的已发表数据进行了综述,发现与对照组(21.9%;相对风险,2.8)和1型胰岛素依赖型糖尿病患者(1型糖尿病)(6.77%;相对风险,9.7)相比,WS中HLA - DR2抗原的频率较高(44.4%)。我们还提到了该综合征中HLA - DQw1抗原的高频率(85.5%),但无统计学意义。已对HLA单倍型进行家族分离研究,未发现归因于WS的常染色体隐性模式与主要组织相容性复合体之间存在相关性。总之,虽然HLA可能会增加对WS的易感性,如HLA - DR2关联的存在所示,但对WS遗传的主要基因影响一定在另一个位点。