Fierabracci Alessandra
Infectivology and Clinical Trials Area, Children's Hospital Bambino Gesù, Rome 00146, Italy.
Int J Mol Sci. 2016 Jul 12;17(7):1106. doi: 10.3390/ijms17071106.
Type 1 autoimmune polyglandular syndrome (APS1) is a rare autosomal recessive disease, caused by mutations in the autoimmune regulator gene (AIRE); the encoded Aire protein plays an important role in the establishment of the immunological tolerance acting as a transcriptional regulator of the expression of organ-specific antigens within the thymus in perinatal age. While a high prevalence for this rare syndrome is reported in Finland and Scandinavia (Norway), autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy syndrome (APECED) cohorts of patients are also detected in continental Italy and Sardinia, among Iranian Jews, as well as in other countries. The syndrome is diagnosed when patients present at least two out of the three fundamental disorders including chronic mucocutaneous candidiasis, hypoparathyroidism, and Addison's disease. Among the associated conditions insulin-dependent diabetes mellitus (Type 1 diabetes) has been rarely reported in different series of patients and occurring more frequently in Finnish APECED patients. In this review, we analyze the incidence of Type 1 diabetes as a clinical manifestation of APECED in different populations highlighting the peculiar genetic and immunological features of the disease when occurring in the context of this syndrome.
1型自身免疫性多腺体综合征(APS1)是一种罕见的常染色体隐性疾病,由自身免疫调节基因(AIRE)突变引起;编码的Aire蛋白在免疫耐受的建立中起重要作用,在围产期作为胸腺内器官特异性抗原表达的转录调节因子。虽然在芬兰和斯堪的纳维亚半岛(挪威)报道了这种罕见综合征的高患病率,但在意大利大陆和撒丁岛、伊朗犹太人以及其他国家也检测到了自身免疫性多内分泌腺病-念珠菌病-外胚层营养不良综合征(APECED)患者队列。当患者出现慢性黏膜皮肤念珠菌病、甲状旁腺功能减退和艾迪生病这三种基本疾病中的至少两种时,即可诊断该综合征。在相关病症中,胰岛素依赖型糖尿病(1型糖尿病)在不同系列的患者中很少被报道,而在芬兰APECED患者中更常见。在这篇综述中,我们分析了1型糖尿病作为APECED在不同人群中的临床表现的发生率,突出了该疾病在这种综合征背景下发生时的特殊遗传和免疫学特征。