Pignata C, Fiore M, Guzzetta V, Castaldo A, Sebastio G, Porta F, Guarino A
Department of Pediatrics, Federico II University, Naples, Italy.
Am J Med Genet. 1996 Oct 16;65(2):167-70. doi: 10.1002/(SICI)1096-8628(19961016)65:2<167::AID-AJMG17>3.0.CO;2-O.
We report on two sisters affected by congenital alopecia, nail dystrophy, and a severe T-cell immunodeficiency, presumably inherited as an autosomal-recessive disorder. The T-cell defect was characterized by severe functional impairment, as shown by the lack of proliferative response and upregulation of activation markers following mitogen stimulation. The functional abnormality occurred in spite of the presence of phenotypically mature of the defect. This is the first observation reported on an ectodermal disorder, characterized by alopecia and nail dystrophy, observed at birth, in association with a primary immunodeficiency. The hypothesis that these two events may be casually related is discussed.
我们报告了两姐妹,她们患有先天性脱发、指甲营养不良以及严重的T细胞免疫缺陷,推测为常染色体隐性遗传疾病。T细胞缺陷的特征是严重的功能障碍,丝裂原刺激后增殖反应缺乏以及激活标志物上调即表明了这一点。尽管存在表型成熟的细胞,但仍出现了功能异常。这是首次报道一种先天性外胚层疾病,其特征为出生时即出现脱发和指甲营养不良,并伴有原发性免疫缺陷。本文还讨论了这两种情况可能存在因果关系的假说。