Dobrescu O, Larbrisseau A, Dubé L J, Weber M L
Can Med Assoc J. 1980 Sep 20;123(6):490-7.
Trichopoliodystrophy (also known as Menkes' kinky or steely hair disease), a recessive sex-linked syndrome, is characterized by severely retarded mental and physical development, convulsions, a particular phenotype and abnormalities of the hair, bones and arteries. Very low levels of copper and ceruloplasmin in the serum confirm the diagnosis. This rare disorder is caused by an inborn error of copper metabolism whose nature is not yet clear. Recent hypotheses favour either an abnormality in the transport of copper across the cell membrane or increased affinity for copper of the intracellular binding protein. Because the metabolic abnormality is expressed autonomously and irregularly in various tissues, the distribution of copper within the body is disordered. Up to now none of the many forms of copper therapy has succeeded in modifying the fatal course of the disease in humans. This article presents a new case, the first in Canada, and a review of the other 69 cases described in the literature. The new case illustrates, in addition to the classic picture, less well known features, such as diverticula of the bladder mucosa and serosa, as well as cortical atrophy and malformed cerebral vessels demonstrated by computer-assisted tomography.
毛发硫营养不良症(也称为门克斯扭结或钢发疾病)是一种隐性性连锁综合征,其特征为严重的智力和身体发育迟缓、惊厥、特定的表型以及毛发、骨骼和动脉异常。血清中铜和铜蓝蛋白水平极低可确诊该病。这种罕见疾病是由铜代谢的先天性缺陷引起的,其本质尚不清楚。最近的假说认为,要么是铜跨细胞膜转运异常,要么是细胞内结合蛋白对铜的亲和力增加。由于代谢异常在各种组织中自主且不规则地表现出来,体内铜的分布紊乱。到目前为止,多种形式的铜疗法都未能成功改变人类这种疾病的致命进程。本文介绍了加拿大首例新病例,并对文献中描述的其他69例病例进行了综述。除了典型症状外,新病例还展现了一些不太为人所知的特征,如膀胱黏膜和浆膜憩室,以及计算机断层扫描显示的皮质萎缩和脑血畸形。