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多种硫酸酯酶缺乏症的早期表现

Early manifestations of multiple sulfatase deficiency.

作者信息

Burk R D, Valle D, Thomas G H, Miller C, Moser A, Moser H, Rosenbaum K N

出版信息

J Pediatr. 1984 Apr;104(4):574-8. doi: 10.1016/s0022-3476(84)80550-8.

Abstract

We describe two boys, presenting by 1 year of age, with developmental delay from birth, mildly coarse facial features, and hepatomegaly. These clinical features were most suggestive of a mucopolysaccharidosis, particularly MPS II. Biochemical studies, including sulfate incorporation in fibroblasts and lysosomal enzyme analyses in fibroblasts, leukocytes, and serum, showed abnormalities in both sulfatide and mucopolysaccharide metabolism and led to the diagnosis of multiple sulfatase deficiency. With time, both patients developed an ichthyotic rash and profound intellectual deterioration. We conclude that findings in the first year of life in some patients with MSD may closely resemble those in patients with a MPS disorder rather than the late infantile form of metachromatic leukodystrophy, as is classically described. Thus, MSD should be considered in the young patient suspected of having a MPS disorder.

摘要

我们描述了两名1岁时就诊的男孩,他们自出生起就有发育迟缓、面部特征轻度粗糙及肝肿大。这些临床特征最提示黏多糖贮积症,尤其是II型黏多糖贮积症。生化研究,包括成纤维细胞中硫酸盐掺入及成纤维细胞、白细胞和血清中的溶酶体酶分析,显示硫脂和黏多糖代谢均异常,从而诊断为多种硫酸酯酶缺乏症。随着时间推移,两名患者均出现鱼鳞病样皮疹和严重的智力衰退。我们得出结论,一些多种硫酸酯酶缺乏症患者在生命第一年的表现可能更类似于黏多糖贮积症患者,而非经典描述的晚发性婴儿型异染性脑白质营养不良。因此,对于疑似患有黏多糖贮积症的年轻患者应考虑多种硫酸酯酶缺乏症。

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