Beck M, Bender S W, Reiter H L, Otto W, Bässler R, Dancygier H, Gehler J
Eur J Pediatr. 1984 Dec;143(2):135-9. doi: 10.1007/BF00445802.
A newborn infant with oedema, ascites and hepatosplenomegaly is described. In ascites fluid foamy macrophages were found, in a liver biopsy cytoplasmic inclusions and membrane-bound vacuoles were seen. Furthermore the child excreted excessive amounts of sialic acid-rich oligosaccharides in the urine, and therefore a neurovisceral degenerative disorder was assumed. The diagnosis of sialidosis was confirmed by enzymatic assay in cultured fibroblasts, in which a complete deficiency of the lysosomal enzyme neuraminidase could be demonstrated. After recurrent septicaemias the child became dystrophic and died at the age of 6 months. Our case is compared with sialidosis observed by other authors, the wide phenotypic diversity within this biochemical defect is emphasised. The occurrence of hydrops fetalis in lysosomal storage diseases is discussed.
描述了一名患有水肿、腹水和肝脾肿大的新生儿。在腹水中发现了泡沫巨噬细胞,肝脏活检可见细胞质内含物和膜结合空泡。此外,该患儿尿液中排泄大量富含唾液酸的寡糖,因此推测为神经内脏退行性疾病。通过对培养的成纤维细胞进行酶分析确诊为唾液酸沉积症,其中可证明溶酶体酶神经氨酸酶完全缺乏。反复发生败血症后,患儿出现营养不良,于6个月龄时死亡。将我们的病例与其他作者观察到的唾液酸沉积症进行了比较,强调了这种生化缺陷内广泛的表型多样性。讨论了溶酶体贮积病中胎儿水肿的发生情况。