Van Acker K J, Eyskens F J, Espeel M F, Wanders R J, Dekker C, Kerckaert I O, Roels F
Department of Pediatrics, University Hospital, Antwerp Belgium.
Kidney Int. 1996 Nov;50(5):1747-52. doi: 10.1038/ki.1996.494.
Considering the clinical heterogeneity of primary hyperoxaluria type I (PH1) and the fact that in many instances this diagnosis was made without enzymatic and immunohistochemical investigation, other disturbances of oxalate metabolism than those presently known can be expected in PH1. Using a gaschromatographic/mass spectrometric method that allows quantification of these acids, hyperoxaluria and hyperglycoluria was found repeatedly in two unrelated patients. The hyperoxaluria was unresponsive to pyridoxine. There was no nephrocalcinosis or urolithiasis. In the liver biopsy normal AGT activity and normal localization of this enzyme in the peroxisome was found. In one patient abnormal Km and maximal activity and mozaicism of AGT were excluded. Hyperoxaluria and hyperglycoluria were also found in other family members, suggesting autosomal dominant transmission. Although the underlying defect leading to hyperoxaluria and hyperglycoluria could not be identified in these patients, it is probable that they represent a separate type of primary hyperoxaluria.
考虑到I型原发性高草酸尿症(PH1)的临床异质性,以及在许多情况下该诊断是在未进行酶学和免疫组化研究的情况下做出的,预计PH1中存在目前已知以外的其他草酸代谢紊乱。使用一种能够对这些酸进行定量的气相色谱/质谱法,在两名无亲缘关系的患者中反复发现高草酸尿症和高乙醇酸尿症。高草酸尿症对吡哆醇无反应。没有肾钙质沉着症或尿路结石。在肝活检中,发现丙氨酸 - 乙醛酸转氨酶(AGT)活性正常且该酶在过氧化物酶体中的定位正常。在一名患者中,排除了AGT的异常米氏常数、最大活性和镶嵌现象。在其他家庭成员中也发现了高草酸尿症和高乙醇酸尿症,提示常染色体显性遗传。尽管在这些患者中未能确定导致高草酸尿症和高乙醇酸尿症的潜在缺陷,但他们很可能代表一种单独类型的原发性高草酸尿症。