在法布里病中，同种异体肾移植未能纠正代谢缺陷。
Failure to correct the metabolic defect by renal allotransplantion in Fabry's disease.
作者信息
Spence M W, MacKinnon K E, Burgess J K, d'Entremont D M, Belitsky P, Lannon S G, MacDonald A S
出版信息
Ann Intern Med. 1976 Jan;84(1):13-6. doi: 10.7326/0003-4819-84-1-13.
Plasma neutral glycolipid levels and plasma and leukocyte alpha-galactosidase activities were measured serially before and after renal allotransplantation in two men, aged 47 and 45 years, with renal failure due to Fabry's disease. The patients were followed posttransplantation for 92 and 64 weeks, respectively. No significant elevation of plasma or leukocyte alpha-galactosidase activities above levels in untreated men with Fabry's disease or decrease in the levels of trihexosyl ceramide was observed in either patient. The results do not support the use of renal allotransplantation for enzyme replacement in Fabry's disease.
对两名分别为47岁和45岁、因法布里病导致肾衰竭的男性患者，在同种异体肾移植前后连续测定了血浆中性糖脂水平以及血浆和白细胞α - 半乳糖苷酶活性。两名患者移植后分别随访了92周和64周。在两名患者中均未观察到血浆或白细胞α - 半乳糖苷酶活性显著高于未经治疗的法布里病男性患者的水平，也未观察到三己糖神经酰胺水平降低。这些结果不支持将同种异体肾移植用于法布里病的酶替代治疗。
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