A 20-year experience of combined liver/kidney transplantation for primary hyperoxaluria (PH1): The European PH1 Transplant Registry experience 1984-2004
A 20-year experience of combined liver/kidney transplantation for primary hyperoxaluria (PH1): The European PH1 Transplant Registry experience 1984-2004
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DOI：
10.1159/000086359
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发表时间：
2005-01-01
影响因子：
4.2
通讯作者：
Jamieson, NV
中科院分区：
文献类型：
--
作者：
Jamieson, NV
原发性高草酸尿症 (PH1) 是一种由肝酶缺陷引起的疾病，可导致草酸盐结石病、梗阻性尿病和肾钙质沉着症导致肾功能衰竭。研究表明，潜在的代谢缺陷可以通过肝移植来纠正，并且在大多数情况下（已经发生肾衰竭）需要进行肾移植。本文描述了 1984 年至 2004 年 20 年间在 35 个欧洲中心对 117 名患者进行的 127 例肝移植的当前结果。出现症状的平均年龄为 5.6 +/- 7.8 岁，做出诊断的平均年龄为 8.8 +/- 9.5 岁。肝活检证实了诊断，68% 的病例存在 AGT 活性降低，74% 存在高草酸尿，37% 存在高乙醇酸尿，50% 存在高草酸血症。经过 3.2 +/- 3.2 年（范围 0 - 14.4 岁）透析期后，患者的平均年龄为 16.5 +/- 11.4 岁。患者1年、5年和10年生存率分别为86%、80%和69%，同一时间间隔的肝移植生存率分别为80%、72%和60%。已有27人死亡，并进行了10例肝脏再移植。当长期透析和系统性草酸盐中毒并发症没有发生时，患者的预后会得到改善。版权所有 (C) 2005 S. Karger AG，巴塞尔。
Primary hyperoxaluria (PH1) is a condition caused by a hepatic-based enzyme defect which can lead to renal failure due to oxalate stone disease, obstructive uropathy and nephrocalcinosis. It has been shown that the underlying metabolic defect can be corrected by liver transplantation and in most cases ( renal failure having already occurred) is accompanied by a kidney graft. This paper describes the current results of 127 liver transplants performed in 117 patients over a 20-year period from 1984 to 2004 in 35 European centres. The mean age at onset of symptoms was 5.6 +/- 7.8 years and the mean age at which a diagnosis was made was 8.8 +/- 9.5 years. The diagnosis was confirmed by liver biopsy proven decreased AGT activity in 68% of cases, hyperoxaluria in 74%, hyperglycolicaciduria in 37% and hyperoxalaemia in 50%. Patients were transplanted at a mean age of 16.5 +/- 11.4 years following a period of dialysis of 3.2 +/- 3.2 years ( range 0 - 14.4 years). 1-, 5- and 10-year patient survival values were 86, 80 and 69%, respectively, and liver graft survival rates of 80, 72 and 60% at the same time intervals. There have been 27 deaths and 10 liver retransplants have been carried out. Patient outcomes are improved when prolonged periods on dialysis and the complications of systemic oxalosis have not occurred. Copyright (C) 2005 S. Karger AG, Basel.