Liver Transplantation for Hereditary Transthyretin Amyloidosis: After 20 Years Still the Best Therapeutic Alternative?
Liver Transplantation for Hereditary Transthyretin Amyloidosis: After 20 Years Still the Best Therapeutic Alternative?
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DOI：
10.1097/tp.0000000000000574
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发表时间：
2015-09-01
期刊：
影响因子：
6.2
通讯作者：
Suhr, Ole
中科院分区：
文献类型：
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作者：
Ericzon, Bo-Goran;Wilczek, Henryk E.;Suhr, Ole
背景。直到最近，肝移植（Ltx）是遗传性甲状腺转蛋白（TTR）淀粉样变性的唯一可用治疗方法；然而，今天有几种药物疗法正在进行测试。在此，我们从可获得的最大的移植遗传性TTR患者数据库中获取生存数据，作为比较的基础。方法。对家族性淀粉样变性多神经病变世界移植登记进行了20年的回顾性分析，对肝移植进行了评估。结果。从1990年4月到2010年12月，收集了77个肝移植中心的数据。该登记处有1940名患者，其中1379人还活着。88例Ltx合并心脏和/或肾脏移植。总体而言，Ltx术后20年生存率为55.3%。多变量分析显示，体重指数改变、疾病早期发病(
Background. Until recently, liver transplantation (Ltx) was the only available treatment for hereditary transthyretin (TTR) amyloidosis; today, however, several pharmacotherapies are tested. Herein, we present survival data from the largest available database on transplanted hereditary TTR patients to serve as a base for comparison. Methods. Liver transplantation was evaluated in a 20-year retrospective analysis of the Familial Amyloidosis Polyneuropathy World Transplant Registry. Results. From April 1990 until December 2010, data were accumulated from 77 liver transplant centers. The Registry contains 1940 patients, and 1379 are alive. Eighty-eight Ltx were performed in combination with a heart and/or kidney transplantation. Overall, 20-year survival after Ltx was 55.3%. Multivariate analysis revealed modified body mass index, early onset of disease (