Effect of liver transplantation on transthyretin Tyr114Cys-related cerebral amyloid angiopathy
Effect of liver transplantation on transthyretin Tyr114Cys-related cerebral amyloid angiopathy
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DOI：
10.1212/01.wnl.0000287089.28847.b5
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发表时间：
2008-01
期刊：
影响因子：
9.9
通讯作者：
T. Yamashita;Yukio Ando;M. Ueda;Masanobu Nakamura;S. Okamoto;M. Zeledon;T. Hirahara;Toshinori Hirai;A. Ueda;Y. Misumi;K. Obayashi;H. Inomata;M. Uchino
中科院分区：
文献类型：
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作者：
T. Yamashita;Yukio Ando;M. Ueda;Masanobu Nakamura;S. Okamoto;M. Zeledon;T. Hirahara;Toshinori Hirai;A. Ueda;Y. Misumi;K. Obayashi;H. Inomata;M. Uchino
目的：患有淀粉样甲状腺素运载蛋白（ATTR）Tyr114 Cys的患者在脑血管中发生淀粉样沉积、脑出血和表现为遗传性脑淀粉样血管病（CAA）的快速进展性痴呆。然而，尚未确定CAA的治疗方法。虽然肝移植已成为TTR相关淀粉样变性的可接受治疗，但肝移植可能无法成功治疗该疾病的CNS表现。在这项研究中，我们研究了肝移植对TTR相关CAA的这些表现的影响。研究方法：我们比较了3例与ATTR Tyr114 Cys相关的CAA患者的临床病程，这些患者接受了肝移植，5例未接受肝移植的CAA患者也接受了肝移植。结果如下：与未接受移植的患者相比，接受移植的患者的死亡率、脑出血和痴呆的发生率降低。两组在意识波动和短暂性脑缺血发作的频率方面没有差异。接受移植组的颅内出血量明显少于未接受移植组。结论：肝移植对与淀粉样甲状腺素运载蛋白Tyr114Cys相关的脑淀粉样血管病的CNS表现有效。
Objective: Patients with amyloidogenic transthyretin (ATTR) Tyr114Cys develop amyloid deposits in cerebral blood vessels, cerebral hemorrhage, and rapidly progressive dementia that presents with hereditary cerebral amyloid angiopathy (CAA). However, no treatment has been identified for CAA. Although liver transplantation has become an acceptable treatment of TTR-related amyloidosis, liver transplantation may not successfully treat CNS manifestations of the disorder. In this study, we examined the effect of liver transplantation on these manifestations of TTR-related CAA. Methods: We compared clinical courses of three patients with CAA associated with ATTR Tyr114Cys who underwent liver transplantation with those of five patients with the disorder who did not undergo liver transplantation. Results: The mortality and occurrence of cerebral hemorrhage and dementia in patients having transplantations were reduced compared with those in patients not having transplantations. The two groups did not differ with regard to the frequency of episodes of fluctuating consciousness and TIAs. The group undergoing transplantations had significantly smaller volumes of intracranial hemorrhage than did the no-transplantation group. Conclusion: Liver transplantation was effective for CNS manifestations of cerebral amyloid angiopathy associated with amyloidogenic transthyretin Tyr114Cys.