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长期获得的转氨基粉症的长期结果
Yohei Misumi1, Toshiya Nomura1, Shiori Yamakawa1
1Department of Neurology, Kumamoto University, Kumamoto, Japan.
概括
获得的跨甲基氨基粉症在肝移植接受者中很常见. 虽然tafamidis有助于控制症状,但它并不能阻止所有的进展,这表明siRNA治疗可能需要长期的结果.
科学领域:
- 移植医学 移植医学
- 神经学 神经学
- 心脏病学 心脏病学
- 遗传学 是一个遗传学.
背景情况:
- 获得的转氨基粉症 (ATTR) 是多米诺肝移植 (DLT) 接受者日益关注的问题.
- 遗传变异TTR (ATTRv) 粉样化症患者的肝脏可以传播疾病.
- 对于DLT患者的长期结果和治疗疗效尚不清楚.
研究的目的:
- 评估从ATTRv粉样化症患者接受移植的DLT受体的长期结果.
- 评估疾病修饰药物的有效性,如tafamidis和siRNA疗法.
主要方法:
- 对30个DLT受益人的回顾性分析.
- 纵向随访包括临床评估,神经传导研究 (NCS) 和各种成像技术 (光学图,心声,心电图).
- 血清生物标志物分析.
主要成果:
- 在DLT后10年的整体存活率为57.7%.
- 粉样蛋白沉积发生在17名接受者身上,平均发病时间为DLT后7.7年.
- 塔法米迪斯在大多数患者中稳定了神经病变,但没有预防亚临床轴突退化;siRNA治疗在两个病例中显示出有前途.
结论:
- 获得的ATTR Amyloidosis是DLT接受者经常出现的长期并发症.
- 塔法米迪斯可以提供临床稳定性,但可能无法完全预防疾病的进展.
- 进一步研究最佳治疗策略,包括siRNA,对于管理这种情况至关重要.
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