• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Prescrire international >tafamidis (VYNDAQEL°) and transthyretin amyloid cardiomyopathy
【24h】

tafamidis (VYNDAQEL°) and transthyretin amyloid cardiomyopathy

机译：Tafamidis（Vyndaqel°）和Transthyretin淀粉片心肌病

获取原文
获取原文并翻译 | 示例
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

In a placebo-controlled trial including 441 patients with transthyretin amyloid cardiomyopathy, tafamidis reduced mortality after 30 months of treatment to 30% of patients compared to 43% in the placebo group. Tafamidis carries a risk in particular of infections and of gastrointestinal, ocular and possibly hepatic disorders. It has not been demonstrated that the 61 mg dose authorised in this setting is more beneficial than a lower dose. Transthyretin amyloidosis is a rare, fatal disease, which generally becomes apparent between 30 and 50 years of age. Patients have a life expectancy of around ten years after appearance of the first symptoms. The transthyretin protein is a stable, soluble tetramer. Transthyretin amyloidosis is due to the dissociation of the transthyretin protein, leading to amyloid deposits in a range of organs and their consequent dysfunction. This dissociation can be linked to advancing age, or it can be hereditary, associated in this case with a mutation in the transthyretin gene. The clinical signs vary depending on the organs affected. Neurological damage manifests as sensory and motor neuropathy. Cardiac involvement manifests as cardiomyopathy leading to progressive heart failure, thrombosis and cardiac conduction disorders causing syncope. Treatment of the cardiac disorders is symptomatic, often combined with anticoagulants. Heart transplantation is rarely feasible due to the involvement of other organs (1-4).
机译：在一项包括441名跨甲状腺淀粉样心肌病患者的安慰剂对照试验中，塔法米迪斯将治疗30个月后的死亡率降至30%，而安慰剂组为43%。塔法米迪斯尤其具有感染和胃肠道、眼部以及可能的肝脏疾病的风险。目前还没有证据表明，在这种情况下，61毫克的授权剂量比较低剂量更有益。转甲状腺素淀粉样变性是一种罕见的致命疾病，通常在30至50岁之间变得明显。在出现第一症状后，患者的预期寿命约为10年。转甲状腺素蛋白是一种稳定的可溶性四聚体。转甲状腺素淀粉样变性是由于转甲状腺素蛋白的解离，导致一系列器官中的淀粉样沉积，并导致其功能障碍。这种分离可能与年龄增长有关，也可能是遗传性的，在这种情况下与转甲状腺素基因突变有关。临床症状因受影响的器官而异。神经损伤表现为感觉和运动神经病变。心脏受累表现为心肌病，导致进行性心力衰竭、血栓形成和心脏传导障碍，导致晕厥。心脏疾病的治疗是有症状的，通常与抗凝剂联合使用。由于涉及其他器官，心脏移植很少可行（1-4）。

著录项

  • 来源
    《Prescrire international》 |2021年第222期|共2页
  • 作者

  • 作者单位
  • 收录信息
  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类 药学;
  • 关键词

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号