• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Journal of Inborn Errors of Metabolism & Screening >Use of Idebenone for the Treatment of Leber?￠????s Hereditary Optic Neuropathy Review of the Evidence
【24h】

Use of Idebenone for the Treatment of Leber?￠????s Hereditary Optic Neuropathy Review of the Evidence

机译：艾地苯醌治疗Leber遗传性视神经病变的证据

获取原文
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Leber?￠????s hereditary optic neuropathy (LHON) is one of the most frequent mitochondrial disorders. It is caused by mutations in genes of the mitochondrial DNA coding for subunits of the respiratory chain and leads to severe bilateral vision loss, from which spontaneous recovery is infrequent. Retinal ganglion cells show a selective vulnerability to mitochondrial dysfunction in LHON. Idebenone is the first medication approved for LHON. It is a short-chain benzoquinone, which is an analogue of coenzyme Q10, but with distinct properties and mechanisms of action. Idebenone is a potent antioxidant and inhibitor of lipid peroxidation. Importantly, it facilitates electron flux directly to complex III, bypassing the dysfunctional complex I of the mitochondrial respiratory chain, thereby increasing adenosine triphosphate (ATP) production. In the Rescue of Hereditary Optic Disease Outpatient Study (RHODOS) randomized placebo-controlled clinical trial, 85 patients with LHON were enrolled, in the first 5 years after symptom onset, and randomized to either idebenone 900 mg/d for 6 months or placebo. Idebenone was well tolerated, and although the prespecified primary end point (best recovery in visual acuity [VA]) did not reach statistical significance, all secondary end points (change in best VA, change of VA of best eye at baseline, and change of VA in all eyes) showed a trend toward visual recovery in favor of idebenone. An increasing body of evidence shows that idebenone is effective and safe for the treatment of patients with LHON, including a large retrospective open-label study, several case reports and case series, an expanded access program, and ongoing post-authorization clinical studies. Here, we review the literature on idebenone for the treatment of patients with LHON.
机译：莱伯（Leber）遗传性视神经病变（LHON）是最常见的线粒体疾病之一。它是由编码呼吸链亚基的线粒体DNA基因突变引起的，并导致严重的双侧视力丧失，自发恢复很少发生。视网膜神经节细胞对LHON中的线粒体功能障碍表现出选择性的脆弱性。艾地苯醌是首个获准用于LHON的药物。它是短链苯醌，是辅酶Q10的类似物，但具有不同的性质和作用机理。艾地苯醌是有效的抗氧化剂和脂质过氧化抑制剂。重要的是，它可以促进电子直接流向复合物III，从而绕过线粒体呼吸链功能障碍的复合物I，从而增加了三磷酸腺苷（ATP）的产生。在《遗传性视神经疾病抢救门诊研究》（RHODOS）随机安慰剂对照临床试验中，在症状发作后的头5年入选了85例LHON患者，随机分为艾地苯醌900 mg / d，6个月或安慰剂。艾地苯醌耐受性良好，尽管预先设定的主要终点（视力最佳恢复[VA]）未达到统计学显着性，但所有次要终点（最佳VA的变化，基线时最佳眼的VA的变化以及在所有眼睛中的VA）显示出视觉恢复的趋势，倾向于艾地苯醌。越来越多的证据表明，艾地苯醌对LHON患者的治疗是有效和安全的，包括一项大型回顾性开放标签研究，数个病例报告和病例系列，扩大的获取计划以及正在进行的授权后临床研究。在这里，我们回顾有关艾地苯醌治疗LHON患者的文献。

著录项

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