Therapeutic Effects of Idebenone on Leber Hereditary Optic Neuropathy
Therapeutic Effects of Idebenone on Leber Hereditary Optic Neuropathy
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艾地苯醌对莱伯遗传性视神经病的治疗作用
DOI：
10.1080/02713683.2020.1736307
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发表时间：
2020-03-08
影响因子：
2
通讯作者：
Yang, Hui
中科院分区：
文献类型：
--
作者：
Zhao, Xiujuan;Zhang, Yuxin;Yang, Hui
摘要目的：采用光学相干断层扫描（OCT）测量视力（VA）、视野（VF）缺损、视觉诱发电位（VEP）和视网膜神经纤维层（RNFL）厚度，以表征艾地苯醌对Leber遗传性视神经病变（LHON）的潜在治疗作用。研究方法：这是一项回顾性病例对照研究，研究艾地苯醌（900 mg/d）对30例因m.3460 G > A、m.11778 G > A和m.14484 T > C突变导致的LHON患者的影响。主要终点是3个月和6个月后VA的恢复。主要次要终点是VF、VEP和RNFL厚度的变化。另一个次要终点是6个月后视力变化与各组基线时VF、VEP和RNFL厚度的相关性。结果：艾地苯醌是安全的，耐受性良好。主要终点达到统计学意义。艾地苯醌组最佳眼和最差眼的VA均有所改善。VF平均缺损减小，VEP振幅增加。各组潜伏期和RNFL厚度无显著差异。治疗以及基线时的VA和振幅对6个月时VA的改善具有显著影响。结论：这项LHON病例对照研究提供的证据表明，艾地苯醌治疗可能对LHON病例有益，并且控制结局的影响因素是基线时VEP的VA和振幅。
ABSTRACT Purpose: To characterize the potential therapeutic effects of idebenone on Leber hereditary optic neuropathy (LHON) in terms of visual acuity (VA), visual field (VF) defects, visual evoked potential (VEP) and retinal nerve fibre layer (RNFL) thickness using optical coherence tomography (OCT) measurements. Methods: This was a retrospective case-controlled study of the effect of idebenone (900 mg/d) on 30 patients with LHON due to m.3460 G > A, m.11778 G > A and m.14484 T > C mutations. The primary end-point was the recovery in VA after 3 and 6 mon. The main secondary end-point was the change in VF, VEP and RNFL thickness. The other secondary end-point was the correlation between visual changes after 6 mon and the VF, VEP and RNFL thickness at baseline of the groups. Results: Idebenone was shown to be safe and well tolerated. The primary end-point reached statistical significance. The VA in the idebenone group improved in both the best eye and worst eye. The mean defect of VF decreased and amplitude of VEP increased. There was no significant difference in latency and RNFL thickness between the groups. The treatment, together with the VA and amplitude at baseline, had a significant effect on the improvement in VA at 6 mon. Conclusion: This case-controlled study of LHON provides evidence that idebenone treatment may be beneficial in cases of LHON and that the influential factors governing outcomes are the VA and amplitude of the VEP at baseline.