We previously documented that idebenone treatment in OPA1‐Dominant Optic Atrophy (OPA1‐DOA) led to some degrees of visual improvement in seven patients. We here present the results of a cohort study, which investigated the effect of off‐label idebenone administration in a larger OPA1‐DOA group compared with untreated patients. Inclusion criteria were: OPA1‐DOA clinical and molecular diagnosis, baseline visual acuity (VA) greater than/equal to counting fingers and treatment duration greater than 7 months. We found a significant difference between the last visit and baseline VA in favor of stabilization/recovery in idebenone‐treated as compared to untreated patients. This effect was retained after controlling for confounders.

中文翻译：

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艾地苯醌增加了以OPA1为主的视神经萎缩的视力稳定/恢复的机会。

先前我们记录了艾地苯醌在OPA1主导性视神经萎缩症（OPA1- DOA）中的治疗可导致7例患者的视觉改善。我们在这里提供了一项队列研究的结果，该研究比较了未经治疗的患者在较大的OPA1- DOA组中使用非标记艾地苯醌的效果。纳入标准为：OPA1- DOA临床和分子诊断，基线视力（VA）大于/等于手指计数且治疗持续时间大于7个月。我们发现，在未接受艾地苯醌治疗的患者中，上次就诊与基线VA之间存在显着差异，有利于艾地苯醌治疗的稳定/恢复。控制混杂因素后，这种效果得以保留。