Idebenone increases chance of stabilization/recovery of visual acuity in OPA1-dominant optic atrophy

Martina Romagnoli, Chiara La Morgia, Michele Carbonelli, Lidia Di Vito, Giulia Amore, Corrado Zenesini, Maria Lucia Cascavilla, Piero Barboni, Valerio Carelli

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish
JournalAnnals of Clinical and Translational Neurology
DOIs
Publication statusE-pub ahead of print - Apr 3 2020

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