Open AccessIdebenone increases chance of stabilization/recovery of visual acuity in OPA1 ‐dominant optic atrophy
Author(s) -
Romagnoli Martina,
La Morgia Chiara,
Carbonelli Michele,
Di Vito Lidia,
Amore Giulia,
Zenesini Corrado,
Cascavilla Maria Lucia,
Barboni Piero,
Carelli Valerio
Publication year - 2020
Publication title -
annals of clinical and translational neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.824
H-Index - 42
ISSN - 2328-9503
DOI - 10.1002/acn3.51026
Subject(s) - medicine , idebenone , visual acuity , atrophy , confounding , ophthalmology
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.