Idebenone increases chance of stabilization/recovery of visual acuity in OPA1‐dominant optic atrophy. Issue 4 (3rd April 2020)

• Record Type: Journal Article
• Authors: Romagnoli, Martina; La Morgia, Chiara; Carbonelli, Michele; Di Vito, Lidia; Amore, Giulia; Zenesini, Corrado; Cascavilla, Maria Lucia; Barboni, Piero; Carelli, Valerio
• Is Part Of: Annals of clinical and translational neurology. Volume 7:Issue 4(2020)
• Page Start: 590
• Page End: 594
• Publication Date: 2020-04-03
• Subjects: Nervous system -- Diseases -- Periodicals; Neurology -- Periodicals; 616.8005
• DOI: 10.1002/acn3.51026
• Languages: English
• ISSNs: 2328-9503

Abstract

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.