Purchase this article with an account.
Martina Romagnoli, Chiara La Morgia, Michele Carbonelli, Lidia Di Vito, Piero Barboni, Valerio Carelli; Idebenone treatment increases chances of stabilization-recovery of visual acuity in patients affected by dominant optic atrophy (DOA). Invest. Ophthalmol. Vis. Sci. 2019;60(9):2271.
Download citation file:
© ARVO (1962-2015); The Authors (2016-present)
Our previous study on idebenone therapy in 7 patients with OPA1-mutant DOA (OPA1-DOA) suggested some degree of visual recovery in 5 of them (Barboni et al., 2013). In this retrospective study we tested the effect of off-label idebenone administration on visual acuity in 50 OPA1-DOA patients compared to 37 untreated OPA1-DOA subjects.
We included OPA1-DOA patients with a genetic confirmation with baseline best corrected visual acuity (VA) greater than/equal to counting fingers and treatment duration of at least 7 months. We evaluated baseline VA data of the best and worst eyes. Sex and OPA1 mutation (haploinsufficiency versus missense mutation) frequencies were analysed by χ2 test. Observation time, age at baseline and VA were compared by Wilcoxon or Mann-Whitney U-tests. Idebenone efficacy was evaluated by multivariate logistic regression, defining the VA outcome as follows: VA stabilization-recovery for a VA change of 0.1 logMAR, positively or negatively, or a VA change<-0.1 logMAR; VA worsening for a VA change>+0.1 logMAR. Statistical significance was set at 0.05.
Clinical and demographic characteristics were comparable between treated and untreated patients: sex (p= 0.70), OPA1 mutation (p=0.47), observation time (p=0.15), age (p=0.85) and baseline VA (Best eye, p=0.26; Worst eye, p=0.50). Only the treated patients showed significant differences between last follow-up and baseline VA values (best eye, p=0.03; worst eye, p=0.03). Evaluating VA for the best eyes, the percentage of treated stable-responders (92%, n=46;) resulted significantly greater that the untreated one (75,7%, n=28) and idebenone was significantly associated with VA stabilization-recovery (p=0.03). Furthermore, idebenone treatment resulted significantly associated with visual stabilization-recovery also after controlling for confounding variables (adjusted odds ratio=4,37; 95% CI=1,01-18,96; p=0,049).
The chance of stabilization-recovery of vision in OPA1-DOA patients treated with idebenone was four times higher than untreated patients. These findings, if confirmed by properly-designed prospective studies, suggests that idebenone therapy may be an effective treatment in OPA1-DOA patients.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
This PDF is available to Subscribers Only