July 2019
Volume 60, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2019
Idebenone treatment increases chances of stabilization-recovery of visual acuity in patients affected by dominant optic atrophy (DOA)
Author Affiliations & Notes
  • Martina Romagnoli
    IRCCS Institute of Neurological Sciences of Bologna, Bellaria Hospital, Bologna, Italy
  • Chiara La Morgia
    IRCCS Institute of Neurological Sciences of Bologna, Bellaria Hospital, Bologna, Italy
    Unit of Neurology, Department of Biomedical and NeuroMotor Sciences (DIBINEM), University of Bologna, Bologna, Italy
  • Michele Carbonelli
    IRCCS Institute of Neurological Sciences of Bologna, Bellaria Hospital, Bologna, Italy
  • Lidia Di Vito
    IRCCS Institute of Neurological Sciences of Bologna, Bellaria Hospital, Bologna, Italy
  • Piero Barboni
    Studio Oculistico d'Azeglio, Bologna, Italy
    IRCCS Scientific Institute San Raffaele, Milan, Italy
  • Valerio Carelli
    IRCCS Institute of Neurological Sciences of Bologna, Bellaria Hospital, Bologna, Italy
    Unit of Neurology, Department of Biomedical and NeuroMotor Sciences (DIBINEM), University of Bologna, Bologna, Italy
  • Footnotes
    Commercial Relationships   Martina Romagnoli, Santhera Pharmaceuticals (R); Chiara La Morgia, Santhera Pharmaceuticals (R); Michele Carbonelli, Santhera Pharmaceuticals (R); Lidia Di Vito, None; Piero Barboni, Omikron Pharmaceuticals (R), Santhera Pharmaceuticals (R); Valerio Carelli, Santhera Pharmaceuticals (R), Stealth Pharmaceuticals (F)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science July 2019, Vol.60, 2271. doi:
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      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.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : 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.

Methods : 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.

Results : 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).

Conclusions : 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.

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