Skip to main content

Conference Program

Subpage Hero

Loading

Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: a systematic review and meta-analysis

Clinical Genetics and Therapeutics
  • Primary Categories:
    • Clinical Genetics
  • Secondary Categories:
    • Clinical Genetics
Introduction:
Leber hereditary optic neuropathy (LHON) is a mitochondrial disorder characterized by the rapid loss of vision due to the degeneration of retinal ganglion cells. Current therapeutic options are limited. However, idebenone, a synthetic analog of coenzyme Q10, has been shown to enhance mitochondrial function as well as great  neuroprotective properties .This systematic review and meta-analysis aims to evaluate the efficacy of idebenone in improving visual acuity in patients with LHON. 

Methods:
We performed a systematic review on PubMed,  Cochrane and Embase databases on July 7, 2024, for studies analyzing idebenone on patients with LHON. Analyses were  conducted using random-effects models..  All statistical analyses were performed in R software version 4.3.2, using the ‘meta’ and ‘metafor’ packages.

Results:
A total of 5 studies (non-randomized and randomized clinical trials and retrospective cohorts), 375 patients  were included.  The effect sizes (TE) for each study ranged from -0.12 to -0.60, indicating that the intervention generally favored the experimental group over the control. The random-effects model produced an overall mean LogMAR difference of -0.32 (95% CI: -0.50 to -0.15), favoring the experimental group (p < 0.01). The pooled effect for the common effect model was -0.17 (95% CI: -0.17, -0.17), but with 98% heterogeneity, this model was deemed less appropriate than the random-effects model.

Conclusion:
This systematic review and meta-analysis provide evidence supporting the therapeutic benefit of idebenone in improving visual acuity in patients with Leber hereditary optic neuropathy. The significant mean LogMAR difference of -0.32 suggests that idebenone is associated with better visual outcomes compared to control treatments. Given the high heterogeneity observed among studies, further research is warranted to identify factors influencing treatment efficacy and to optimize patient selection for idebenone therapy. These findings highlight idebenone as a promising therapeutic option in the management of LHON, offering hope for improved vision in affected individuals.

Agenda

Sponsors