ArticleEye (London, England)2025
Outcomes of idebenone therapy for Leber hereditary optic neuropathy in a cohort of patients from Wales.
Article in Eye (London, England), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Archetypal Visual Field Analysis of Patients With Chronic Leber Hereditary Optic Neuropathy in Relation to Visual Recovery.Investigative ophthalmology & visual science · 2026Article
- Effects of Pre- and Post-treatment of Idebenone on the Side Effects of the Chemotherapy Model with Cyclophosphamide in the Rat Ovary.Reproductive sciences (Thousand Oaks, Calif.) · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundLeber hereditary optic neuropathy (LHON) poses a significant burden to patients, with the majority not showing significant spontaneous improvement in their vision. The recent validation of idebenone as a therapy provides some avenue for benefit for patients with LHON in Wales, where it has been approved for use within the NHS.
methodsFrom March 2021, all patients being seen in a tertiary referral clinic and diagnosed with LHON by targeted genetic testing were offered treatment with idebenone and commenced on idebenone as part of standard care. For such patients, their clinical records were used to collect demographic and outcome data. Visual acuity was analysed for clinically relevant recovery (CRR), defined as improvement from 'off-chart' to 'on-chart' or 'on-chart' improvement of at least 10 letters.
resultsA total of 12 (67% male) individuals were treated with idebenone 300 mg TDS for LHON for a mean period of 30.2 (±9.9) months. Mean visual acuity at initiation of therapy was 2.22 (±0.32) LogMAR, improving to a peak of 1.12 (±0.77) LogMAR at 27 months. This time point was coincident with the maximum CRR achieved, with 86% demonstrating CRR. At 24 months, CRR was significantly higher when compared to a natural history cohort.
conclusionThe present cohort demonstrates evidence of CRR in a high proportion of patients reaching 27 months of treatment. Further follow-up and a larger cohort of patients will provide further insight into the real-world efficacy of idebenone in LHON.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.