Idebenone as a treatment for Leber hereditary optic neuropathy: A plain language summary of the LEROS study publication

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Plain Language SummaryWhat is this summary about?This is a summary of the LEROS study (Open-Label Study to assess the Efficacy and Safety of Raxone® [idebenone] in LHON Patients). The LEROS study investigated the effects of a medication called idebenone on vision in patients with Leber hereditary optic neuropathy (LHON), as well as its safety. LHON is a rare genetic disease caused by specific changes in the DNA known as mutations. The mutations can cause small structures in the cells, called mitochondria, to stop working properly, leading to vision loss.Idebenone is a medication that helps restore the function of mitochondria. In the LEROS study, researchers treated LHON patients with idebenone and then compared them to a group of untreated patients. The researchers were interested in any changes in vision (particularly improvement and/or slowing of vision loss) experienced by patients treated with idebenone, compared to the untreated group.What were the results?The researchers found that at 12 months, more eyes of idebenone-treated patients (42%) experienced a visual benefit when compared to eyes of untreated patients (21%). This visual benefit was also observed after 24 months of idebenone treatment. The researchers looked at patients who were in the early phase (up to 1 year after the start of symptoms) and late phase (1 to 5 years after the start of symptoms) of the disease. They found that both groups experienced a visual benefit from idebenone treatment.The researchers additionally found that the level of benefit depended on the specific mutation causing LHON; for patients with m.11778G>A mutations, there was a clear visual benefit with idebenone treatment. However, patients with m.3460G>A mutations did not experience a benefit. The number of patients with m.3460G>A mutations was small.The researchers also collected safety data in the LEROS study by recording any negative medical occurrences that were reported during the study, whether or not they were related to treatment with idebenone. These are known as adverse events. In the LEROS study, 11.3% of the adverse events were considered related to idebenone treatment. These safety data are similar to what healthcare providers already know from treating their patients with idebenone.What do the results mean?The results from the LEROS study showed that when compared to untreated LHON patients, idebenone treatment can improve or reduce further worsening of vision in both the early and late phases of LHON. However, the treatment seems to be more effective for certain mutations and disease stages than others. This means that it is important for patients to discuss with their healthcare provider about whether idebenone is the right treatment for them.This is an abstract of the Plain Language Summary of Publication article.View the full Plain Language Summary PDF of this article to read the full-text

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