Factors associated with rapid improvement in visual acuity in patients with Leber's hereditary optic neuropathy after gene therapy.
Liu Hong-Li, Yuan Jia-Jia, Zhang Yong, Tian Zhen, Li Xin, Wang Dan, Du Yang-Yang, Song Lin, Li Bin
AI Summary
This study found that shorter onset-to-treatment time and better baseline vision predict rapid visual improvement in LHON patients after gene therapy, guiding patient selection.
Abstract
Purpose
To analyse the factors associated with rapid and significant improvement in visual acuity in patients with Leber's hereditary optic neuropathy (LHON) after gene therapy and explain the theory of this improvement.
Methods
We recruited 149 patients with LHON, who underwent gene therapy, and divided them into two groups according to the absence or presence of rapid and significant visual acuity improvements within 3 days of treatment. A bivariate logistic regression model was used to analyse relevant factors including age, the period between onset and treatment, baseline values of best corrected visual acuity (BCVA), visual field index (VFI) and pretreatment average retinal nerve fibre-layer thickness (GRNFL). The corresponding parameters for the injected and non-injected eyes were analysed separately.
Results
The period between onset and treatment, and pretreatment baseline BCVA was significantly associated with rapid and significant improvement in visual acuity for both, the injected and non-injected eyes. Moreover, pretreatment baseline VFI and GRNFL were not significantly associated with rapid and significant improvement in visual acuity. Age was significantly associated with rapid and significant improvement in visual acuity of the injected eyes.
Conclusion
The period between onset and treatment, and pretreatment baseline BCVA may be important predictors of rapid and significant improvement in visual acuity in patients with LHON after gene therapy.
MeSH Terms
Shields Classification
Key Concepts4
The period between onset and treatment for Leber's hereditary optic neuropathy (LHON) and pretreatment baseline best corrected visual acuity (BCVA) were significantly associated with rapid and significant improvement in visual acuity for both injected and non-injected eyes after gene therapy.
Pretreatment baseline visual field index (VFI) and average retinal nerve fibre-layer thickness (GRNFL) were not significantly associated with rapid and significant improvement in visual acuity in patients with Leber's hereditary optic neuropathy (LHON) after gene therapy.
Age was significantly associated with rapid and significant improvement in visual acuity of the injected eyes in patients with Leber's hereditary optic neuropathy (LHON) after gene therapy.
A bivariate logistic regression model was used to analyse relevant factors for rapid and significant visual acuity improvements within 3 days of treatment in 149 patients with Leber's hereditary optic neuropathy (LHON) who underwent gene therapy.
Related Articles5
Predictors of Final Visual Outcome in Patients With Leber Hereditary Optic Neuropathy Treated With Lenadogene Nolparvovec Gene Therapy.
Clinical TrialGene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results.
Clinical TrialLeber hereditary optic neuropathy associated with use of ephedra alkaloids.
Case ReportLeber hereditary optic neuropathy associated with antiretroviral therapy for human immunodeficiency virus infection.
Case ReportMitochondrial retinopathies and optic neuropathies: The impact of retinal imaging on modern understanding of pathogenesis, diagnosis, and management.
ReviewIs this article assigned to the wrong chapter(s)? Let us know.