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Retinitis Pigmentosa and Therapeutic Approaches: A Systematic Review
Filippo Confalonieri1,2, Antonio La Rosa1, Giovanni Ottonelli1
1Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, 20090 Milan, Italy.
Abstract:
Background: Retinitis pigmentosa (RP) is a group of hereditary retinal dystrophies characterized by progressive degeneration of photoreceptor cells, which results in debilitating visual impairment. This systematic review aims to evaluate the efficacy and safety of emerging treatment modalities for RP, including gene therapy, mesenchymal-cell-based approaches, and supplementary interventions. Methods: A comprehensive search of electronic databases was conducted to identify relevant studies published up to February 2024. Studies reporting outcomes of treatment interventions for RP, including randomized controlled trials, non-randomized studies, and case series, were included. Data extraction and synthesis were performed according to predefined criteria, focusing on assessing the quality of evidence and summarizing key findings. Results: The search yielded 13 studies meeting inclusion criteria, encompassing diverse treatment modalities and study designs. Gene therapy emerged as a promising therapeutic approach, with several studies reporting favorable outcomes regarding visual function preservation and disease stabilization. Mesenchymal-cell-based therapies also demonstrated potential benefits, although evidence remains limited and heterogeneous. Supplementary interventions, including nutritional supplements and neuroprotective agents, exhibited variable efficacy, with conflicting findings across studies. Conclusions: Despite the lack of definitive curative treatments, emerging therapeutic modalities promise to slow disease progression and preserve visual function in individuals with RP. However, substantial gaps in evidence and heterogeneity in study methodologies underscore the need for further research to elucidate optimal treatment strategies, refine patient selection criteria, and enhance long-term outcomes. This systematic review provides a comprehensive synthesis of current evidence and highlights directions for future research to advance the care and management of individuals with RP.
Insights
Emerging treatments like gene therapy show promise for slowing vision loss in retinitis pigmentosa (RP). While mesenchymal cell therapies and supplements have potential, more research is needed to confirm their effectiveness and optimize RP management.
Area of Science:
- Ophthalmology
- Genetics
- Regenerative Medicine
Background:
- Retinitis pigmentosa (RP) encompasses hereditary retinal dystrophies causing progressive photoreceptor degeneration and severe vision loss.
- Current management for RP lacks definitive cures, necessitating exploration of novel therapeutic strategies.
Purpose of the Study:
- To systematically review the efficacy and safety of emerging treatments for retinitis pigmentosa (RP).
- To evaluate gene therapy, mesenchymal cell-based approaches, and supplementary interventions for RP.
Main Methods:
- Comprehensive literature search of electronic databases up to February 2024.
- Inclusion of randomized controlled trials, non-randomized studies, and case series reporting RP treatment outcomes.
- Data extraction and synthesis focused on evidence quality and key findings.
Main Results:
- Thirteen studies met inclusion criteria, evaluating diverse RP treatment modalities.
- Gene therapy demonstrated promise in preserving visual function and stabilizing RP progression.
- Mesenchymal cell therapies showed potential but with limited and heterogeneous evidence; supplementary interventions had variable efficacy.
Conclusions:
- Emerging RP treatments offer potential for slowing disease progression and preserving vision, though not curative.
- Significant evidence gaps and methodological heterogeneity necessitate further research for optimal RP treatment strategies.
- Future research should focus on refining patient selection and enhancing long-term outcomes for RP management.
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