Efficacy and safety of mesenchymal stem cell therapies in retinitis pigmentosa: a systematic review and meta-analysis.

Background: Retinitis pigmentosa (RP) is a retinal dystrophy and genetically heterogeneous group that causes vision loss and necessitates innovative therapeutic strategies, and mesenchymal stem cell (MSC) therapy has shown potential due to its regenerative and immunomodulatory properties. This meta-analysis aims to evaluate the efficacy and safety of MSC therapies in improving visual outcomes, focusing on the impact of various MSC types, administration methods, and duration of benefits.

Methods: A systematic search of peer-reviewed studies was conducted to identify clinical trials and observational studies investigating MSC therapies for retinal conditions. Outcomes of interest included best-corrected visual acuity (BCVA), central macular thickness, retinal sensitivity, quality of life, and safety profiles. Data were synthesized and analyzed using random-effects meta-analysis to calculate pooled effect sizes and heterogeneity.

Prospero: CRD42024618158.

Results: Eleven studies involving 355 RP patients were included. Umbilical cord-derived MSCs and bone marrow-derived MSCs demonstrated significant short-term improvements in BCVA and retinal function. Subretinal and suprachoroidal delivery methods were associated with better outcomes compared to systemic infusion. Adverse effects were minimal, with transient inflammation being the most reported. The duration of benefits varied, with most studies reporting sustained improvements up to 12 months, while long-term efficacy beyond this period was less conclusive.

Conclusions: MSC therapies show promise in improving visual function and retinal health, with safety profiles supporting their clinical feasibility. However, differences in administration methods and MSC types influence outcomes. Further large-scale, long-term randomized controlled trials are needed to optimize treatment protocols and validate sustained benefits.