Long-term predictive outcomes of stem cell therapy for degenerative retinal diseases

Abstract

Background: Stem cell transplantation is a promising therapy for degenerative retinal diseases, including age-related macular degeneration (AMD), retinitis pigmentosa (RP), and Stargardt's disease (STGD). This study quantitatively evaluates long-term outcomes of different stem cell treatments.

Methods: A systematic review and outcome prediction analysis were conducted using data published before September 1, 2024. The primary outcome was the change in best-corrected visual acuity (BCVA), expressed as logarithm of the minimum angle of resolution (LogMAR), adjusted for baseline BCVA. Predictions were disease-specific for dry AMD, wet AMD, RP, and STGD, refined through regression analysis while ensuring data standardization across studies.

Results: A meta-analysis of 43 studies (666 eyes) revealed varying BCVA improvements at 6 months post-treatment. For dry AMD, adipose-derived mesenchymal stem cells (ADMSCs) showed the best improvement (0.65, 95% CI: 0.57-0.72 logMAR), exceeding that of human embryonic stem cells (hESCs, 0.49, 95% CI: 0.33-0.65 logMAR). For wet AMD, hESCs improved BCVA by 0.45 logMAR (95% CI: 0.28-0.61 logMAR). For RP, Wharton's jelly-derived mesenchymal stem cells (WJMSCs) achieved the highest improvement (0.50, 95% CI: 0.28-0.73 logMAR), while umbilical cord-derived mesenchymal stem cells (UCMSCs, 0.39, 95% CI: 0.31-0.46 logMAR) showed moderate effects. For STGD, ADMSCs were most effective (0.53, 95% CI: 0.42-0.64 logMAR).

Conclusions: Stem cell therapy demonstrates significant potential for treating AMD, RP, and STGD, with ADMSCs showing superior benefits in dry AMD and STGD, hESCs being more effective in wet AMD, and WJMSCs outperforming other therapies in RP. These comparative insights highlight disease-specific advantages of different stem cell sources, informing future clinical applications.

Keywords

age-related macular degeneration / degenerative retinal diseases / retinitis pigmentosa / Stargardt's disease / stem cell therapy / systematic review / visual function

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