To evaluate the efficacy of a new stem cell treatment for patients with aniridia-related keratopathy (ARK).
Approach:
Study Design: A first-in-human clinical trial involving nine patients with ARK, where one eye received the stem cell treatment and the other served as a control.
Treatment Method: A collagen scaffold was engineered to deliver limbal epithelial stem cells and stromal stem cells to the damaged eye surface.
Key Findings:
Treated eyes showed significant improvement in ocular surface scores, decreasing from 9.4 to 5.9 after three months and to 6.7 after twelve months.
Untreated eyes showed minimal change, with scores starting at 8.4 and ending at 8.0 after one year.
Vision improved by an average of 24 letters on the ETDRS eye test, equivalent to nearly five lines on a standard vision chart.
Limitations:
The study involved a small sample size of only nine patients.
Long-term effects and safety of the treatment remain to be fully evaluated.
Ophthalmologists caring for adults with this condition may encounter persistent inflammation, treatment dependence, and the cumulative ocular effects of childhood disease.
