A single-arm, open-label nonrandomized clinical trial evaluated RAFT-OS — a tissue-engineered collagen scaffold seeded with allogeneic limbal epithelial stem cells and stromal keratocytes — in 9 adults with advanced aniridia-related keratopathy (ARK), with untreated fellow eyes as natural history comparators, over 12 months.
Treated eyes showed meaningful ocular surface improvement: mean OSS dropped from 9.4 at baseline to 5.9 at 3 months (mean difference −3.6; 95% CI −5.6 to −1.6) and 6.7 at 12 months (mean difference −2.8; 95% CI −4.5 to −1.0), while untreated fellow eyes were essentially unchanged. BCVA improved from 2.23 to 1.77 logMAR at 12 months (mean difference −0.47; 95% CI −0.97 to 0.04), though the CI crossed zero. One early serious adverse event led to a protocol amendment; 2 participants developed persistent epithelial defects.
- Very small sample (n=9), single-arm design with no randomized control — limits ability to draw causal conclusions. - Fellow eye comparator is imperfect; disease severity differed at baseline (OSS 9.4 vs 8.4). - 12-month follow-up may be insufficient to assess durability or late safety signals.
RAFT-OS transplant appears feasible and safe enough to proceed to larger trials in advanced ARK, with early ocular surface gains that partially persist at 1 year. Clinicians should not yet adopt this outside a trial setting — controlled studies with longer follow-up are still needed.
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