Combined Limbal Epithelial and Stromal Cell Transplant for Aniridia-Related Keratopathy: A Nonrandomized Clinical Trial.
Stem cell transplant was associated with better ocular surface scores by 3 months in aniridia-related keratopathy.
Combined Limbal Epithelial and Stromal Cell Transplant for Aniridia-Related Keratopathy: A Nonrandomized Clinical Trial.
IMPORTANCE: Advanced aniridia-related keratopathy (aniridia-related keratopathy) is a progressive ocular surface disorder associated with limbal stem cell deficiency and limited effective treatment options.
To evaluate the safety, feasibility, and clinical outcomes of Real Architecture for 3D Tissues-Ocular Surface (RAFT-OS), a tissue-engineered collagen scaffold incorporating allogeneic limbal epithelial stem cells and stromal keratocytes, in adults with advanced aniridia-related keratopathy.
DESIGN, SETTING, AND PARTICIPANTS: This single-arm, open-label nonrandomized clinical trial was conducted at a tertiary referral center in London, United Kingdom.
eye surface health checks showed clear improvement three months after transplant
One early serious adverse event prompted amendment of the manufacturing protocol; no further major RAFT-OS-related safety events occurred.
In untreated fellow eyes, mean (SD) Ocular Surface Score was 8.4 (2.7) at baseline, 8.4 (2.4) at 3 months (mean difference, 0; 95% CI, -2.5 to 2.5), and 8.0 (2.5) at 12 months (mean difference, -0.4; 95% CI, -2.4 to 1.5).
Mean (SD) treated eye BCVA was 2.23 (0.16) logMAR (Snellen equivalent, <20/1600) at baseline and 1.77 (0.67) logMAR (20/1280) at 12 months (mean difference, -0.47; 95% CI, -0.97 to 0.04).
Mean (SD) fellow eye BCVA was 1.47 (0.71) logMAR (Snellen equivalent, 20/640) at baseline and 1.44 (0.75) logMAR (20/640) at 12 months (mean difference, -0.04; 95% CI, -0.44 to 0.37).
In this 9-participant nonrandomized clinical trial, RAFT-OS transplant was feasible and associated with early ocular surface improvement, partly sustained through 12 months, without substantial safety concerns.
Additional controlled studies with longer follow-up are needed to define safety and clinical effects.