While the success of allograft ocular surface stem cell transplant has been studied in adults, the procedure’s efficacy in children has not been as thoroughly vetted. Exploring this unchartered area, a research team recently found 75% of its pediatric participants had a stable ocular surface for at least a year after the surgery.

The study conducted a retrospective chart review of 429 eyes with limbal stem cell deficiency that required surgical management between 2000 and 2017 at the Cincinnati Eye Institute. Of these, 20 eyes of 13 patients were included. Each patient was 18 or younger at the time of the procedure, received systemic immunosuppression and was followed up for at least 12 months.

After surgery, all patients received oral tacrolimus and mycophenolate mofetil, and 10 patients received oral prednisone tapered over the first three-to-four months. One patient switched from mycophenolate mofetil to azathioprine secondary to gastrointestinal symptoms.

The study’s main outcome measures were ocular surface stability, visual acuity and systemic immunosuppression adverse events. The approximate follow-up time period was six years.

At the last follow-up, 75%) of eyes had a stable ocular surface, 5% developed partial failure and 20% developed total surface failure. The preoperative minimum angle of resolution visual acuity of 1.5 improved to 1.1 at the last follow-up. Additionally, when four eyes of three non-adherent patients were excluded, the results were more pronounced and statistically significant (1.5 improved to 1.0).

The study also reported systemic immunosuppression was well tolerated by all patients with minimal adverse events. Minor adverse side effects included transient gastrointestinal symptoms, elevated lipid profile, oropharyngeal ulcers, metabolic syndrome, acute kidney injury that spontaneously resolved and transient neutropenia (after an episode of enterovirus).

This study marks the first series that looked at allograft ocular surface stem cell transplant with systemic immunosuppression in a pediatric population, the researchers noted in their paper.

The results lead them to believe the procedure may be a reasonable treatment option for children who have significant limbal stem cell deficiency and vision loss.