Phase II clinical trial of cultivated oral mucosal epithelial transplantation (COMET) for patients with severe ocular surface disorders.

PURPOSE: To prospectively evaluate the safety and efficacy of cultivated oral mucosal epithelial transplantation (COMET) for the treatment of severe ocular surface disorders (OSDs). METHODS: This phase II, nonrandomized clinical trial involved patients with severe OSDs (i.e., Stevens-Johnson syndrome, ocular cicatricial pemphigoid, or severe chemical/thermal injury) who underwent COMET at the Department of Ophthalmology, Kyoto Prefectural University of Medicine, Kyoto, Japan. Patients were assigned to three groups according to the purpose of the surgery: visual improvement (VI) group, epithelial repair (ER) group, or release of adhesion (RA) group. The primary outcomes (i.e., from before surgery to 24-weeks post COMET) were change in distance vision, change in epithelial abnormality score, and change in fornix shortening score, respectively. The secondary outcomes included any change in Ocular Surface Grading Score (OSGS) and subjective symptoms evaluated by National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) findings. All intra- and postoperative adverse events were recorded. RESULTS: Of the 27 registered patients in this study, 22 (i.e., 14 SJS cases, 5 OCP cases, and 3 severe chemical/thermal injury cases) underwent COMET. At 24-weeks postoperative, logMAR visual acuity and the fornix shortening score were significantly improved in the VI group (11 cases, 2.2 to 1.5, p = 0.0060) and RA group (5 cases, 4.7 to 1.2, p = 0.0010), respectively. The total epithelial abnormality score improved in 50% of the ER-group patients (4 cases). All OSGS and NEI-VFQ scores improved, and no serious adverse events were observed. CONCLUSION: COMET is safe and effective for treating severe OSDs.