Treatment of severe ocular-surface disorders with corneal epithelial stem cell transplantation.

Abstract

THE NEW ENGLAND JOURNAL OF MEDICINE Treatment of Severe Ocular-Surface Disorders With Corneal Epithelial Stem-Cell Transplantation Kazuo Tsubota, MD; Yoshiyuki Satake, MD; Minako Kaido, MD; Naoshi Shinozaki, MS; Shigeto Shimmura, MD; Hiroko Bissen-Miyajima, MD; Jun Shimazaki, MD Background Conditions that destroy the limbal area of the peripheral cornea, such as the Stevens-Johnson syndrome, ocular pemphigoid, and chemical and thermal injuries, can deplete stem cells of the corneal epithelium. The result is scarring and opacification of the normally clear cornea. Standard corneal transplantation cannot treat this form of functional blindness. Methods We performed and evaluated 70 transplantations of corneal epithelial stem cells from cadaveric eyes into 43 eyes of 39 patients with severe ocular-surface disorders and limbal dysfunction. Medical treatment had failed in all patients. The patients had a mean preoperative visual acuity of 0.004 (only being able to count the number of fingers presented by the examiner) in the affected eyes, which satisfies the criteria for legal blindness in most countries. In 28 eyes, we also performed standard corneal transplantation. Stem-cell transplantations were performed as many as four times on 1 eye if the initial results were not satisfactory; 19 eyes had multiple transplantations. Patients were followed for at least one year after transplantation. Results A mean of 1163 days after stem-cell transplantation, 22 of the 43 eyes (51 percent) had corneal epithelialization; of the 22 eyes, 7 eyes had corneal stromal edema and 15 eyes had clear corneas. Mean visual acuity improved from 0.004 to 0.02 (vision sufficient to distinguish the largest symbol on the visual-acuity chart from a distance of 1 m) (P Conclusions Transplantation of corneal epithelial stem cells can restore useful vision in some patients with severe ocular-surface disorders. N Engl J Med. 1999;340:1697-1703