Clinical results were assessed by means of Kaplan–Meier, Kruskal–Wallis, and univariate and multivariate logistic-regression analyses. We also assessed the clinical outcome according to the percentage of holoclone-forming stem cells, detected as cells that stain intensely (p63-bright cells) in the cultures.

Results Permanent restoration of a transparent, renewing corneal epithelium was attained in 76.6% of eyes. The failures occurred within the first year. Restored eyes remained stable over time, with up to 10 years of follow-up (mean, 2.91±1.99; median, 1.93). In post hoc analyses, success — that is, the generation of normal epithelium on donor stroma — was associated with the percentage of p63-bright holoclone-forming stem cells in culture. Cultures in which p63-bright cells constituted more than 3% of the total number of clonogenic cells were associated with successful transplantation in 78% of patients. In contrast, cultures in which such cells made up 3% or less of the total number of cells were associated with successful transplantation in only 11% of patients. Graft failure was also associated with the type of initial ocular damage and postoperative complications.

Conclusions Cultures of limbal stem cells represent a source of cells for transplantation in the treatment of destruction of the human cornea due to burns.

 

Source Information

From San Raffaele Scientific Institute, Ophthalmology Unit, Milan (P.R., S.M., G.P., A.S.); and the Center for Regenerative Medicine Stefano Ferrari, University of Modena and Reggio Emilia, Modena, Italy (M.D.L., G.P.).

This article (10.1056/NEJMoa0905955) was published on June 23, 2010, at NEJM.org.

Address reprint requests to Professor Pellegrini at the Center for Regenerative Medicine Stefano Ferrari, University of Modena and Reggio Emilia, Via Glauco Gottardi 100, Modena, Italy