Three-year outcomes of cultured limbal epithelial allografts in aniridia and Stevens-Johnson syndrome evaluated using the Clinical Outcome Assessment in Surgical Trials assessment tool.

[aniridia]

Limbal stem cell deficiency (LSCD ) is an eye disorder in which the stem cells responsible for forming the surface skin of the cornea are destroyed by disease . This results in pain , loss of vision , and a cosmetically unpleasant appearance . Many new treatments , including stem cell therapies , are emerging for the treatment of this condition , but assessment of these new technologies is severely hampered by the lack of biomarkers for this disease or validated tools for assessing its severity . The aims of this study were to design and test the reliability of a tool for grading LSCD , to define a set of core outcome measures for use in evaluating treatments for this condition , and to demonstrate their utility . This was achieved by using our defined outcome set (which included the Clinical Outcome Assessment in Surgical Trials of Limbal stem cell deficiency [COASTL ] tool ) to evaluate the 3 -year outcomes for allogeneic ex vivo cultivated limbal epithelial transplantation (allo-CLET ) in patients who had bilateral total LSCD secondary to aniridia or Stevens-Johnson syndrome . The results demonstrate that our new grading tool for LSCD , the COASTL tool , is reliable and repeatable , and that improvements in the biomarkers used in this tool correlate positively with improvements in visual acuity . The COASTL tool showed that following allo-CLET there was a decrease in LSCD severity and an increase in visual acuity up to 12 months post-treatment , but thereafter LSCD severity and visual acuity progressively deteriorated .

Diseases
Validation

Diseases presenting "loss of vision" symptom

aniridia

coats disease

This symptom has already been validated