Does Stem Cell Implantation Have an Effect on Severity of Retinitis Pigmentosa: Evaluation with a Classification System?

Abstract

Background: Cell replacement therapies have been evaluated in recent years as an alternative for various retinal pathologies to evaluate the therapeutic efficacy of cell therapy, it is important to measure the severity of the disease. The aim of this study was to evaluate the effect of umbilical cord derived Mesenchymal Stem Cell (UC-MSC) implantation on severity of Retinitis Pigmentosa (RP). Methods: This single-center, clinical study included data of 138 eyes of 92 patients who had a confirmed diagnosis of RP and received stem cell implantation to the suprachoroidal area with a surgical procedure. Patients were evaluated before and 1 year after the surgery regarding to the outcome measures of Best Corrected Visual Acuity (BCVA), Optical Coherence Tomography (OCT) and Visual Field (VF) tests. BCVA, VF width and ellipsoid zone (EZ) width on OCT were recorded for each patient and a scoring criterion was established for each variable varying from 0 to 5 depending on its distribution. The cumulative score (from 0 to 15) was used to classify disease severity from grade 0 to 5. Results: All of the patients completed 12-month follow-up period. The median age of the patients was 40.8 years, 46% were female, 77% had been diagnosed within 10 years and 41% had a family history. 79% of the patients with family history had autosomal recessive inheritance pattern. There were statistically significant improvements in the mean BCVA and VF scores during the study (p < 0.05). The mean score and the mean grade of the disease also improved after the treatment (p < 0.05). There was a negative correlation between BCVA improvement and scoring and grading of the disease. Conclusions: This study demonstrated beneficial effect of suprachoroidally applied UC-MSCs on BCVA, VF and the severity score and grade of the disease during 12-month follow-up period. Cell mediated therapy based on the secretion of Growth Factors (GFs) seems to be an effective and safe option for the treatment of degenerative retinal diseases. This classification is simple, produces objective measure of disease severity and gives opportunity to compare the results of different treatment modalities.