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Published on: May 25, 2020
Microperimetry is a valuable tool for assessing changes in visual function following voretigene neparvovec-rzyl gene
Caio Marques1,2, Mariana Matioli da Palma1,2, Huber Martins Vasconcelos Junior1,2
1Department of Ophthalmology and Visual Sciences, Universidade Federal de São Paulo, Escola Paulista de Medicina, São Paulo, Brazil.
Introduction:
Gene therapy with voretigene neparvovec-rzyl was approved primarily based on improvements observed in the Multi-Luminance Mobility Test (MLMT), while the full-field stimulus threshold (FST) test, a secondary endpoint, also demonstrated efficacy. However, the MLMT is difficult to apply in routine practice, and the FST does not provide spatial information regarding retinal function, which limits direct structural-function correlation.
Purpose:
Therefore, this study investigated whether microperimetry could serve as a simpler, more practical alternative by evaluating its changes after this gene therapy.
Methods:
Mean sensitivity and fixation parameters were evaluated in a cohort of 5 patients (10 eyes) who underwent microperi-metry following gene therapy in São Paulo, Brazil.
Results:
Results demonstrated a significant increase in mean sensitivity (MS, p = 0.0006) and a better perception of dimmer stimuli (p = 0.0004), indicating higher sensitivity. Results from individual eyes showed consistent improvements in retinal sensitivity and fixation stability. These findings were consistent with improvements in daily functioning-particularly in low-light conditions-reported by the patients during medical history assessment during the routine visits.
Conclusion:
Overall, the findings suggest that microperimetry could be an useful tool for monitoring functional outcomes after gene therapy.
