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Microperimetry in Three Inherited Retinal Disorders.
Laura Bagdonaite-Bejarano1, Ronald M Hansen1, Anne B Fulton1
1a Ophthalmology Department , Boston Children's Hospital/Harvard Medical School , Boston , MA , USA.
Seminars in Ophthalmology
|June 1, 2019
Summary
Microperimetry (MP) is feasible for assessing inherited retinal disorders in gene therapy trials. While showing condition-specific patterns, MP results exhibit variability, requiring careful interpretation for treatment efficacy evaluation.
Area of Science:
- Ophthalmology
- Genetics
- Clinical Trials
Background:
- Microperimetry (MP) assesses central retinal visual sensitivity.
- MP is a potential outcome measure for gene therapy trials.
- Inherited retinal disorders (IRDs) like choroideremia, Stargardt disease, and X-linked juvenile retinoschisis affect the central retina and are targets for gene therapy.
Purpose of the Study:
- To review microperimetry (MP) results in three IRDs undergoing gene therapy trials.
- To evaluate the feasibility and utility of MP as an outcome measure in these trials.
Main Methods:
- Review of microperimetry (MP) data from patients with choroideremia, Stargardt disease, and X-linked juvenile retinoschisis.
- Analysis of MP sensitivity maps, fixation stability, and patterns of sensitivity loss.
Main Results:
- Microperimetry (MP) is feasible across the studied inherited retinal disorders.
- MP sensitivity maps reflect the distinct effects of each condition on the central retina.
- Significant variability in fixation stability and sensitivity loss patterns exists within each disorder.
Conclusions:
- Microperimetry (MP) is valuable for monitoring central retinal function in individuals and can aid gene therapy efficacy evaluation, especially with multimodal imaging (OCT, autofluorescence, acuity).
- Variability in MP parameters necessitates caution when using it as an outcome measure in small gene therapy trials.
- MP is expected to remain a relevant tool in future gene therapy research for inherited retinal disorders.
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