July 2018
Volume 59, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2018
Test-Retest Variability of Functional and Structural Parameters in patients with Usher Syndrome Type-1B (USH1B)
Author Affiliations & Notes
  • Maria A Parker
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Andreas Lauer
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Tim Stout
    Cullen Eye Center, Baylor College of Medicine, Houston, Texas, United States
  • Mark E Pennesi
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Paul Yang
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • David J Wilson
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Dongseok Choi
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
    OHSU-PSU School of Public Health, Oregon Health & Science University, Portland, Oregon, United States
  • Laura R Erker
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Brandon J Lujan
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Ou Tan
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Pierre-Olivier Barale
    Institut de la Vision, Sorbornne Universités, Paris, France
  • Caroline Cohen
    Sanofi, Paris, France
  • Saddek Mohand-Said
    Institut de la Vision, Sorbornne Universités, Paris, France
  • Isabelle S Audo
    Institut de la Vision, Sorbornne Universités, Paris, France
  • Jose A. Sahel
    Institut de la Vision, Sorbornne Universités, Paris, France
    Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, United States
  • Richard G Weleber
    Casey Eye Institute, Oregon Health & Sciences University, Portland, Oregon, United States
  • Footnotes
    Commercial Relationships   Maria Parker, None; Andreas Lauer, Accreditation Council for Graduate Medical Education (R), Allergan (F), American Board of Ophthalmology (S), Clearside Biomedical (F), Genentech (F), NIH/NEI (F), Oxford BioMedica (F); Tim Stout, Oxford Biomedica (F), Oxford Biomedica (C); Mark Pennesi, AGTC (C), AGTC (S), Astellas Pharmaceuticals (C), Biogen (C), Editas (C), FFB (S), Gensight (C), IONIS Pharmaceuticals (C), Nacuity Pharmaceuticals (C), NightstaRX (S), Ophthotech (C), ProQR Therapeutics (C), RegenexBio (C), Sanofi (S), Sparks Therapeutics (C); Paul Yang, FFB (F); David Wilson, None; Dongseok Choi, None; Laura Erker, None; Brandon Lujan, BioTime (C), Cell Cure Neurosciences (C), Genentech/Roche (R), Optovue (F), UC Berkeley (P), Zeiss (F), Zeiss (R); Ou Tan, Optovue (F), Optovue (P); Pierre-Olivier Barale, None; Caroline Cohen, Sanofi (E); Saddek Mohand-Said, None; Isabelle Audo, None; Jose Sahel, Banque publique d'Investissement (F), Choroideremia Research Foundation (S), Chronocam (I), Chronolife (I), ERC Synergy "HELMHOLTZ" (F), FFB (F), FFB (S), Fondation NRJ de l’Institut de France (S), Fovea pharmaceuticals (S), Genesignal (C), GenSight Biologics (I), GenSight Biologics (C), LabEx LIFESENSES (F), Pixium Vision (I), Pixium Vision (C), Scientific Advisory Board of the Paris Region (S), Sparing Vision (I), Steering Committee of the European Vision Institute (S), Steering Committee of the French Translational Research Program in Health (S), Tilak Healthcare (I), Vision Medicines Member of the Strategic Committee of the City of Paris (S); Richard Weleber, AGTC (S), AGTC (C), FFB (R), FFB (S), OHSU (P), Sanofi (C)
  • Footnotes
    Support  The study was funded by Sanofi.
Investigative Ophthalmology & Visual Science July 2018, Vol.59, 3896. doi:https://doi.org/
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      Maria A Parker, Andreas Lauer, Tim Stout, Mark E Pennesi, Paul Yang, David J Wilson, Dongseok Choi, Laura R Erker, Brandon J Lujan, Ou Tan, Pierre-Olivier Barale, Caroline Cohen, Saddek Mohand-Said, Isabelle S Audo, Jose A. Sahel, Richard G Weleber; Test-Retest Variability of Functional and Structural Parameters in patients with Usher Syndrome Type-1B (USH1B). Invest. Ophthalmol. Vis. Sci. 2018;59(9):3896. doi: https://doi.org/.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : The goal of this analysis is to determine the test re-test variability of functional and structural measures from a cohort of patients with advanced forms of retinitis pigmentosa (RP) secondary to USH1B.

Methods : Nine participants aged 22-56 years diagnosed with RP caused by MY07A mutation were enrolled in the SAR421869 (NCT01505062) gene therapy clinical trial and screened over three visits within three weeks or less. Functional visual evaluations included: best corrected visual acuity (BCVA) ETDRS letter score, kinetic perimetry (KP) isopter area (I4e, III4e, V4e, and VI4e); and central 30-degree static perimetry (SP) using a 185-loci grid (size V) and a 111-loci grid (size VI) with volumetric endpoints from hill of vision modeling (VFMA). Retinal structural changes were assessed by spectral domain optical coherence tomography (SD-OCT) and included central macular thickness (CMT), macular volume (MV) and ellipsoid zone (EZ) area. Repeatability coefficients (RC) and 95% confidence intervals (CI) were calculated for each parameter by using hierarchical mixed-effects model and bootstrapping.

Results : The criteria for statistically significant changes were the following: BCVA (11 letter score); KP 19.29 (I4e), 90.78 (III4e), 190.37 (V4e) and 141.88 (VI4e) dg2; 30 degree HOV 0.64 (size V) and 1 (size VI) dB-sr; CMT, MV and EZ-area (22.65 μm, 0.11 mm3 and 0.14 mm2 respectively). The RCs and associated CIs are summarized in Table 1.

Conclusions : Test-retest variability repeatability coefficients will serve as a reference in determining whether measured changes in structural and functional outcomes are truly due to disease progression. Moreover, this information will serve to assess efficacy and safety in treatment trials involving patients with Usher Syndrome Type-1B.

This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.

 

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