Test–Retest Variability of Functional and Structural Parameters in Patients with Stargardt Disease Participating in the SAR422459 Gene Therapy Trial

• DOI: 10.1167/tvst.5.5.10
• Publication Date: 2016-10-03T22:32:59Z
• Authors: Maria A. Parker; Dongseok Choi; Laura R. Erker; Mark E. Pennesi; Paul Yang; Elvira N. Chegarnov; Peter N. Steinkamp; Catherine L. Schl...; Claire-Marie Dhae...; Saddek Mohand-Said; Isabelle Audo; Jose Sahel; Richard G. Weleber; David J. Wilson
• Keywords: Stargardt disease; ABCA4

The goal of this analysis was to determine the test-retest variability functional and structural measures from a cohort patients with advanced forms Stargardt Disease (STGD) participating in SAR422459 (NCT01367444) gene therapy clinical trial.Twenty-two participants, aged 24 66, diagnosed STGD, at least one pathogenic ABCA4 mutation on each chromosome trial, were screened over three visits within 3 weeks or less. Functional visual evaluations included: best-corrected acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score, semiautomated kinetic perimetry (SKP) using isopters I4e, III4e, V4e, hill vision (HOV) calculated static fields (SVF) by 184n point centrally condensed grid stimulus size V test target. Retinal changes such as central macular thickness volume assessed spectral-domain optical coherence tomography (SD-OCT). Repeatability coefficients (RC) 95% confidential intervals (CI) for parameter hierarchical mixed-effects model bootstrapping.Criteria statistically significant various parameters found be following: BCVA score (8 letters), SKP V4e (3478.85; 2488.02 2622.46 deg2, respectively), SVF full HOV (VTOT, 14.62 dB-sr), thickness, (4.27 μm 0.15 mm3, respectively).This provides important information necessary if are occurring assessments commonly used measure disease progression STGD. Moreover, is useful future trials assessing safety efficacy treatments STGD.Determination participants stages STGD assess treatment involving patients.