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ARVO 2016 Annual Meeting Abstracts 282 Macular diseases/dystrophy Monday, May 02, 2016 3:45 PM–5:30 PM Exhibit/Poster Hall Poster Session Program #/Board # Range: 2677–2704/C0128–C0155 Organizing Section: Retina Program Number: 2677 Poster Board Number: C0128 Presentation Time: 3:45 PM–5:30 PM Comparison of Microperimetry and Humphrey Perimetry for Hydroxychloroquine Toxicity Screening Sophia Wong, Eric Chin, Susanna S. Park. University of California Davis, Sacramento, CA. Purpose: Humphrey perimetry (HP) is the most widely used functional test for hydroxychloroquine toxicity screening. Microperimetry (MP) is an alternative that tests macular sensitivity and correlates functional deficits with anatomical location and has the advantage of fixation tracking. In this prospective study, we compared Humphrey 10-2 perimetry and microperimetry in normal subjects and those on hydroxychloroquine (HCQ) therapy to assess their relative sensitivity. Methods: Patients on chronic HCQ therapy for an autoimmune condition and normal subjects were included in this study. HP (Zeiss HFA) and MP (CenterVue MAIA) were performed in the same visit. Tests with poor reliability (greater than 10% fixation loss, false positives or negatives) and poor fixation stability were excluded. Normal HP was defined as zero depressions on the pattern deviation map. Normal MP was defined as normal sensitivity at all test points (compared to normative data provided by the manufacturer: average threshold greater than 26 dB and percent reduced thresholds less than 8%). Results: A total of 4 normal subjects (8 eyes) and 11 subjects on HCQ (22 eyes) were enrolled. One eye from a normal subject and 4 eyes of 3 HCQ subjects were excluded from analysis due to unreliable testing (3 eyes with poor HP reliability and 2 eyes with poor HP reliability and poor MP fixation). Qualitative analysis was carried out on 14 subjects (25 eyes). Among 7 eyes (4 subjects) studied in the normal group, only 4 eyes were normal in both HP and MP. Among 18 eyes (10 subjects) tested in the HCQ group, 5 eyes (27.8%) showed abnormality on HP only, 8 eyes (44.4%) showed abnormality on HP and MP, and 5 (27.8%) showed no abnormality with either method. Four eyes (2 subjects) were diagnosed with toxic maculopathy based on concurrent abnormality on OCT or mfERG. Among these 4 eyes diagnosed with toxic maculopathy, 3 eyes had both abnormal HP and MP and 1 eye had abnormal HP and normal MP. Conclusions: In this small pilot exploratory study, we note that MP with fixation tracking may provide a more reliable method of testing macular function. However, results on HP and MP may not be interchangeable as abnormalities noted with one method were not always reproduced with the other method. Further study with a larger sample is needed to determine the relative sensitivity and specificity of HP versus MP in diagnosing toxic maculopathy secondary to HCQ use. Commercial Relationships: Sophia Wong, None; Eric Chin; Susanna S. Park, None Program Number: 2678 Poster Board Number: C0129 Presentation Time: 3:45 PM–5:30 PM Exacerbation of Macular Edema Associated with Hyperbaric Oxygen Therapy Stephen M. Hypes1, 2, Ashkan Abbey3, 4, Yoshihiro Yonekawa3, 4, Jeremy D. Wolfe3, 4. 1Neurology & Ophthalmology, Michigan State University COM, Dearborn, MI; 2Ophthalmology, Beaumont Health - Southshore Campus, Trenton, MI; 3Associated Retina Consultants, Royal Oak, MI; 4Ophthalmology, William Beaumont Hospital, Royal Oak, MI. Purpose: Hyperbaric oxygen therapy (HBOT) is being marketed as a treatment for retinovascular diseases without scientific evidence. HBOT has been shown to increase VEGF levels in tissues; therefore, we hypothesize HBOT may in fact worsen the vascular permeability of diseased retinal vasculature. We report a patient who developed an abnormally acute and diffuse macular edema from a preexisting retinal arterial macroaneurysm (RAM) following HBOT. Methods: Retrospective clinical analysis of visual acuity (VA), spectral domain optical coherence tomography (SD-OCT), and fluorescein angiography (FA) before and after administration of HBOT in a 71-year-old woman with a preexisting RAM. Results: The patient presented with acute onset of blurry vision OS. BCVA was 20/200, and examination revealed submacular hemorrhage OS and a new RAM as seen on FA. The patient underwent vitrectomy with subretinal tissue plasminogen activator and pneumatic displacement. VA improved to 20/150 one week after surgery. One week post-operative SD-OCT showed trace residual subretinal fluid, and no intraretinal edema. In the following weeks, the patient underwent 7 consecutive sessions of HBOT. At post-operative week 5, VA OS had decreased to 20/200 and a new, acute, diffuse, and severe macular edema was noted. Fluorescein angiography demonstrated increased leakage from the RAM and surrounding angiopathic vessels. The patient was directed to discontinue HBOT, and the edema quickly resolved with intravitreal ranibizumab treatment. Conclusions: HBOT may exacerbate macular edema from retinovascular diseases. We recommend that retinovascular disease not be listed as an indication, but as a relative contraindication for HBOT, until proven otherwise. Commercial Relationships: Stephen M. Hypes, None; Ashkan Abbey, None; Yoshihiro Yonekawa, None; Jeremy D. Wolfe, None Program Number: 2679 Poster Board Number: C0130 Presentation Time: 3:45 PM–5:30 PM Drusen-like deposits in young adults affected by Systemic Lupus Erythematosus Alessandro Invernizzi1, 2, Laura Dell’Arti1, Elena Garoli1, Gaia Leone1, Daniela Galimberti1, Alessandro Santaniello3, Gabriella Moroni4, Francesco Viola1. 1Department of Clinical Sciences and Community Health, University of Milan, Ophthalmological Unit, IRCCS-Cà Granda Foundation – Ospedale Maggiore Policlinico, Milan, Italy; 2Department of Biomedical and Clinical Science - University of Milan, Eye Clinic - Luigi Sacco Hospital, Milan, Italy; 3Department of Clinical Sciences and Community Health, University of Milan, Immunology Unit, IRCCS-Cà Granda Foundation – Ospedale Maggiore Policlinico, Milan, Italy; 4Department of Clinical Sciences and Community Health, University of Milan, Nephrology Unit, IRCCS-Cà Granda Foundation – Ospedale Maggiore Policlinico, Milan, Italy. Purpose: To assess the prevalence of drusen-like deposits in eyes of patients affected by Systemic Lupus Erythematosus (SLE) by the use of a non invasive multimodal imaging approach, to study These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts their size and distribution throughout the fundus and to find possible correlations between these alterations and systemic involvement. Methods: 60 consecutive emmetropic eyes from 60 subjects affected by SLE under 55 years of age, without visual symptoms, and 60 emmetropic eyes from 60 healthy volunteers age and sex matched were enrolled into the study. SLE patients medical history was recorded with particular attention to the presence of SLE American Rheumatology Association diagnostic criteria (ARA). All the included eyes underwent a complete ocular examination and a non invasive multi-imaging assessment including: spectral domain optical coherence tomography (SD-OCT), infrared (IR), fundus autofluorescence (FAF), red-free (RF) and fundus photography (FP). The prevalence, distribution and size (small<63 microns, medium=63-125, large>125) of drusen-like deposits identified by each imaging technique were analyzed in both patients and controls. Correlations between ARA criteria and funduscopic alterations characteristics were studied. Results: Drusen-like deposits were identified in 41.6% of SLE eyes and in 3% of controls resulting in a statistically significant difference (p<0.001) between the two groups. In SLE patients showing drusenlike defects small, medium and large deposits were present in 70.8%, 50% and 41,6% of the eyes respectively. Most involved posterior pole quadrants were temporal (78%) and nasal (72%). Superior and inferior areas were involved in 44% and 48% of cases respectively whereas central field showed the lowest lesions prevalence (24%). No significant correlations were found between the presence of drusen-like deposits and any of the eleven ARA criteria. On the other hand, large deposits and a wider distribution of the lesions (more than 3 quadrants involved) correlated with the presence of SLE glomerulonephritis (p=0.03 and p=0.02 respectively). SD-OCT showed the highest sensitivity (96%) in detecting drusenlike deposits as compared to the other imaging techniques performed in the study. Conclusions: SLE correlates with the presence of drusen-like deposits in young adults. SLE glomerulonephritis correlates with deposits size and distribution. Commercial Relationships: Alessandro Invernizzi; Laura Dell'Arti, None; Elena Garoli, None; Gaia Leone, None; Daniela Galimberti, None; Alessandro Santaniello, None; Gabriella Moroni, None; Francesco Viola, None Program Number: 2680 Poster Board Number: C0131 Presentation Time: 3:45 PM–5:30 PM Macular function assessment with microperimetry in patients with macular dystrophy Jung Lo, Jong-Jer Lee, Wei-Yu Chiang, Hsi-Kung Kuo, Pei-Chang Wu. Ophthalmology, Kaohsiung Chang Gung Memorial Hospital, Taiwan, Kaohsiung, Taiwan. Purpose: The shift of fixation in patient with macular degeneration may impair visual acuity (VA) and daily life. The aim of this study is to assess correlation between fixation shift in microperimetry and visual function in patients with macular dystrophy (MD). Methods: Nineteen patients (age 24-76, mean 56.7 ± 12.2 years) with MD, including 14 females and 5 males, were enrolled in this retrospective study. One eye from each patient with a better VA was analyzed. Fixation shift (FS), defined as the direction and distance between location of fovea and preferred retinal locus (PRL), was determined by superposition of images of microperimetry-1 (MP-1) and spectral domain optical coherence tomography (SD-OCT). The alignment of MP-1 and SD-OCT images based on vascular landmarks was conducted with Photoshop. Pearson’s correlation test (r) was used for analyzing the correlations of parameters. Results: The mean sensitivity (MS) in MP-1 was 8.94 ± 6.18 dB, and the best corrected visual acuity (BCVA) ranged from 20/800 to 20/20. FS was observed in 14 (73.7%) patients, mostly to the supero-nasal quadrant of macula (11/14, 78.5%), and two patients presenting inferior FS had low percentage of fixation points within 2 degree in MP-1 (32 - 33%) and poor BCVA (20/800 - 20/200). Five eyes without FS presented BCVA 20/25 to 20/20 while MS ranging from 1.4 to 17.8 dB. The mean distance of FS was 1080 ± 818 micrometers in patient with FS. The distance of FS was correlated to the logarithm of the minimum angle of resolution (logMAR) VA (r = 0.765, P = 0.001), and the percentage of fixation points within 2 degree in MP-1 (r = -0.586, P = 0.027), but not correlated to MS in MP-1 (r = -0.151, P = 0.606). The percentage of fixation location in MP-1 was correlated to the distance of FS (r = -0.521, P = 0.022), and the central macular thickness in SD-OCT (r = 0.673, P= 0.008). Superimposed fovea and PRL was observed if the ellipsoid zone in fovea was spared without complete degeneration in SD-OCT. Conclusions: The presenting VA is correlated to FS in MD patients. The MS in MP-1 may be reduced before VA and FS deteriorates when MD progresses, and the percentage of fixation location in MP-1 significantly correlates with FS. When compared to VA and SD-OCT, MP-1 could be more sensitive to macular dysfunction when fovea is preserved anatomically, and suitable for early detection of the disease progression. Commercial Relationships: Jung Lo, None; Jong-Jer Lee, None; Wei-Yu Chiang, None; Hsi-Kung Kuo, None; Pei-Chang Wu, None Program Number: 2681 Poster Board Number: C0132 Presentation Time: 3:45 PM–5:30 PM Risk factors of the antimalarial drug’s maculopathy: retrospective study in 3580 patients’ cohort Clémentine David2, Christine Fardeau2, Claude Simon1, Bahram Bodaghi2, Phuc Lehoang2, Juliette Knoeri2, Benedicte Lebrun-vignes3. 1Fédération Electrophysiologie, Hôpital Pitié-Salpétrière, Université Paris VI, Paris, PARIS, France; 2Service d’Ophtalmologie, Centre de Référence en Maladies Rares, Hôpital Ptié-Salpétrière, Université Paris VI, DHU Vision et Handicap, PARIS, France; 3Department Pharmacovigilance, Hôpital PitiéSalpétrière, University Paris VI, Paris., PARIS, France. Purpose: Antimalarial drug are widely prescribed to treat systemic lupus erythematosus, rheumatoid arthritis and other connective tissue These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts disease, and also for the antimalarial prevention. The incidence of the maculopathy is about 1% to 5 years. The main risk factor is the cumulative dose. The daily overdose has also been involved. Some risk factor are conventionally referred, including: age over 60 years, renal or hepatic failure, high body mass index (BMI), and a preexisting retinopathy. The objective of this study is to analyze the statistical value of theses classical risk factors. Methods: This retrospective study was realized from 1.01.2009 to 1.01.2013, in cohort of 3580 patients treated by antimalarial drug. The diagnostic of toxic maculopathy was retained if at least two exams among ocular fundus, macular autofluorescence, central visual field, sd optical tomography, and multifocal electrophysiology, showed compatible signs with the diagnosis. Correlations with the molecule’s nature, and classical risk factors were analyzed. Results: 55 patients had a toxic maculopathy after a treatment period from 3 to 20 years. The cumulative dose differed statistically between chloroquine (CQ) (633±198 grams) and hydroxychloroquine (HCQ) (1681±413 grams, p=0.02). Daily overdose has been found for 2 patients with HCQ and one patient with CQ. High BMI has been found for 3 patients(OR = 1,1). 4 patients had an elevated serum creatinine, 2 patients had elevated serum transaminases(OR = 1,5). 3 patients had a preexisting retinopathy(OR = 1,4). Monitoring for 2 years didn’t showed a poor outcome for theses 3 patients. Conclusions: Two thirds of the patients exhibiting a toxic maculopathy didn’t have any classical risk factors. The presence of a preexisting retinopathy, that can interfere with early diagnosis, didn’t appear like an evolving risk factor. Commercial Relationships: Clémentine David, None; Christine Fardeau, None; Claude Simon, None; Bahram Bodaghi; Phuc Lehoang, None; Juliette Knoeri, None; Benedicte Lebrunvignes, None Program Number: 2682 Poster Board Number: C0133 Presentation Time: 3:45 PM–5:30 PM Comparing macula thickness between sickle cell patients of HbSS and HbSC genotype using spectral domain optical coherence tomography Wei Sing Lim1, Juliana Helou2, Tejal Magan2, Moin Mohamed2. 1 Ophthalmology, Princess Royal University Hospital, London, United Kingdom; 2St Thomas Hospital, London, United Kingdom. Purpose: HbSS patients have less severe proliferative retinopathy but more severe systemic complications compared to HbSC patients. We previously shown mean macula thickness to be lower in HbSS patients compared to HbSC patients based on a small cohort of 41 eyes. We have extended the study here to include 380 eyes to confirm if there is a real difference in macula thickness between HbSS and HBSC patients. Methods: A list of HbSC and HbSS patients seen in the eye clinic at St Thomas Hospital was generated. 197 HbSC eyes and 183 HbSS eyes from 204 patients were included. Eyes with other ocular pathologies that affected macula thickness were excluded. The latest macula OCT were retrospectively analysed. Macula thickness were recorded in all subfields of the ‘early treatment of diabetic retinopathy study ‘ (ETDRS) grid for both right and left eyes. The thickness measured were compared between HbSS and HbSC eyes. 99 HbSC right eyes were compared with 90 HbSS right eyes. 98 HBSC left eyes were compared with 93 HbSS left eyes. Analysis of right and left eyes were not combined. Unpaired t-test was used to verify the significance of the results. Results: The mean age (SD) was 42 (13) and 36(11) for HbSC and HBSS patients respectively. Macula thickness was lower in HBSS eyes compared to HbSC eyes in all subfields of the EDTRS grid but only reached significance at p value of <0.05 in the outer temporal subfields. This statistically significant difference was found in both right and left eyes. The mean outer temporal macula thickness (SD) was 240 (23) microns in HbSC right eyes compared with 231 (26) microns in HbSS right eyes and 242 (21) microns in HbSC left eyes compared with 234 (26) microns in HbSS left eyes. Table 1 and 2 compared the macula thickness of all the other subfields on the EDTRS grid. Conclusions: HbSS eyes have thinner outer temporal macula compared to HbSC eyes. This is likely due to greater retinal ischaemia caused by sickling in HbSS eyes compared to HbSC eyes. This is in contrast to more advanced proliferative sickle retinopathy in HBSC eyes compared to HBSS shown in other studies. Table 1 Macula thickness of HbSC and HbSS right eyes in all subfields of the ETDRS grid Table 2 Macula thickness of HbSC and HbSS left eyes in all subfields of the ETDRS grid Commercial Relationships: Wei Sing Lim, None; Juliana Helou, None; Tejal Magan, None; Moin Mohamed, None Program Number: 2683 Poster Board Number: C0134 Presentation Time: 3:45 PM–5:30 PM Mapping metamorphopsia and central visual field loss in macular disease patients Niall C. Strang1, David B. Yorston2, McGowan Gerard5, David Thomson4, Milena Mihaylova3. 1Life Sciences, Glasgow Caledonian University, Glasgow, United Kingdom; 2Gartnavel Hospital, Glasgow, United Kingdom; 3Sensory Neurobiology, Institute of Neurobiology, Sofia, Bulgaria; 4City University, London, United Kingdom; 5Ophthalmology, University Hospital Ayr, Ayr, United Kingdom. These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Purpose: The card version of D-Charts are effective at mapping changes in metamorphopsia pre- and post-surgery within the central visual field in patients with macular disease. Recently a software version of the D-Charts has been developed to reduce recording time. Here we measure metamorphopsia and central field loss using the software version of the D-Chart and compare the results with the Amsler chart. Furthermore we use the D-Charts to monitor changes in the central visual field that occur during anti-VEGF treatment. Methods: The D-Charts use octagonal grids, divided into eight sectors, formed by black elements (squares) of various densities. The grids are at varying eccentricities between 0.5 and 12 degrees from fixation. Subjects fixate the centre of the D-charts and touch the screen in the distorted sector. The metamorphopsia score (the maximal element separation which produced distortion or field loss) is determined for each ring and sector. The results provide two dimensional maps of metamorphopsia severity and visual loss. Twenty fully corrected subjects with macular disease were tested using the software version of the D-Chart and the Amsler chart. In the second part of the study a further seven subjects were measured during a period of anti-VEGF treatment to examine changes in central field occurring during treatment. Results: The total metamorphopsia score (MTotal) did not correlate with the area of distortion reported on the Amsler chart although the size and location of the field plots using the two charts were similar. The D-Chart provided greater detail relating to the distribution of distortion and field loss and recording was completed in less than 2 minutes per eye. In the second part of the study the D-Chart plots changed in line with the predicted effects of anti-VEGF treatment. Conclusions: The increased information provided by the D-Chart makes comparison with Amsler chart results difficult although the size and location of the field plots were consistent. The software version of the D-Chart is easy to use and maps changes in metamorphopsia and central field loss that occur in patients with macular disease. Commercial Relationships: Niall C. Strang, None; David B. Yorston, None; McGowan Gerard, None; David Thomson, Thomson Software Solutions; Milena Mihaylova, None Program Number: 2684 Poster Board Number: C0135 Presentation Time: 3:45 PM–5:30 PM Clinical and genetic study of late onset Stargardt disease Ada Orrico, Paolo Melillo, Valentina Di Iorio, Settimio Rossi, Michele Della Corte, Francesco Testa, Francesca Simonelli. Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Second University of Naples, Naples, Italy. Purpose: To provide a detailed phenotype characterization of late onset Stargardt disease (STGD1) compared to the juvenile form. Methods: We reviewed the medical charts of STGD1 patients, who underwent best-corrected visual acuity (BCVA), fundus photography, optical coherence tomography, full-field electroretinography, and microperimetry. Statistical analysis of onset age was done to identify a cut-off to discriminate between typical juvenile and late onset forms. Regression models were fitted in order to evaluate the disease progression. The study adhered to the tenets of the Declaration of Helsinki and received approval by the Local Ethics Committee. Results: The study cohort consisted of 205 patients with clinical diagnosis of STGD1 and mutations on both ABCA4 alleles. The onset age showed a bimodal distribution and the cut-off between typical juvenile and late-onset forms was estimated as 30 years. These findings led to the identification of a group of 56 patients with the typical juvenile onset (group A) and 20 patients with onset age over 30 years (group B), with at least one follow-up visit. The age of patients at baseline in group A ranged from 11 to 52 years (29.9±1.4 years), the onset age from 4 to 27 years (17.0±0.7 years) and disease duration from 1 to 46 (11.8±1.4 years). The age of patients at baseline in group B ranged from 38 to 71 years (48.2±1.9 years), the onset age from 32 to 48 years (38.5±1.1 years) and disease duration from 2 to 28 years (9.7±1.5 years). No significant differences (p>0.05) were observed in the Fishman and Lois class distribution. Disease progression was estimated in both groups, with reference to disease length. The BCVA decreased with a rate of 0.093 (p<0.001) and 0.019 (p=0.111) logMAR per year in group A and B, respectively. Both groups showed a significant decrease of MS (β=-0.028, p=0.001 in group A; β=-0.037, p=0.017 in group B), whereas the fixation stability significantly worsened in group A (β=-0.010, p=0.014), while appeared to be stable in group B (β=-0.007, p=0.302). Genetic analysis revealed that missense mutations (particularly G1961E) were more frequent in group B than group A. Conclusions: The current study accurately describes a disease form characterized by a later onset, enabling to estimate its prevalence as 10% of STGD1 population. The late onset form was associated with a milder clinical phenotype and, genetically, more frequent missense mutations compared to typical juvenile STGD1. Commercial Relationships: Ada Orrico, None; Paolo Melillo, None; Valentina Di Iorio, None; Settimio Rossi, None; Michele Della Corte, None; Francesco Testa, None; Francesca Simonelli, None Support: NEY 1R24EY019861-01A1 Program Number: 2685 Poster Board Number: C0136 Presentation Time: 3:45 PM–5:30 PM TEASE: a phase 2 clinical trial assessing the tolerability and effects of oral once-a-day ALK-001 on Stargardt disease Hendrik P. Scholl1, Syed M. Shah1, Christine N. Kay2, Stephen H. Tsang7, Kimberly E. Stepien3, Paul S. Bernstein4, Byron L. Lam5, Michael B. Gorin6, Ilyas Washington7, Leonide Saad8. 1 Ophthalmology, Johns Hopkins University, Baltimore, MD; 2 Vitreoretinal Associates, Gainesville, FL; 3Eye Institute, Medical College of Wisconsin, Milwaukee, WI; 4Moran Eye Center, University of Utah, Salt Lake City, UT; 5Bascom Palmer Eye Institute, University of Miami, Miami, FL; 6Jules Stein Eye Institute, University of California Los Angeles, Los Angeles, CA; 7Harkness Eye Institute, Columbia University Medical Center, New York, NY; 8 Alkeus Pharmaceuticals, Boston, MA. Purpose: Stargardt disease (STGD1) is the leading cause of inherited juvenile macular degeneration. The disease stems from mutations in ABCA4, a gene that encodes a vitamin A transport protein in the retina. Defective vitamin A transport results in accelerated formation of vitamin A dimers, thought to cause retinal degenerations including STGD1. The tolerability and effects of ALK-001 in Stargardt disease (“TEASE”) study is the first of several prospective clinical trials to test the extent to which slowing the dimerization of vitamin A prevents the progression of STGD1. Methods: ALK-001 is a vitamin A replacement: the chemical structure of ALK-001 is similar to that of vitamin A but 3 hydrogen atoms have been exchanged by deuterium. This change prevents ALK-001 from dimerizing. However, ALK-001 keeps all known functions of vitamin A. In preclinical evaluation, ALK-001 slows the rate of vitamin A dimerization approximately 4 to 5 fold but does not affect the visual cycle. TEASE is a 24-month, multicenter, randomized, double-masked, placebo-controlled study evaluating the effects of ALK-001 in up to 50 patients diagnosed with molecularly confirmed STGD1 aged between 12 and 60 years. Study subjects may have any visual acuity to enroll and must have a lesion of welldefined decreased signal on fundus autofluorescence imaging (FAF). These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts TEASE subjects are randomly assigned to receive one of two doses of ALK-001 (30 subjects) or placebo (20 subjects) for one year. After one year, half of the subjects receiving placebo will be randomly crossed over to receive ALK-001 for the following 12 months, while all other subjects will continue receiving their initial treatment. A Data Monitoring Committee (DMC) reviews safety and efficacy data throughout the study. Results: Enrollment is ongoing. As of late 2015, 15 subjects have been enrolled, with the longest treatment duration of about 3 months. The median best corrected visual acuity at baseline was 63 letters (range, 26-85) in the best eye. Median age was 44 years (range, 2059) and median disease duration 8 years (range, 1-25). Median lesion size in the best eye on FAF was 1.80 mm2 (range, 0.25-17.74). Conclusions: The TEASE study is one of the first FDA-regulated phase 2 clinical trials assessing a novel oral compound for retinal degenerative disease secondary to vitamin A dimerization, including STGD1. TEASE trial design Commercial Relationships: Hendrik P. Scholl, None; Syed M. Shah, None; Christine N. Kay, None; Stephen H. Tsang; Kimberly E. Stepien, None; Paul S. Bernstein, None; Byron L. Lam, None; Michael B. Gorin, None; Ilyas Washington, Alkeus Pharmaceuticals (C), Alkeus Pharmaceuticals (P); Leonide Saad, Alkeus Pharmaceuticals, Alkeus Pharmaceuticals (S) Clinical Trial: NCT02402660 Program Number: 2686 Poster Board Number: C0137 Presentation Time: 3:45 PM–5:30 PM Clinical and genetic characteristics of intermediate-onset Stargardt disease Nathalie Bax, Stanley Lambertus, Cecile van der Poort, B. Jeroen Klevering, Frans P. Cremers, Carel C. Hoyng. Ophthalmology, Radboud UMC, Nijmegen, Netherlands. Purpose: To describe the phenotypic and genotypic features in intermediate-onset Stargardt patients. We already described the phenotypes of early- and late-onset Stargardt disease. Methods: Eighty-three Stargardt patients with an age at onset between 11 and 44 years. We reviewed medical records and ophthalmologic examinations for age at onset, best corrected visual acuity (BCVA), ophthalmoscopy, color fundus photography, fundus autofluorescence, fluorescein angiography (FA), spectraldomain optical coherence tomography (SD-OCT), full-field and multifocal electroretinography (ffERG and mfERG), visual field testing and color vision testing. The ABCA4 gene had been analysed for mutations in the protein-coding exons and flanking intronic sequences. A subset of these cases were also analyzed for heterozygous deletions using the MLPA technique. Results: The mean age at onset was 21.18±8.94 years. Mean duration of follow-up was 17.7±13.9 years. BCVA at last recorded visit was 0.93 logMAR (20/170 Snellen). Irregular yellow-white fundus flecks were present at 91% of the patients at first visit. FA showed a dark choroid in 17 out of 58 patients (29.3%). Forty-nine of 50 patients with SD-OCT images showed (central) retinal pigment epithelium loss. On ffERG at first visit in 48 patients, 44 had normal amplitudes (91.7%). mfERG in 23 patients showed reduced or extinguished central responses. Central visual field loss occurred in 57 out of 68 patients (83.8%). Eight of 51 patients (15.7%) tested for color vision defects had normal color vision. Genetic screening of 143 patients revealed ≥2 ABCA4 mutations in 109 patients, single heterozygous mutations in 29 patients, and no mutations in four patients. The mild variant c.2588G>C was identified in a compound heterozygous manner in 33% of the 143 patients, most frequently in combination with the non-canonical splice variants c.768G>T (n= 13) or c.546110T>C (n=7). Based on other studies, both were deemed to be severe variants. Conclusions: We show that intermediate-onset Stargardt patients also form a distinctive group. Patients have better visual acuity than patients with early-onset Stargardt, but show a more severe phenotype than late-onset Stargardt patients. Visual acuity declines progressively in 5-10 years after age at onset. Yellowish fundus flecks are present in the majority of the patients. This phenotype is often caused by a combination of a mild with a severe ABCA4 mutation. Commercial Relationships: Nathalie Bax, None; Stanley Lambertus, None; Cecile van der Poort, None; B. Jeroen Klevering, None; Frans P. Cremers, None; Carel C. Hoyng, None Program Number: 2687 Poster Board Number: C0138 Presentation Time: 3:45 PM–5:30 PM Progression of visual acuity and fundus autofluorescence in recent onset Stargardt disease: ProgStar study Sheila K. West1, Xiangrong Kong1, Beatriz E. Munoz1, Artur V. Cideciyan2, Michel Michaelides3, Ann-Margret Ervin4, Srinivas R. Sadda5, 6, Hendrik P. Scholl1. 1Medicine, Johns Hopkins Wilmer Eye Inst, Baltimore, MD; 2Ophthalmology, Scheie Eye Institute, U Penn, Philadelphia, PA; 3Moorfields Eye Hospital, University of London, London, United Kingdom; 4Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD; 5Doheny Eye Institute, Los Angeles, CA; 6David Geffen School of Medcine, UCLA, Los Angeles, CA. Purpose: The progression rates for functional loss (visual acuity, VA) and structural change (fundus autofluorescence, AF) in Stargardt patients appear to vary by the degree of loss at presentation. Amongst participants of the Progstar study, we evaluated the progression rates in patients who were recently symptomatic, defined as presenting within 2 years of reported symptom onset. Methods: 36 participants (71 eyes) of the 251 participants enrolled in the retrospective study had onset of symptoms within 2 years of their first visit, and had at least one visit with both VA and AF reported. Among them, 65 eyes had ≥2 visits with both VA and AF. VA was reported as best corrected/presenting acuity and converted to LogMAR. Atrophic lesions identified from AF images were graded using a standard protocol, and areas of decreased AF were reported as follows: Definitely decreased AF (DDAF), poorly-demarcated questionably decreased AF, and well–demarcated questionably decreased AF. We used linear models with generalized estimating equations to compare baseline VA and lesion areas, and linear mixed effects models to estimate the yearly progression rate of VA and lesion areas. These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Results: The median age at first visit was 21 years (range, 7 to 63), and 78% were within one year of symptom onset. At the first visit, median LogMAR VA was 0.70 (IQR 0.3-0.9). The median DDAF area was 0, (IQR 0-0.12), and the mean area in the 32% of eyes with DDAF was 1.85mm2. The mean total area of decreased AF was 1.51mm2 (SD=1.88). The median duration of follow-up was 3 years (IQR 2-4 years). In longitudinal analysis, the loss of VA was 0.05 LogMAR (95%CI 0.04-0.07) per year, and a greater rate of VA loss was associated with better initial VA (p<.0001). The yearly progression rate for total area of decreased AF was 0.50 (95%CI: 0.40-0.60)mm2. The yearly progression rate for DDAF was 0.38 (95%CI: 0.27-0.50) mm2. An increase in both DDAF and total lesion area was associated with a loss of VA (p<.001). Conclusions: This cohort of Stargardt participants who reported onset of symptoms within two years had highly variable visual acuity and lesion area, with slow progression per year. Increase in lesion area over time was associated with loss of VA. Commercial Relationships: Sheila K. West, None; Xiangrong Kong, None; Beatriz E. Munoz, None; Artur V. Cideciyan, None; Michel Michaelides, None; AnnMargret Ervin, None; Srinivas R. Sadda, Avalanche (C), Novartis (C), Optos (C), Bayer (C), Allergan (F), Genetech (F), Carl Zeiss Meditec (F), Genetech (C), Stem Cells, Inc (C), Thrombogenics (C), Optos (F), Regeneron (C), Iconic (C), Allergan (C); Hendrik P. Scholl, None Support: Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC, Fort Meade, Maryland (grant numbers W81-XWH-07-1-0720 and W81XWH-09-2-0189). Program Number: 2688 Poster Board Number: C0139 Presentation Time: 3:45 PM–5:30 PM Biofeedback Rehabilitation in Patients with Stargardt disease: a randomized controlled trial Francesco Maria D’Alterio1, Paolo Melillo1, Valentina Di Iorio1, Gaia Olivo2, Anna Prinster3, Arturo Brunetti2, Mario Quarantelli3, Francesco Testa1, Francesca Simonelli1. 1Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Second University of Naples, Naples, Italy; 2Department of Advanced Biomedical Sciences, University of Naples “Federico II”, Naples, Italy; 3Institute of Biostructure and Bioimaging, National Research Council, Naples, Italy. Purpose: Few previous case series studies reported data on microperimetry (MP1) biofeedback rehabilitation in macular diseases, including Stargardt disease (STGD). We performed a randomized clinical trial to evaluate the efficacy of biofeedback rehabilitation of the extrafoveal Preferred Retinal Locus (PRL) of fixation in STGD patients. Methods: 24 patients (aged 29.2±11.8 years; 8F) with ABCA4 related STGD were enrolled and randomized to treatment (TG) or control (CG) group. Inclusion criteria were: best corrected visual acuity (BCVA)≥ 1/20; age ≥8 years; no other ocular or systemic diseases. Patients who underwent ocular surgery or experimental therapy in the last 6 months were excluded. The 2 groups were homogeneous for age (TG 29.66±12.9 years; CG 28,75±11.33 years) and for disease severity (measured with Fishmann and Lois classifications). At the baseline and after 3 months patients underwent an ocular examination including: BCVA measured with ETDRS chart, reading speed (RS) and minimum print size (MPS) measured with Colenbrander Reading Chart and MP1 (NIDEK Technologies). TG underwent 12 biofeedback rehabilitation sessions of 10 min for each eye by the same operator, once a week. Outcome measures were variations between the groups in BCVA and Reading Test parameters (RS, MPS) in the dominant eye (determined by the Miles Test). Measures are presented as mean±SD. Differences in the measures were assessed by unpaired t-test (significant p-value <5%). The study adhered to the tenets of the Declaration of Helsinki and received approval by the Local Ethics Committee; each patient gave informed consent. Results: BCVA in TG improved (5-10 letters) in 8 patients, decreased (10 letters) in 1 patient and remained stable in 3 patients, whereas in CG remained stable in 8 patients and decreased (5-10 letters) in 4 patients. The RS on average increased in the TG (13.4±17.4 words/min) whereas it remained stable in the CG (-0.6±4.0 words/ min). Moreover, after the rehabilitation patients were able to read smaller size letters (-0.5±0.6), while patients in the CG showed no improvement or worsening (-0.08±0.16). Statistical analysis showed that variations of BCVA (p=0.014), RS (p=0.013) and MPS (p=0.005) between the groups were significant. Conclusions: Our findings confirm that MP1 biofeedback rehabilitation improves visual function and reading abilities, thereby ameliorating overall quality of vision in STGD patients. Commercial Relationships: Francesco Maria D'Alterio; Paolo Melillo, None; Valentina Di Iorio, None; Gaia Olivo, None; Anna Prinster, None; Arturo Brunetti, None; Mario Quarantelli, None; Francesco Testa, None; Francesca Simonelli, None Program Number: 2689 Poster Board Number: C0140 Presentation Time: 3:45 PM–5:30 PM Stargardt Disease Expression on a Background of Low Lipofuscin: The Impact of the p.G1961E Mutation of ABCA4 Winston Lee1, Kalev Noupuu1, 3, Jana Zernant1, Kaspar Schuerch1, Peter Gouras1, Stephen H. Tsang1, 2, Janet R. Sparrow1, 2, Rando Allikmets1, 2. 1Ophthalmology, Columbia University, New York, NY; 2Pathology & Cell Biology, Columbia University, New York, NY; 3Ophthalmology, Tartu University, Tartu, Estonia. Purpose: 488nm autofluorescence images and spectral domainoptical coherence tomography scans were analyzed in 195 patients harboring two disease-causing ABCA4 mutations. The cohort was divided into two groups: those with at least one p.G1961E allele (G, n=61) and those with all other ABCA4 mutation combinations (N, n=134). Methods: A longitudinal and cross-sectional analysis of autofluorescence images and spectral domain-optical coherence tomography scans was conducted in 195 patients harboring two disease-causing ABCA4 mutations. The cohort was divided into two groups: those with at least one p.G1961E allele (G, n=61) and those with all other ABCA4 mutation combinations (N, n=134). Results: Groups were age-matched (G, mean=37.4, range=5-79; N, mean=37.9, range=8-83). Fleck distributions were different between groups. A proportion of group G patients exhibited no flecks (28%) or flecks that were distributed and morphologically “lesioncentric” (confined to/and along the contour of the central lesion). Flecks in group N were present in almost all patients (96%), were comparatively more advanced and evenly distributed across the posterior pole. Fleck severity and distribution in group G, but not N, increased with age. Incidence of geographic atrophy (GA) was lower in group G (26%) but measured area increased with fleck severity and age, while GA was evident in a majority of group N (71%) to a more varying extent. GA area never extended beyond the macula in group G while 37% of group N exhibited extensive atrophy across the posterior pole. Group N lesions were often multifocal or contained small “satellite” lesions peripherally that expanded and coalesced at differing rates (mean=1.84 mm2/yr, SD=1.84). Single, well-defined lesions were observed in all group G patients which expanded at a more consistent rate (mean=0.74 mm2/yr, SD=0.55). These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Conclusions: STGD1 expression in patients with the p.G1961E allele is characterized by a focal lesion that progresses to a single, steadily expanding area of GA with lesion-centric flecks. Significant lipofuscin accumulation in all other ABCA4 mutation combinations results in the earlier widespread development of flecks which resorb and progress as GA lesions. Over time, these non-central or “satellite” lesions expand and coalesce across the posterior pole. Commercial Relationships: Winston Lee; Kalev Noupuu, None; Jana Zernant, None; Kaspar Schuerch, None; Peter Gouras, None; Stephen H. Tsang, None; Janet R. Sparrow, None; Rando Allikmets, None Support: NEI EY021163; NEI EY019861 Program Number: 2690 Poster Board Number: C0141 Presentation Time: 3:45 PM–5:30 PM Comparison of en-face Optical Coherence Tomography and Fundus Autofluorescence for assessment of macular lesion area in Stargardt disease Paolo Melillo, Ada Orrico, Valentina Di Iorio, Anna Nesti, Settimio Rossi, Michele Della Corte, Francesco Testa, Francesca Simonelli. Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Second University of Naples, Naples, Italy. Purpose: This study compared the macular lesion area in Retinal Pigmented Epithelium (RPE), computed by an automatic algorithm (sub-RPE slab) from en-face Spectral Domain Optical Coherence Tomography (SD-OCT), with the fundus lesion area, assessed by Fundus Autofluorescence (FAF) imaging, in Stargardt disease (STGD1). Moreover, the progression of RPE lesion area was estimated over a multi-year follow-up in a large STGD1 patient cohort. Methods: We reviewed the medical records of STGD1 patients with mutations in ABCA4 gene, who underwent best-corrected visual acuity (BCVA), fundus photography, FAF, SD-OCT, and full-field electroretinography. Linear models based on GEE were fitted in order to evaluate the disease progression over a multi-year follow-up. BCVA values were converted in LogMAR for statistical analysis. The data are presented as mean ± standard error of mean. The study adhered to the tenets of the Declaration of Helsinki and received approval by the Local Ethics Committee. Moreover, each patient gave written informed consent. Results: We analyzed a subgroup of 25 patients (13 females; aged: 37.1 ± 2.8 years), undergoing both OCT and FAF imaging, to evaluate the correlation between the two parameters. The linear regression model showed a significant correlations between the two parameters (β=0.843; p-value <0.001; R2=0.947). To assess the annual rate of disease progression, a cohort of 101 patients (aged: 30.0 ± 1.4 years), followed up for a mean time of 3.1 ± 0.1 years, was analyzed. Mean BCVA was 20/100 ± 20/1000 for both eyes. The mean RPE lesion area, measured by OCT, was 2.97 ± 0.39 mm2, in right eyes, and 3.16 ± 0.42 mm2, in left eyes. Furthermore, RPE lesion area was significantly correlated with BCVA (β=0.61, p=0.045). The longitudinal analysis revealed a significant enlargement of RPE lesion area at a mean linear rate of 0.146 mm2/ year (p=0.021). Finally, the BCVA significantly worsened at a mean rate of 0.065 logMar (≈3 ETDRS letters) / year (p<0.001). Conclusions: The current study describes, for the first time in literature, a longitudinal analysis of the macular lesion area assessed by SD-OCT in a STGD1 disease cohort, showing a significant progression over the follow-up. Our findings suggest that the evaluation of lesion area by SD-OCT could be a useful measurement of disease progression, in particular to design future clinical trials. Commercial Relationships: Paolo Melillo, None; Ada Orrico, None; Valentina Di Iorio, None; Anna Nesti, None; Settimio Rossi, None; Michele Della Corte, None; Francesco Testa, None; Francesca Simonelli, None Support: NEI 1R24EY019861-01A1 Program Number: 2691 Poster Board Number: C0142 Presentation Time: 3:45 PM–5:30 PM Impact of segmentation density on spectral domain optical coherence tomography (SD-OCT) assessments in Stargardt disease Anamika Jha1, Alexander Ho1, Muneeswar G. Nittala1, Rupert W. Strauss2, Hendrik P. Scholl2, Srinivas R. Sadda1, 3. 1Doheny Eye Institute, Los Angeles, CA; 2Ophthalmology, Johns Hopkins Wilmer Eye Institute, Baltimore, MD; 3David Geffen School of Medicine, UCLA, Los Angeles, CA. Purpose: SD-OCT segmentation algorithms currently do not perform well in segmenting intraretinal layers in eyes with Stargardt disease. These inaccuracies can be managed by tedious manual correction. We compared selective B-scan segmentation strategies in generating mean retinal layer thickness and preserved area data from SD-OCT scans in patients with Stargardt disease (STGD1). Methods: 13 eyes from 13 STGD1 patients were randomly selected from the ongoing Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies. For each eye, a 49-section 20°x20° high resolution SD-OCT scan was collected. The space between each B-scan was approximately 120-125 µm. All 49 B-scans were segmented manually to quantify retinal pigment epithelium (RPE), photoreceptor outer segments (OS) and inner segments (IS), outer nuclear complex (ONC), and inner retina (InR). For each volume scan, mean thickness (MT) and total area (TA) were generated with three different B-scan selection strategies, using: all 49 B-scans; 1 of every 2 B-scans; and an “adaptive” method, containing a subset of (or all) 49 B-scans that the grader deemed as significantly different from adjacent B-scans. The Mann-WhitneyWilcoxon test was used to test for significant (α = 0.05) differences. Results: No statistically significant (p < 0.05) differences were detected between the three strategies (Table 1). For the adaptive strategy, the average # of segmented B-scans was 35 (SD=10; range=22-49). Conclusions: These data suggest that a selective segmentation strategy may be a viable alternative to exhaustive, comprehensive manual segmentation in Stargardt disease. These results may have implications for increasing the efficiency of SD-OCT grading strategies in clinical trials for Stargardt disease and other related macular degenerative disorders. Table 1. Average MT (± SD; µm) and average TA (± SD; mm2) of intraretinal layers quantified by 3 strategies. Commercial Relationships: Anamika Jha, None; Alexander Ho, None; Muneeswar G. Nittala, None; Rupert W. Strauss, None; Hendrik P. Scholl, None; Srinivas R. Sadda, Carl Zeiss Meditec (F), Optos (F) These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Support: Supported by the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC, Fort Meade, Maryland (grant numbers W81-XWH-07-1-0720 and W81XWH-09-2-0189). Clinical Trial: NCT01977846 Program Number: 2692 Poster Board Number: C0143 Presentation Time: 3:45 PM–5:30 PM Ellipsoid Zone Mapping and Outer Retinal Assessment in Stargardt disease Sruthi Arepalli, Elias I. Traboulsi, Justis P. Ehlers. Ophthalmology, Cole Eye Institute, Cleveland, OH. Purpose: Visual decline is a common occurrence in Stargardt disease secondary to macular atrophy with associated ellipsoid zone (EZ) loss. Reliable and objective measurement of the EZ is difficult with conventional methods. This study utilizes a novel EZ mapping analysis tool for en face visualization and volumetric EZ-retinal pigment epithelium (RPE) assessment in eyes diagnosed with Stargardt disease. The purpose of the study is to quantitatively characterize the EZ status in Stargardt disease and correlate the findings with clinical characteristics. Methods: This is an IRB-approved retrospective case series examining the OCT features of Stargardt disease. Macular cube scan data was exported for analysis. A novel EZ mapping tool was utilized to analyze volumetric measurements and en face characterization of relative EZ volume and atrophy. En face characterization included the percentage of macular scan area with total atrophy (EZ-RPE thickness = 0 microns) and percentage of macular scan area with EZ attenuation (EZ-RPE thickness < 20 microns). Clinical characteristics were correlated with EZ mapping features. EZ mapping values were compared to a normal cohort. Results: Thirty-two eyes were included in the study with a diagnosis of Stargardt disease. Clinical features included 20 eyes with macular flecks and 17 eyes with clinical macular atrophy. Mean EZ volume in Stargardt patients was 1.0 +/- 0.33 mm3. In normal eyes, the EZ volume was 1.27 +/- .09 mm3. Mean percentage of macular EZ attenuation area was 42% in Stargardt patients compared to < 1% in normal eyes. Similarly, mean percentage of macular EZ atrophy was 22% compared to < 1% in normal eyes. EZ maps demonstrated variable levels of atrophy [e.g., moderate (Figure 1), severe (Figure 2)]. EZ volume was directly correlated with visual acuity. Conversely, the percentage of EZ attenuation and EZ atrophy were negatively correlated with visual acuity. Conclusions: EZ mapping provided objective quantitative assessment of outer retinal integrity in Stargardt disease. Multiple EZ mapping parameters were correlated with visual acuity. Utilizing EZ mapping may provide a unique opportunity for longitudinal assessment in Stargardt disease and detect early features of macular atrophy. Blue demonstrating moderate EZ loss Blue demonstrating severe EZ loss Commercial Relationships: Sruthi Arepalli, None; Elias I. Traboulsi, Sparks Therapeutics (C), Retrophin (C), Sanofi (C); Justis P. Ehlers Support: NIH/NEI K23-EY022947-01A1; Ohio Department of Development TECH-13-059 These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Program Number: 2693 Poster Board Number: C0144 Presentation Time: 3:45 PM–5:30 PM Progression of late-onset Stargardt disease Stanley Lambertus1, Moritz Lindner2, Nathalie Bax1, Matthias M. Mauschitz2, Matthias Schmid3, Steffen Schmitz-Valckenberg5, Bernhard H. Weber4, Frank G. Holz2, Gert Jan van der Wilt5, Monika Fleckenstein2, Carel C. Hoyng1. 1 Department of Ophthalmology, Radboud university medical center, Nijmegen, Netherlands; 2Department of Ophthalmology and GRADE Reading Center, University of Bonn, Bonn, Germany; 3Informatics and Epidemiology, Institute for Medical Biometry, Bonn, Germany; 4 Institute of Human Genetics, University of Regensburg, Regensburg, Germany; 5Department for Health Evidence, Radboud university medical center, Nijmegen, Netherlands. Purpose: Accurate biomarkers are crucial to determine potential effects of emerging therapeutic trials for Stargardt disease. The natural course of late-onset Stargardt includes a typical phenotype of retinal pigment epithelium (RPE) atrophy that often spares the fovea, and is surrounded by yellow-white flecks. To this end, areas of RPE atrophy could serve as a sensitive monitor for this disease. We therefore performed a retrospective cohort study to show the accuracy of this outcome measure and calculated sample sizes for a simulated trial. Methods: We analyzed retinal features, visual acuity, and RPE atrophy progression using fundus autofluorescence and near-infrared reflectance imaging in 47 late-onset Stargardt patients (disease onset ≥45 years). Endpoints of visual acuity were observed using Turnbull’s estimators. Progression of RPE atrophy was analyzed by a two-level random effects linear mixed model. Sample size calculations were performed for a 2-year study duration and a dropout rate of 15%. Results: Survival analysis yielded a median interval between disease onset and a decline in visual acuity to 20/32, 20/80, and 20/200 of 2.74 (95% confidence interval, 0.54-4.41), 10.15 (95% CI, 6.1311.38), and 11.38 (95% CI, 6.13-13.34) years, respectively. Over time, progression of RPE atrophy was observed (mean, 0.22±0.02 mm/year; p<0.001). If only patients with bilateral RPE atrophy are included in a 2-year study, 32 patients are needed to reach a power of 80% (95% CI, 79.3-82.4), assuming a fixed therapeutic effect size of 30%. Conclusions: The development of RPE atrophy reflects the most prominent feature of disease progression in late-onset Stargardt. Expansion rates appeared to be fairly slow. Nonetheless, measurements of RPE atrophy resulted in a robust biomarker for disease progression. Statistical power of a simulated 2-year interventional study in late-onset Stargardt as a function of sample size. Calculations were performed for an expected effect size of 50 % (red), 40 % (blue) and 30 % (green) reduction of atrophy progression. Shown are means (±95% confidence intervals) of 10000 simulations per data point. Commercial Relationships: Stanley Lambertus, None; Moritz Lindner, Alimera Sciences (R), Carl Zeiss Meditec (F), Heidelberg Engineering (F), Heidelberg Engineering (R), Optos (F), Genentech (F); Nathalie Bax, None; Matthias M. Mauschitz, None; Matthias Schmid, None; Steffen Schmitz-Valckenberg; Bernhard H. Weber, None; Frank G. Holz, Genentech (R), Carl Zeiss Meditec (F), Optos (F), Genentech (F), Heidelberg Engineering (F), Novartis (C), Boehringer Ingelheim (C), Optos (C), Novartis (R), Bayer (C), Bayer (R), Heidelberg Engineering (C), Novartis (F), Merz (C), Genentech (C), Heidelberg Engineering (R), Acucela (C), Bayer (F), Allergan (C); Gert Jan van der Wilt, None; Monika Fleckenstein, Heidelberg Engineering (F), Novartis (R), US20140303013 A1 pending (P), Bayer (R), Genentech (R), Carl Zeiss Meditec (F), Merz (C), Optos (F), Heidelberg Engineering (R), Genentech (F); Carel C. Hoyng, None Support: This study was supported by the Stichting A.F. Deutman Researchfonds Oogheelkunde, Nijmegen, The Netherlands; Deutsche Forschungsgemeinschaft, Bonn, Germany, Grant No FL 658/4-1 and Ho1926/3-1; BONFOR GEROK Program, Faculty of Medicine, University of Bonn, Grant No O-137.0020; and by the following foundations that contributed through UitZicht: Stichting MD fonds, Landelijke Stichting voor Blinden en Slechtzienden, and Oogfonds. The funding organizations had no role in the design or conduct of this research. They provided unrestricted grants. These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Program Number: 2694 Poster Board Number: C0145 Presentation Time: 3:45 PM–5:30 PM Cross-sectional evaluation of microperimetric fixation location and stability in Stargardt disease in the ProgStar study Etienne M. Schonbach1, Mohamed A. Ibrahim1, Rupert W. Strauss1, 2, Xiangrong Kong1, Alexander Ho3, Paul S. Bernstein4, Janet S. Sunness5, Eberhart Zrenner6, Srinivas R. Sadda3, 7, Sheila K. West1, Hendrik P. Scholl1. 1Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD; 2Moorfields Eye Hospital, London, United Kingdom; 3Doheny Eye Institute, Los Angeles, CA; 4Moran Eye Center, University of Utah School of Medicine, Salt Lake City, UT; 5Greater Baltimore Medical Center, Baltimore, MD; 6Center for Ophthalmology, Institute for Ophthalmic Research, Eberhard Karls Universität, Tübingen, Germany; 7David Geffen School of Medicine, Los Angeles, CA. Purpose: To investigate the relationship between microperimetric fixation location (FL) and fixation stability (FS), demographic features, and best-corrected visual acuity (BCVA) in Stargardt disease (STGD1) as part of the multicenter ProgStar study (The Natural History of the Progression of Atrophy Secondary to Stargardt Disease). Methods: We enrolled patients with ABCA4-related STGD1 in a multicenter, prospective, cross-sectional, observational study. We used MP1 data (Nidek Technologies): FL was expressed as the eccentricity of the preferred retinal locus (PRL) from the anatomical fovea, FS as the 95.4 %-bivariate contour ellipse area (BCEA), and best- corrected visual acuity (BCVA) as ETDRS letters. Linear models with generalized estimating equations were used for statistical analysis while accounting for between-eye correlations. Results: Of 238 patients, 105 (44 %) were males. Median age was 32 years (mean, 33.8 years; range, 7- 69 years) and the median duration of symptoms was 9 years (mean, 11.8 years; range, 0- 55 years). Median PRL eccentricity from the fovea was 6° (mean, 6.2°; range, 0°- 25°), median BCEA was 21.4 deg2 (mean, 31.1 deg2; range, 0.370 deg2- 267 deg2), median BCVA was 42 ETDRS letters (mean, 46.2 letters; range, 20- 88 letters). Each year of additional disease duration was associated with 0.11° more eccentric FL (p =0.0008) but not with FS (p =0.936). Each year of later onset of symptoms of STGD1 was associated with 0.14° more central FL (p < 0.0001) and 0.84 deg2 smaller BCEA (p <0.0001). Each year of additional age was associated with 0.67° more central fixation (p =0.0007) and 0.46 deg2 smaller BCEA (p =0.0014). A single linear model best described the relationship between FL and BCVA: one degree farther PRL eccentricity was associated with 2.3 letters loss of BCVA (p <0.0001) and one additional letter of BCVA with 0.15° more central FL (p <0.0001). The association of FL with BCVA was robust after adjusting for BCEA. Pearson correlation coefficients between patients’ right and left eyes were 0.892 (p <0.0001) for FL and 0.851 (p <0.0001) for FS. After 10 years of disease duration, 82 % of patients had more than 2° PRL eccentricity in both eyes. Conclusions: Longer disease duration is associated with an increase of eccentricity and loss of stability of fixation in STGD1. Commercial Relationships: Etienne M. Schonbach; Mohamed A. Ibrahim, None; Rupert W. Strauss, None; Xiangrong Kong, None; Alexander Ho, None; Paul S. Bernstein, None; Janet S. Sunness, None; Eberhart Zrenner, None; Srinivas R. Sadda, Novartis (C), Avalanche (C), Stem Cells Inc (C), Optos (C), Bayer (C), Allergan (F), Carl Zeiss Meditec (F), Genentech (C), Thrombogenics (C), Optos (F), Regeneron (C), Genentech (F), Iconic (C), Allergan (C); Sheila K. West, None; Hendrik P. Scholl, None Support: Supported by the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC, Fort Meade, Maryland (grant numbers W81-XWH-07-1-0720 and W81XWH-09-2-0189) Clinical Trial: NCT01977846 Program Number: 2695 Poster Board Number: C0146 Presentation Time: 3:45 PM–5:30 PM The natural history of the progression of atrophy secondary to Stargardt disease type 4 (Progstar-4 Study): Baseline demographics and ocular characteristics of patients with PROM1 related retinal dystrophy Syed Mahmood A. Shah1, 2, Mohamed Ahmed1, 2, Nadia Junaid1, 2, Saghar Bagheri1, 2, Beatriz E. Munoz2, Rupert W. Strauss2, Etienne M. Schonbach1, 2, Michel Michaelides3, Ann-Margret Ervin2, Hendrik P. Scholl2. 1Quantum Vision Reading Center, Baltimore, MD; 2 Wilmer Eye Institute, Johns Hopkins University, Batlimore, MD; 3 Moorfields Eye Hospital, London, United Kingdom. Purpose: The longitudinal, multicenter ProgStar-4 study aims to characterize the natural history of Prominin-1 (PROM1) related retinal dystrophy (Stargardt disease type 4/STGD4; OMIM #603786). The baseline characteristics of patients enrolled in the ProgStar-4 Study are described. Methods: Patients with disease-causing mutations in the PROM1 gene and associated retinal dystrophy were enrolled. At baseline, best corrected visual acuity (BCVA), fundus autofluorescence (AF, Heidelberg Engineering), optical coherence tomography (OCT, Heidelberg Engineering) and microperimetry (MP-1, Nidek Technologies) were obtained and analyzed by the Quantum Vision Reading Center (QVRC). Results: Ten eyes from 5 patients with mean age of 38.2±7.6 years have been enrolled to date into the study. The mean age at onset of symptoms was 25.8±10.5 years with an average disease duration of 12.4±12.4 years. Mean BCVA at baseline was 60.8±19.2 letters (range: 38-91). All patients had areas of decreased AF with a mean area of 6.2±2.2 mm2. Eight (80%) eyes had poorly demarcated questionably decreased AF (PD-QDAF) and 2 (20%) had definitely decreased AF (DDAF) with one multifocal subfoveal DDAF lesion. Eight out of 10 eyes had an increased edge AF and none had any flecks. Mean central subfield thickness was 181.7±42.0 µm, total macular volume was 6.8 ± 0.6 mm2 and 6 eyes had loss of outer retinal architecture involving the fovea. Mean sensitivity (MS) as measured by MP-1 was 9.5±5.0 dB, with unstable fixation in 4 eyes (40%) and an average 1 standard deviation bi-contour ellipse area (BCEA) of 2.17±3.3 degrees2. Fixation was eccentric in 5 eyes (50%) with an average PRL location of 3.9±3.5 degrees from the center of the anatomic fovea. Conclusions: Progstar-4 is the first multicenter study to prospectively follow patients with STGD4 to characterize disease progression. Our baseline characteristics show that the main AF lesion type was PDQDAF. Four eyes from 2 patients showed preservation of the sub-foveal outer retina, but only one of them had both preservation on OCT and absence of AF lesions involving the fovea. Longitudinal data will provide valuable insights into the natural history of STGD4 and will inform clinical trial design for interventional studies that aim to slow down disease progression. Commercial Relationships: Syed Mahmood A. Shah, None; Mohamed Ahmed; Nadia Junaid, None; Saghar Bagheri, None; Beatriz E. Munoz, None; Rupert W. Strauss, None; Etienne M. Schonbach, None; Michel Michaelides, None; AnnMargret Ervin, None; Hendrik P. Scholl, None Support: The Shulsky Foundation Clinical Trial: NCT02410122 These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Program Number: 2696 Poster Board Number: C0147 Presentation Time: 3:45 PM–5:30 PM Scotopic and photopic macular functions as assessed with microperimetry (MP1) in patients with Stargardt disease type 1 – The SMART Study Mohamed A. Ibrahim6, Rupert W. Strauss6, 1, Xiangrong Kong6, Ann-Margret Ervin6, Alexander Ho2, Janet S. Sunness3, Isabelle S. Audo4, David G. Birch5, Srinivas R. Sadda2, 7, Millena G. Bittencourt6, Hendrik P. Scholl6. 1Moorfields Eye Hospital and UCL Institute of Ophthalmology, London, United Kingdom; 2 Doheny Eye Institute, Los Angeles, CA; 3Greater Baltimore Medical Center, Baltimore, MD; 4CHNO des Quinze-Vingts, DHU Sight Restore, INSERM-DHOS CIC 1423, Sorbonne Universités, UPMC Univ Paris 06, INSERM, CNRS, Institut de la Vision, Paris, France; 5 Retina Foundation of the Southwest, Dallas, TX; 6Wilmer Eye Institute at the Johns Hopkins University, Baltimore, MD; 7David Geffen School of Medicine, UCLA, Los Angeles, CA. Purpose: To characterize scotopic and photopic macular function with MP1S in patients with Stargardt disease type 1 (STGD1) in the SMART Study (Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease) Methods: Patients with confirmed mutations in the ABCA4 gene and STGD1 phenotype were enrolled (sub-cohort of the ProgStar Study). One eye from every patient (best eye) underwent testing using a custom pattern on the MP1S system (Nidek Technologies), before (mesopic; m) and after (scotopic; s) dark-adaptation. Mesopic testing used a white stimulus; scotopic testing a blue stimulus with a combined neutral density filter to align sensitivity with the dynamic range of the MP1S and a 500nm short-pass filter. Tests were graded at the Doheny Image Reading Center. Mean sensitivity (MS) in the ring 4-10 degrees from the fovea was calculated for both the mesopic (mMS) and scotopic (sMS) tests. The distance of center of gravity of fixation points (preferred retinal locus or PRL) from the anatomical fovea was measured along with the 2SD bivariate contour ellipse area (BCEA). Descriptive statistics and correlation coefficients were used to assess the relationship between photopic and scotopic outcome variables. Results: 136 patients from 7 centers were enrolled. 54% were female, 82% were white, mean age was 34.5 (SD±14) years, mean age of onset was 24.6 (SD±13.7) years, and mean disease duration (DD) was 10 (SD±7.3) years. The mean BCVA was 53.4 letters (SD±17.9). The mean mMS was 12.2 (SD±4.6) dB and the mean sMS was 11.1 (SD±5.0) dB with the one-log unit filter. mMS and sMS were highly correlated with Pearson correlation coefficient of 0.81 (p<0.0001). The mean mPRL was 4.9 (SD±3.9) degrees and the mean sPRL was 5.34 (SD±3.8) degrees. mPRL and sPRL were highly correlated with correlation coefficient of 0.77 (p<0.0001). The mean 2SD mBCEA was 26.8 (SD±34.7) deg2 and the mean sBCEA was 30.4 (SD±31) deg2. mBCEA and sBCEA were correlated with correlation coefficients of 0.68 (p<0.0001). Conclusions: In our cohort of patients with STGD1, photopic and scotopic macular performance were highly correlated when assessed using mean macular sensitivity in the perifoveal area. There were no significant differences in the distance of PRL from fovea or the 2SD BCEA of fixation. Commercial Relationships: Mohamed A. Ibrahim, None; Rupert W. Strauss, None; Xiangrong Kong, None; AnnMargret Ervin, None; Alexander Ho, None; Janet S. Sunness, None; Isabelle S. Audo, None; David G. Birch, None; Srinivas R. Sadda, Avalanche (C), Novartis (C), Optos (C), Stem Cell Inc (C), Bayer (C), Thrombogenetics (C), Allergan (F), Carl Zeiss Meditec (F), Genetech (C), Optos (F), Regeneron (C), Genentech (F), Iconic (C), Allergan (C); Millena G. Bittencourt, None; Hendrik P. Scholl, None Support: Supported by the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC, Fort Meade, Maryland (grant numbers W81-XWH-07-1-0720 and W81XWH-09-2-0189). Clinical Trial: NCT01977846 Program Number: 2697 Poster Board Number: C0148 Presentation Time: 3:45 PM–5:30 PM Results from spectral-domain optical coherence tomography (SDOCT) at baseline compared with normative data: The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study Alexander Ho1, Beatriz E. Munoz2, Rupert W. Strauss2, 6, Anamika Jha1, Ngoc Lam1, Zhihong Hu5, David G. Birch3, Eberhart Zrenner4, Sheila K. West2, Srinivas R. Sadda1, 7, Hendrik P. Scholl2. 1Doheny Eye Institute, Los Angeles, CA; 2 Ophthalmology, Johns Hopkins Wilmer Eye Institute, Baltimore, MD; 3Retina Foundation of the Southwest, Dallas, TX; 4Institute for Ophthalmic Research, Centre for Ophthalmology, University of Tübingen, Tübingen, Germany; 5Doheny Image Analysis Laboratory, Doheny Eye Institute, Los Angeles, CA; 6Moorfields Eye Hospital and UCL Institute of Ophthalmology, London, United Kingdom; 7 David Geffen School of Medicine, UCLA, Los Angeles, CA. Purpose: The multicenter observational ProgStar studies aim to characterize the natural history of Stargardt disease (STGD1). Retinal architecture metrics derived from SD-OCT in a subset of patients enrolled into the prospective ProgStar study at baseline visit are compared to those of normal subjects. Methods: Baseline visit SD-OCT images for 113 genetically confirmed, randomly-selected STGD1 participants enrolled at 9 sites into ProgStar were evaluated at a central reading center (Doheny Image Reading Center/DIRC). SD-OCT images from both eyes were graded, when available. 20 eyes from 20 normal subjects were also evaluated. If thickness data were available for both eyes in the STGD1 data subset, one eye was randomly selected for inclusion in the analysis. Proprietary DIRC software was used to generate mean thicknesses in the central subfield and inner ring of the ETDRSgrid for the retinal pigment epithelium (RPE), photoreceptor outer segments (OS) and inner segments (IS), outer nuclear complex (ONC), and inner retina (InR) layers. ProgStar eye data were normalized using Z-scores. Results: The mean age for ProgStar subjects was 33 years (SD=15). The mean age of the normal subjects was 29 years (SD=8). RPE, OS, IS, and ONC thickness measures collected from the STGD1 subset were outside 2 SDs of the normal eye data (Table 1). Conclusions: Outer retinal layers (RPE, OS, IS, ONC) in the STGD1 data subset have statistically significantly less thickness as compared to normals. These differences are important for the design of interventional clinical trials in STGD1. These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts & RPE as well as intense SHRM in the SRS corresponding to the fibrosis Conclusions: Classical clinical/FP stages of BVMD have characteristic corresponding OCT findings. In the previtelliform stage, OCT can reveal SHRM not evident on clinical exam. Early stages VA can be normal, but deteriorates as atrophy & fibrosis appear on exam, and photoreceptor & RPE loss are seen on OCT. OCT appears to be an essential tool for more precise staging of BVMD Table 1. Intraretinal layer metrics (average and SD of mean thickness) for normal eyes and ProgStar baseline visit eyes. Commercial Relationships: Alexander Ho, None; Beatriz E. Munoz, None; Rupert W. Strauss, None; Anamika Jha, None; Ngoc Lam, None; Zhihong Hu, None; David G. Birch, None; Eberhart Zrenner, None; Sheila K. West; Srinivas R. Sadda, Carl Zeiss Meditec (F), Optos (F); Hendrik P. Scholl, None Support: Supported by the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC, Fort Meade, Maryland (grant numbers W81-XWH-07-1-0720 and W81XWH-09-2-0189). Clinical Trial: NCT01977846 Program Number: 2698 Poster Board Number: C0149 Presentation Time: 3:45 PM–5:30 PM Characterization of Best’s Vitelliform Macular Dystrophy (BVMD) on Multimodal Imaging Nizar S. Abdelfattah1, David S. Boyer2, Srinivas R. Sadda1. 1 Ophthalmology, Doheny Eye Institute, South Gate, CA; 2Retina Vitreous Associated Medical Group, Beverly Hills, CA. Purpose: To describe a comprehensive range of BVMD phenotypic features on multimodal imaging in a large cohort & to correlate these features with visual acuity Methods: Cases were identified from the records of a large private practice retina group. Diagnosis was based on clinical exam & EOG. Patients were evaluated by a complete exam, biomicroscopy, color fundus photography (FP), fluorescein angiography & spectral domain OCT. Demographic data, visual function data & imaging findings were recorded for 1 random eye of each subject Results: Fifty eyes were included (69±17 years old, 68% female) in this study. FP analysis revealed eyes in all stages of BVMD. Previtelliform stage (n=12) mean BCVA (mBCVA) was 0.65±0.33, and FPs were normal. OCT showed normal retina or a characteristic vitelliform lesion (subfoveal hyperreflective material (SHRM) between the ellipsoid zone & the retinal pigment epithelium (RPE). Vitelliform stage (n=20) mBCVA was 0.46±0.2. FPs showed yellow and/or mottled lesions, sometimes multifocal. Vitelliform material was evident on OCT, but in 3 cases was located in the sub-RPE space. Pseudohypopyon stage (n=3) mBCVA was 0.1. FPs showed a vitelliform lesion with a horizontally oriented fluid-level separating an upper serous component from a yellowish lower component. OCT through serous areas showed a hyporeflective accumulation in the subretinal space (SRS) with variable RPE atrophy. The inferior yellowish component corresponded to SHRM on OCT. Vitelliruptive stage (n=8) mBCVA was 0.5±0.17. FP images showed a disrupted yellow lesion with a ring of yellowish material. OCT showed multiple vitelliform accumulations in the SRS. Atrophic stage (n=4) mBCVA was 0.19±0.1. FPs showed a vitelliform lesion with atrophy without fibrosis. Corresponding OCT showed residual SHRM in some areas but loss of the overlying retinal bands as well as RPE atrophy. In the cicatricial stage (n=2), mBCVA was 0.2±0.06. FP showed atrophy & definite fibrosis. OCT showed atrophy of the ONL Commercial Relationships: Nizar S. Abdelfattah, None; David S. Boyer, Alcon (R), bayer (C), Santaris (C), KalVista (C), GS (C), genetech (C), Genentech (R), Santen (C), Alcon (C), Aerpio (C), Allergan (R), Ohr (C), Regeneron (C), Regeneron (R), Novartis (C), allergan (C), Novartis (R), Neurotech (C), Nicox (C), ThromboGenics (C); Srinivas R. Sadda, Avalanche (C), Novartis (C), Optos (C), Bayer (C), Allergan (F), Carl Zeiss Meditec (F), Genetech (C), Stem Cells (C), Thrombogenics (C), OPtos (F), Regeneron (C), Genentech (F), Iconic (C), Allergan (C) Program Number: 2699 Poster Board Number: C0150 Presentation Time: 3:45 PM–5:30 PM Acetazolamide for the treatment of cystic macular lesions in children and young adults with hereditary macular dystrophies Sanne K. Verbakel1, John van de Ven1, Linda M.P. Le Blanc1, Joannes M.M. Groenewoud2, Eiko de Jong1, Carel C. Hoyng1. 1 Ophthalmology, Radboud University Medical Center, Nijmegen, Netherlands; 2Epidemiology, Biostatistics, and HTA, Radboud University Medical Center, Nijmegen, Netherlands. Purpose: Little is known regarding the therapeutic effect of oral acetazolamide in the management of cystic macular lesions in children and young adults with hereditary macular dystrophies, despite that this group is of special interest in view of the age of onset of most hereditary macular dystrophies. Therefore, the aim of this retrospective, observational clinical study was to determine the efficacy of oral acetazolamide in the treatment of cystic macular lesions in children and young adults with hereditary macular dystrophies. Methods: We studied a total of 17 children and young-adults with X-linked retinoschisis (XLRS) or dominant cystoid macular dystrophy (DCMD), treated with oral acetazolamide for cystic macular lesions. All patients were assessed for best-corrected visual acuity and foveal zone thickness (FZT) with spectral-domain optical coherence tomography before and during treatment. A reduction of more than 21,7% or 43,8% in FZT, equal to exceeding normal FZT variability, was considered significant in patients with XLRS and DCMD, respectively. Results: Overall, mean FZT decreased from 435,7 µm at baseline to 312,0 µm at the most recent visit while receiving treatment (p<0.001). 5 of 7 (71,4%) XLRS patients and 4 of 10 (40,0%) DCMD These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts patients showed a significant reduction of FZT in at least one eye. 4 of 7 (57,1%) XLRS patients and 2 of 10 (20,0%) DCMD patients showed a reduction in both eyes over a median treatment interval of 14,4 (range; 1-54) months. In 8 of 9 (88,9%) patients, this reduction was already present after one month of treatment. An improvement of visual acuity in one eye was observed in 4 of 17 patients (2 XLRS patients; 2 DCMD patients), but none of the patients showed an improvement of visual acuity in both eyes. Reported minor side effects were limb paresthesias, hypokalemia or fatigue in 10 patients (58,9%) and one patient suffered from the major side effect of kidney stones. Conclusions: We showed that treatment with oral acetazolamide decreases FZT in up to half of the children and young-adults with XLRS or DCMD, and that this effect is already visible within one month in the majority of patients. Further examination will be necessary to study long-term benefit in preserving and restoring normal retinal anatomy in relation to side effects. Commercial Relationships: Sanne K. Verbakel, None; John van de Ven, None; Linda M.P. Le Blanc, None; Joannes M.M. Groenewoud, None; Eiko de Jong, None; Carel C. Hoyng, None Program Number: 2700 Poster Board Number: C0151 Presentation Time: 3:45 PM–5:30 PM Multimodal imaging evaluation of the pseudohypopyon stage in Adult-Onset Foveomacular Vitelliform Dystrophy Liran Tiosano, Edward Averbukh, Itay Chowers. Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel. Purpose: A pseudohypopyon stage (PHS) was described in AdultOnset Foveomacular Vitelliform Dystrophy (AFVD). At this stage, optical coherence tomography (OCT) demonstrate a lesion that is composed of hyperreflective material that was described to settle in the inferior part of the lesion and a hyporeflective material at the superior part. We sought to characterize this stage of the disease. Methods: Clinical and multimodal imaging database including 126 AFVD eyes (n= 68 patients) was evaluated to identify the PHS. All patients in the database were previously genotyped negative for mutations in genes previously associated with AFVD (PRPH2, BEST1, IMPG-1, and IMPG-2). Demographics, clinical characteristics, SD-OCT findings, color and fundus autofluorescence findings and microperimetry studies were retrospectively analyzed. Results: Seven eyes (6 patients; male/female=3/3) harboring PHS, follow-up was 27±7.2 months (range: 18-32). All eyes remained in the PHS during the follow-up. In SD-OCT, hyperreflective material was demonstrated between the retinal pigmented epithelium and the ellipsoid zone in the temporal aspect of the lesion while hyporeflective material was evident nasally in six eyes; one eye had hyporeflective material in the superior lesion portion (P=0.01 for vertical vs. lateral distribution of lesion material). Ellipsoid zone remained intact above the hyperreflective area in 6 eyes, and disrupted above the hyporeflective area in 6 eyes. Mean±SD lesion size (width and height) at baseline were 1518±498 and 212±60 micron, respectively, and 1404±364 and 172±52 micron at the last visit (P=NS). The mean best corrected visual acuity (BCVA) at presentation and at last follow-up was 0.20±0.25 LogMAR and 0.26±0.19 LogMAR, respectively (P=0.014). Microperimetry demonstrated better retinal sensitivity over the hyperreflective compared with the hyporeflective lesion area. FAF demonstrated intense hyperautofluorescence over the hyperreflective area. Conclusions: PHS in AFVD is uncommon. It composes a relatively large lesion where the degenerative process is usually initiated at the nasal side, and does not respect gravity. This process is accompanied by altered retinal function manifested by decreased sensitivity and progressive visual loss at a mean the rate of less than one ETDRS line during 2 years. Commercial Relationships: Liran Tiosano, None; Edward Averbukh, None; Itay Chowers, None Program Number: 2701 Poster Board Number: C0152 Presentation Time: 3:45 PM–5:30 PM Multimodal Imaging Analysis of Cone Photoreceptors Mosaic in Human Juvenile X-linked Retinoschisis Lucia Ambrosio1, 2, James D. Akula1, 2, Tara L. Favazza1, Emily A. Swanson1, Robert J. Munro1, Matthew Swanson1, Anne Moskowitz1, 2, Ronald M. Hansen1, 2, Anne B. Fulton1, 2. 1 Ophthalmology, Boston Children’s Hospital, Boston, MA; 2 Ophthalmology, Harvard Medical School, Boston, MA. Purpose: Juvenile X-linked retinoschisis (XLRS) is characterized by the formation of schitic cavities within the retinal layers. XLRS is a monogenic disease caused by mutation in the RS1 gene. Retinoschisin (RS) is an extracellular binding protein secreted from retinal cells as a disulphide-linked octamer. Although most classes of adult retinal neurons express RS, it is most abundant in photoreceptors. One putative mechanism in XLRS is the failure of RS to cross the photoreceptor membrane to the extracellular space, causing it to accumulate in photoreceptors which become swollen and sick. We studied the cone mosaic in adaptive optics (AO) scanning light ophthalmographic (SLO) images of XLRS retinae. Methods: A 17 year old boy with a Trp96Arg missense mutation in the RS1 gene and a 12 year old boy with an unspecified mutation were studied. Following a standard clinical examination, images of the macula from 2°-10° eccentric along the horizontal meridian were obtained using our multimodal adaptive optics retinal imager (MAORI). Cones were identified in these images using a semiautomated routine. The diameter of every identified cone was measured by calculating the number of pixels at the half-height of the intensity distribution of the cone 2(pixels/π)½. To convert pixels to degrees to microns, the ‘angular subtense’ of 1° of retina was estimated using the parameters of Gullstrand’s Schematic Eye No. 2. Results: The 17 year old XLRS subject presented with Snellen visual acuity 20/38 (1.91 minutes of arc) in the imaged eye. His spherical equivalent was -0.75 D. Central macular thickness was 296 µm; the AO-SLO also showed apparent swelling of the inner segments. Patchy views of the photoreceptors were obtained, between the schitic cavities, in the AO-SLO. Cone diameter was 4.2 µm. In the 12 year old subject, Snellen acuity was 20/100 (5.01 minutes of arc); spherical equivalent was 1.13 D, central macular thickness was 335 µm and cone diameter was 3.5 µm. In contrast, in control subjects (n=7), cone diameter is 3.1±0.45 µm. Conclusions: The diameters of the cones displayed in AO-SLOs were larger in XLRS than normal subjects, consistent with putative swelling. Conceivably the abnormal protein is trapped in the inner segment. However, poor fixation XLRS subjects may have led to more eccentric scans, or, perhaps, only the largest and brightest cones were visible due to disease. Commercial Relationships: Lucia Ambrosio, None; James D. Akula, None; Tara L. Favazza, None; Emily A. Swanson, None; Robert J. Munro, None; Matthew Swanson, None; Anne Moskowitz, None; Ronald M. Hansen, None; Anne B. Fulton, None Support: EY010597 (ABF) These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts Program Number: 2702 Poster Board Number: C0153 Presentation Time: 3:45 PM–5:30 PM Long term visual and anatomic outcomes of Type 1 Macular Telangiectasia Bish Pal1, Celine Sys2, Maria Gkika1. 1Medical Retina, Moorfields Eye Hospital, London, United Kingdom; 2AZ Sint-Lucas, Bruges, Belgium. Purpose: To report visual and anatomic outcomes of different management options in eyes with Type 1 idiopathic Macular Telangiectasia (MacTel) Methods: A retrospective review of patients with symptomatic MacTel Type 1 seen at Moorfields Eye Hospital was performed. Eyes with diabetic retinopathy, age-related macular degeneration, or any other macular pathology were excluded. Demographic data, medical and ocular history, best-corrected visual acuity (BCVA) and central retinal thickness (CRT) measured by optical coherence tomography (OCT) were recorded Results: 12 eyes were included in this study. 6 eyes were treated with macular laser (Group1) and 4 eyes with a combination of treatment (macular laser±intravitreal antivascular endothelial growth factor±intravitreal steroids) (Group2). 2 patients were just observed (Group3). In all treated eyes treatment was initiated if BCVA was less than 6/9 Snellen. Mean age was 48.3±15.3 years. At the last follow-up, there was a mean±SD decrease in BCVA of 1±1.3 lines in Group1, 0.8±1.7 lines in Group2 and 0.5±0.7 lines in Group3. In 5 eyes BCVA was stable. These were eyes that either left untreated because BCVA was better than 6/9 Snellen or their treatment was initiated when BCVA was better than 6/18 Snellen. In 1 eye (Group2) there was an improvement of 1 line. However, BCVA changes are not at a statistically significant level (p=0.105, Wilcoxon Singed Rank Test). Also, CRT changes are not statistically significant (p=0.638, Wilcoxon Singed Rank Test). OCT revealed intraretinal cysts and exudates in all eyes, however their presence is not correlated to BCVA (p=0.777, Spearman Test). BCVA preservation is related to extrafoveal disease manifestation. Disruption of the photoreceptor layer is related to worse initial and final BCVA. Conclusions: Macular laser alone or in combination with other treatments stabilised, but did not improve the BCVA or CRT significantly in eyes with Type 1 MacTel. The suboptimal outcomes may be related to the retrospective nature of the study with small sample size. If the treatment is initiated early in the disease the outcomes maybe better. Commercial Relationships: Bish Pal, Novartis (R); Celine Sys, None; Maria Gkika, None Support: Moorfields Eye Hospital and National Institute of Health Research Grant Program Number: 2703 Poster Board Number: C0154 Presentation Time: 3:45 PM–5:30 PM Comparsion of autofluorescence patterns in patients with myopic neovascular membrane with BCVA Ali Hadi AlHassany, Manju Chandran, Geeta Menon. Retina, Frimley Health NHS Foundation Trust, Swindon, United Kingdom. Purpose: It has been well known that metabolic activity of the photoreceptor- RPE complex can be reflected in distinguished autofluorescence patterns of different pathologies of the retina. These patterns can help early disease detection and monitoring of its activity where conventional colour fundus photographs show little if any of these changes. The current study is to correlate visual outcome with pre-treatment fundus autofluorescence in myopic choroidal neovascular membrane. Methods: 11 eyes from 11 patients were involved in this study with diagnosis of myopic CNV that has been confirmed at the initial visit. Patients’ age range was 23-69 with mean age of 47. Patients’ baseline BCVA was recorded and FAF were recorded and follow up was four weekly. Results: There was an average gain of 19 lettres in patients whether receiving Ranibizumab or Bevacizumab for myopic CNV. The median BCVA for patients before treatment was 64, and the median was 82.75 after first treatment. Subfoveal hypoflourescence on autoflourescence was associated with thickness of more than 300microns in 8 patients, less than 300microns in 2 patients. Hyperflourescence was associated with thickness of less than 300 microns in 1 patient. Conclusions: We tried to correlate the autofluorescence patterns in a group of patients with myopic CNV, before and soon after starting first treatment, with BCVA and OCT images. From our findings, we can notice there is improvement in the majority of patients gaining an average of 19 letters whether those received Bevacizumab or Ranibizumab. We can also notice that there is hypofluorescnence on FAF associated with thickness of more than 300 microns in 8 patients, less than 300 microns in 2 patients. Hyperfluorescence was noticed in 1 patient associated with thickness of less than 300 microns. Using a paired t-test, The two-tailed P value equals 0.0009, the difference is considered statistically very significant. We can conclude, therefore, in the majority of patients there is a gain of average of 19 letters on vision chart associated with hypofluorescence on FAF and thickness of more than 300 microns on OCT. Therefore, patients receiving anti-VEGF treatment for myopic CNV could be followed up and visual prognosis could be anticipated from this association. Commercial Relationships: Ali Hadi AlHassany, None; Manju Chandran, None; Geeta Menon, None Program Number: 2704 Poster Board Number: C0155 Presentation Time: 3:45 PM–5:30 PM OCT Angiography in Adult Onset Foveomacular Vitelliform Dystrophy Komal Joshi, Peter L. Nesper, Amani A. Fawzi, Rukhsana Mirza. Ophthalmology, Northwestern University, Chicago, IL. Purpose: To describe findings of Adult-Onset Foveomacular Vitelliform Dystrophy (AOFVD) on Optical Coherence Tomography Angiography. Methods: Case series of eight consecutive patients (14 eyes) with AOFVD. Patients were studied with Spectral Domain Optical Coherence Tomography (SD-OCT) and OCT Angiography. OCT Angiography images were obtained using the RTVue-XR Avanti system (Optovue Inc., Femont, California) with split-spectrum amplitude-decorrelation angiography (SSADA) software. Main Outcome measures were presence or absence of choroidal neovascularization and any unifying patterns that could be identified on OCT angiography for AOFVD. Results: Eight consecutive patients (14 eyes) were studied with SD-OCT and OCT angiography. Patients with clinical exam findings consistent with AOFVD received SD-OCT. Eyes with hyperreflective material above the RPE and below the Inner Segment/ Outer Segment layer, consistent with vitelliform lesion on SD-OCT were further analyzed with OCT angiography. 1 of 14 eyes (7%) had Choroidal Neovascularization under the vitelliform lesion on OCT angiography. 9 of 14 eyes (64%) had shadowing of superficial/deep capillary plexus onto the outer retina giving the false appearance of choroidal neovasculariztion in the region of the vitelliform lesion. These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record. ARVO 2016 Annual Meeting Abstracts All 14 eyes (100%) had blockage of flow signal under the vitelliform lesion that presented as artifactual loss of choriocapillaris under the vitelliform lesion. Conclusions: OCT angiography can be used as a non-invasive imaging modality to visualize choroidal neovascularization in AOFVD. There are common artifacts that must be considered when analyzing OCT angiography with vitelliform lesions, including shadowing of superficial retinal vessels onto the outer retina and blockage of signal giving the appearance of lost choriocapillaris under the vitelliform lesion. Commercial Relationships: Komal Joshi, None; Peter L. Nesper, None; Amani A. Fawzi, None; Rukhsana Mirza, None These abstracts are licensed under a Creative Commons Attribution-NonCommercial-No Derivatives 4.0 International License. Go to http://iovs.arvojournals.org/ to access the versions of record.