Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)

Kong, Xiangrong; Strauss, Rupert W.; Michaelides, Michel; Cideciyan, Artur V.; Sahel, José‐Alain; Muñoz, Beatriz; West, Sheila K.; Scholl, Hendrik P. N.; Wolfson, Yulia; Bittencourt, Millena; Shah, Syed Mahmood; Ahmed, Mohammed; Schönbach, Etienne M; Fujinami, Kaoru; Traboulsi, Elias I.; Ehlers, Justis P.; Marino, Meghan J; Crowe, Susan; Briggs, Rachael; Borer, Angela; Pinter, Anne; Fecko, Tami; Brugnoni, Nikki; Sunness, Janet S.; Applegate, Carol A.; Russell, Leslie; Moore, Anthony T.; Webster, Andrew R.; Connor, Sophie; McCudden, Victoria; Pefkianaki, Maria; Aboshiha, Jonathan; Liew, Gerald; Holder, Graham E.; Robson, Anthony G.; King, Alexa; Narvaez, Daniela Ivanova Cajas; Barnard, Katy; Grigg, Catherine; Dunbar, Hannah; Obadeyi, Yetunde; Girard-Claudon, Karine; Swann, Hilary; Rughani, Avani; Amoah, Charles; Carrington, Dominic; Bibi, Kanom; Co, Emerson Ting; Carter, Andrew; Georgiou, Anne; Lewis, Selma A.; Shaheen, Saddaf; Shinmar, Harpreet; Burton, Linda M.; Bernstein, Paul S.; Wegner, Kimberley; Sawyer, Briana Lauren; Carlstrom, Bonnie; Farnsworth, Kellian; Fry, Cyrie; Chandler, Melissa; Jenkins, Glen; Creel, Donnel; Birch, David G.; Wang, Yizhong; Rodriguez, Luis; Locke, Kirsten; Klein, Martin; Mejia, Paulina; Jacobson, Samuel G.; Schwartz, Sharon; Matsui, Rodrigo; Gruzensky, Michaela; Román, Alejandro J.; Zrenner, Eberhart; Nasser, Fadi; Hahn, Gesa Astrid; Wilhelm, Barbara; Peters, Tobias; Beier, Benjamin; Koenig, Tilman; Krämer, Susanne; Mohand‐Saïd, Saddek; Audo, Isabelle; Laurent-Coriat, Caroline; Sliesoraitytė, Ieva; Zeitz, Christina; Boyard, Fiona; Tran, Minh Ha; Chapon, Mathias; Chaumette, Céline; Amaudruz, Juliette; Ganem, Victoria J.; Sancho, Serge; Girmens, Aurore; Wojciechowski, Robert; Khan, Shazia; Emmert, David; Cain, Dennis; Herring, Mark; Bassinger, Jennifer; Liberto, Lisa; Ervin, Ann‐Margret; Dreger, Kurt; Jones, Jennifer; Sadda, Srinivas R; Jha, Anamika; Ho, Alex; Kramer, Brendan; Hariri, Amirhossein; Blanquel, Gloria; Lam, Ngoc; Pitetta, Sean; Shi, Yue; Tawdros, Rita; Petrossian, Christine; Jenkins, Dennis; Gupta, Muneeswar; Zhou, Yong Dong; Aguilar, Katherine Elizabeth Gonzaga; Chan, Cynthia; Santos, Lisa; Seo, Brian; Sison, Christopher; Pérez, Silvia; Chao, Stephanie; Miyasato, Kelly; Higgins, Julia; Luna, Zoila; Menchaca, Anita; Gonzalez, Norma; Robledo, Vicky; Carig, Karen; Baker, Kirstie; Ellenbogen, David; Russell, J. Ronaldson; Bluemel, Daniel; Moreno, Álex; Pham, Royal; Sanford, Theo; Linares, Daisy; Tran, Mei

doi:10.1016/j.ophtha.2016.05.027

Cited by 63 publications

(60 citation statements)

References 23 publications

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“…The success of clinical trials evaluating efficacy is largely dependent on sensitive and reproducible outcome measures. Visual acuity is unlikely to be an appropriate outcome measure for STGD1 treatment trials with 1-year duration given its slow and also inconsistent progression [19,20]. ProgStar was therefore designed to evaluate different imaging modalities (FAF, SD-OCT) and psychophysical tests (mesopic MP) that may serve as surrogate endpoints.…”

Section: Discussionmentioning

confidence: 99%

Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) Study: Design and Baseline Characteristics (Report No. 1)

et al. 2018

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Purpose: To describe the study design and characteristics at first visit of participants in the longitudinal Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) study. Methods: Scotopic microperimetry (sMP) was performed in one designated study eye in a subset of participants with molecularly proven ABCA4-associated Stargardt disease (STGD1) enrolled in a multicenter natural history study (ProgStar). Study visits were every 6 months over a period ranging from 6 to 24 months, and also included fundus autofluorescence (FAF). Results: SMART enrolled 118 participants (118 eyes). At the first visit of SMART, the mean sensitivity in mesopic microperimetry was 11.48 (±5.05; range 0.00–19.88) dB and in sMP 11.25 (±5.26; 0–19.25) dB. For FAF, all eyes had a lesion of decreased autofluorescence (mean lesion size 3.62 [±3.48; 0.10–21.46] mm²), and a total of 76 eyes (65.5%) had a lesion of definitely decreased autofluorescence with a mean lesion size of 3.46 (±3.60; 0.21–21.46) mm². Conclusions: Rod function is impaired in STGD1 and can be assessed by sMP. Testing rod function may serve as a potential outcome measure for future clinical treatment trials. This is evaluated in the SMART study.

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Section: Discussionmentioning

confidence: 99%

Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) Study: Design and Baseline Characteristics (Report No. 1)

et al. 2018

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“…Visual acuity was then divided into categories of visual impairment as proposed by the World Health Organization [22,23] 3) (i.e., blindness) [22].…”

Section: Discussionmentioning

confidence: 99%

The Progression of the Stargardt Disease Type 4 (ProgStar-4) Study: Design and Baseline Characteristics (ProgStar-4 Report No. 1)

et al. 2018

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Background/Aims: To describe the design and baseline characteristics of patients enrolled in the multicenter, prospective natural history study of Stargardt disease type 4. Methods: Fifteen eligible patients aged 6 years and older at baseline, harboring disease-causing variants in the PROM1 gene, and with specified ocular lesions were enrolled. They were examined at baseline using a standard protocol, with 6 monthly follow-up visits for a 2-year period including best-corrected ETDRS visual acuity, spectral-domain optical coherence tomography, fundus autofluorescence (FAF), mesopic and scotopic microperimetry (MP). Areas of definitely decreased FAF (DDAF) and questionably decreased FAF were outlined and quantified on FAF images. Results: Amongst the 15 patients (29 eyes) that were enrolled at 5 centers in the USA and Europe, 10 eyes (34.5%) had areas of DDAF with an average lesion area of 3.2 ± 3.5 mm² (range 0.36–10.39 mm²) at baseline. The mean retinal sensitivity of the posterior pole derived from mesopic MP was 8.8 ± 5.8 dB. Conclusions: Data on disease progression in PROM1-related retinopathy from this study will contribute to the characterization of the natural history of disease and the exploration of the utility of several modalities to track progression and therefore to potentially be used in future interventional clinical trials.

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“…Although there are political proposals to detect visual problems, most are focal and expensive, without continuity, and do not ensure access to specialized consultations and corrective actions (9) . However, simple and cheap actions such as the ophthalmologic exam made by qualified personnel, in spite of not being specialists, allows the diagnosis and treatment of ocular conditions with early referrals for ophthalmologists (10) . Studies conducted in different nations reveal that deficient knowledge in eye health care can contribute to the onset of blindness (11) .…”

Section: Discussionmentioning

confidence: 99%

“…Therefore, the proposal of eye self-examination is essential for enabling people to learn throughout life, and seek the development of personal skills. It is a task to be accomplished at schools, homes, workplaces and other community spaces (10) . In this regard, the effective ocular health promotion involves a combination of a few components, namely, guided education for adoption of prevention measures, health service improvements, and a greater accessibility and expansion of public prevention policies on ocular disorders (15) .…”

Section: Discussionmentioning

confidence: 99%

Evaluation of the self-eye examination method for health promotion

Lima

Nascimento²,

Silva

et al. 2018

Rev. esc. enferm. USP

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Objective:To evaluate the self-eye examination practice as a tool to promote the ocular self-evaluation. Method: Quasi-experimental study developed in a university of Piauí with sample of 324 students between January and May 2014. A team of thirteen researchers made observations during the self-examination followed by an ocular screening. Results: There was no agreement on the hypothesis of defense that the ocular exam could help to observe the eyes health, p-value>0.3. However, the exam was considered as easy to accomplish (99.7%) and that not only the physician can perform the eye exam (99.4%). The awareness about eye care is identified as the main purpose of this booklet (97.6%), (χ 2 = 186.01; p=0.001). The results highlight the booklet can be used in the teaching process of self-eye examination (84.6%). Although the defense that its fulfilment can replace the consultation with the ophthalmologist (23.8%%) cannot be accepted (χ 2 = 46.34), the self-exam must be done routinely (82.4%). Conclusion: The results prove that the learning through virtual booklet is possible and supports self-care with the eyes by performing the self-exam.

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Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)

Cited by 63 publications

References 23 publications

Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) Study: Design and Baseline Characteristics (Report No. 1)

Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) Study: Design and Baseline Characteristics (Report No. 1)

The Progression of the Stargardt Disease Type 4 (ProgStar-4) Study: Design and Baseline Characteristics (ProgStar-4 Report No. 1)

Evaluation of the self-eye examination method for health promotion

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