학술논문
Faster Sensitivity Loss around Dense Scotomas than for Overall Macular Sensitivity in Stargardt Disease: ProgStar Report No. 14
Document Type
Clinical report
Author
Schonbach, Etienne M.; Strauss, Rupert W.; Ibrahim, Mohamed A.; Janes, Jessica L.; Birch, David G.; Cideciyan, Artur V.; Sunness, Janet S.; Munoz, Beatriz; Ip, Michael S.; Sadda, SriniVas R.; Scholl, Hendrik P.N.; Wolfson, Yulia; Bittencourt, Millena; Shah, Syed Mahmood; Ahmed, Mohamed; Schonbach, Etienne; Fujinami, Kaoru; Traboulsi, Elias; Ehlers, Justis; Marino, Meghan; Crowe, Susan; Briggs, Rachael; Borer, Angela; Pinter, Anne; Fecko, Tami; Burgnoni, Nikki; Applegate, Carol; Russell, Leslie; Michaelides, Michel; Degli Esposti, Simona; Moore, Anthony; Webster, Andrew; Connor, Sophie; Barnfield, Jade; Salchi, Zaid; Alfageme, Clara; McCudden, Victoria; Pefkianaki, Maria; Aboshiha, Jonathan; Liew, Gerald; Holder, Graham; Robson, Anthony; King, Alexa; Cajas Narvaez, Daniela Ivanova; Barnard, Katy; Grigg, Catherine; Dunbar, Hannah; Obadeyi, Yetunde; Girard-Claudon, Karine; Swann, Hilary; Rughani, Avani; Amoah, Charles; Carrington, Dominic; Bibi, Kanom; Ting, Emerson; Illiyas, Mohamed Nafaz; Begum, Hamida; Carter, Andrew; Georgiou, Anne; Lewism, Selma; Shaheen, Saddaf; Shinmar, Harpreet; Burton, Linda; Bernstein, Paul; Wegner, Kimberley; Sawyer, Briana Lauren; Carlstrom, Bonnie; Farnsworth, Kellian; Fry, Cyrie; Chandler, Melissa; Jenkins, Glen; Creel, Donnel; Birch, David; Wang, Yi-Zhong; Rodriguez, Luis; Locke, Kirsten; Klein, Martin; Mejia, Paulina; Jacobson, Samuel G.; Schwartz, Sharon B.; Matsui, Rodrigo; Gruzensky, Michaela; Charng, Jason; Roman, Alejandro J.; Zrenner, Eberhart; Nasser, Fadi; Hahn, Gesa Astrid; Wilhelm, Barbara; Peters, Tobias; Beier, Benjamin; Koenig, Tilman; Kramer, Susanne; Sahel, Jose-Alain; Mohand-Said, Saddek; Audo, Isabelle; Laurent-Coriat, Caroline; Sliesoraityte, Ieva; Zeitz, Christina; Boyard, Fiona; Tran, Minh Ha; Chapon, Mathias; Chaumette, Celine; Amaudruz, Juliette; Ganem, Victoria; Sancho, Serge; Girmens, Aurore; Wojciechowski, Robert; Khan, Shazia; Emmert, David G.; Cain, Dennis; Herring, Mark; Bassinger, Jennifer; Liberto, Lisa; West, Sheila; Ervin, Ann-Margret; Kong, Xiangrong; Dreger, Kurt; Jones, Jennifer; Sadda, Srinivas; Jha, Anamika; Ho, Alex; Kramer, Brendan; Lam, Ngoc; Tawdros, Rita; Zhou, Yong Dong; Carmona, Johana; Uji, Akihito; Hariri, Amirhossein; Lock, Amy; Elshafei, Anthony; Ganegoda, Anushika; Petrossian, Christine; Jenkins, Dennis; Strnad, Edward; Baghdasaryan, Elmira; Ito, Eric; Samson, Feliz; Blanquel, Gloria; Akil, Handan; Melendez, Jhanisus; Lei, Jianqin; Huang, Jianyan; Chau, Jonathan; Falavarjani, Khalil G.; Espino, Kristina; Li, Manfred; Mendoza, Maria; Nittala, Muneeswar Gupta; Roded, Netali; Saleh, Nizar; Huang, Ping; Pitetta, Sean; Balasubramanian, Siva; Leahy, Sophie; Srinivas, Sowmya J.; Velaga, Swetha B.; Margaryan, Teresa; Tepelus, Tudor; Brown, Tyler; Fan, Wenying; Murillo, Yamileth; Shi, Yue; Aguilar, Katherine; Chan, Cynthia; Santos, Lisa; Seo, Brian; Sison, Christopher; Perez, Silvia; Chao, Stephanie; Miyasato, Kelly; Higgins, Julia; Luna, Zoila; Menchaca, Anita; Gonzalez, Norma; Robledo, Vicky; Carig, Karen; Baker, Kirstie; Ellenbogen, David; Bluemel, Daniel; Sanford, Theo; Linares, Daisy; Tran, Mei; Nava, Lorane; Oberoi, Michelle; Romero, Mark; Chiguil, Vivian; Bynum-Bain, Grantley; Kim, Monica; Mendiguren, Carolina; Huang, Xiwen; Smith, Monika; Sarreal, Natalie
Source
American Journal of Ophthalmology. August 2020, Vol. 216, 219
Subject
Language
English
ISSN
0002-9394
Abstract
Highlights: * This study reports a novel automated approach to quantify progression of visual dysfunction. * This automated approach was validated relative to a manual grading. * The progression rate at the disease front in Stargardt disease is reported. * The new method may allow shorter clinical trials or smaller cohorts or both. Purpose Mean sensitivity (MS) derived from a standard test grid using microperimetry is a sensitive outcome measure in clinical trials investigating new treatments for degenerative retinal diseases. This study hypothesizes that the functional decline is faster at the edge of the dense scotoma (eMS) than by using the overall MS. Design Multicenter, international, prospective cohort study: ProgStar Study. Methods Stargardt disease type 1 patients (carrying at least 1 mutation in the ABCA4 gene) were followed over 12 months using microperimetry with a Humphrey 10-2 test grid. Customized software was developed to automatically define and selectively follow the test points directly adjacent to the dense scotoma points and to calculate their mean sensitivity (eMS). Results Among 361 eyes (185 patients), the mean age was 32.9 [plus or minus] 15.1 years old. At baseline, MS was 10.4 [plus or minus] 5.2 dB (n = 361), and the eMS was 9.3 [plus or minus] 3.3 dB (n = 335). The yearly progression rate of MS (1.5 [plus or minus] 2.1 dB/year) was significantly lower ([beta] = -1.33; P < .001) than that for eMS (2.9 [plus or minus] 2.9 dB/year). There were no differences between progression rates using automated grading and those using manual grading ([beta] = .09; P = .461). Conclusions In Stargardt disease type 1, macular sensitivity declines significantly faster at the edge of the dense scotoma than in the overall test grid. An automated, time-efficient approach for extracting and grading eMS is possible and appears valid. Thus, eMS offers a valuable tool and sensitive outcome parameter with which to follow Stargardt patients in clinical trials, allowing clinical trial designs with shorter duration and/or smaller cohorts.