The natural history of the progression of atrophy secondary to stargardt disease (ProgStar) studies: Design and Baseline characteristics: Progstar Report No. 1

Ophthalmology

Volumn 123, Issue 4, 2016, Pages 817-828

  Strauss, Rupert W ^a,b,c,m   Ho, Alex ^d   Muñoz, Beatriz ^a   Cideciyan, Artur V ^e   Sahel, José Alain ^f   Sunness, Janet S ^g   Birch, David G ^h   Bernstein, Paul S ⁱ   Michaelides, Michel ^b   Traboulsi, Elias I ^j   Zrenner, Eberhart ^k   Sadda, Srinivas ^d   Ervin, Ann Margret ^a,l   West, Sheila ^a   Scholl, Hendrik P N ^a  

^a JOHNS HOPKINS UNIVERSITY   (United States)

^b MOORFIELDS EYE HOSPITAL   (United Kingdom)

^c MEDICAL UNIVERSITY OF GRAZ   (Austria)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF PENNSYLVANIA   (United States)

^f SORBONNE UNIVERSITÉ   (France)

^g GREATER BALTIMORE MEDICAL CENTER   (United States)

^h RETINA FOUNDATION OF THE SOUTHWEST   (United States)

ⁱ UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^j LERNER RESEARCH INSTITUTE   (United States)

^k UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^l JOHNS HOPKINS BLOOMBERG SCHOOL OF PUBLIC HEALTH   (United States)

^m JOHANNES KEPLER UNIVERSITY LINZ   (Austria)

more..

Author keywords

[No Author keywords available]

Indexed keywords

PROTEIN; PROTEIN ABCA4; RETINOL; UNCLASSIFIED DRUG; ABC TRANSPORTER; ABCA4 PROTEIN, HUMAN;

ADULT; ARTICLE; AUTOFLUORESCENCE; COHORT ANALYSIS; DEMOGRAPHY; DISEASE COURSE; DISEASE SEVERITY; EUROPE; EYE INJURY; FEMALE; FOLLOW UP; GENETIC VARIABILITY; HISTORY; HUMAN; MAJOR CLINICAL STUDY; MALE; MEDICAL RECORD REVIEW; PHENOTYPE; PRIORITY JOURNAL; PROSPECTIVE STUDY; RETROSPECTIVE STUDY; SPECTRAL DOMAIN OPTICAL COHERENCE TOMOGRAPHY; STARGARDT DISEASE; UNITED STATES; VISUAL ACUITY; ADOLESCENT; CHILD; CLINICAL TRIAL; COMPLICATION; FLUORESCENCE ANGIOGRAPHY; FLUORESCENCE IMAGING; GENETICS; GEOGRAPHIC ATROPHY; MACULAR DEGENERATION; METHODOLOGY; MIDDLE AGED; MULTICENTER STUDY; MUTATION; OPTICAL COHERENCE TOMOGRAPHY; PATHOLOGY; PATHOPHYSIOLOGY; PERIMETRY; PHYSIOLOGY; RETINA; RETINAL PIGMENT EPITHELIUM; VISUAL FIELD; YOUNG ADULT;

ADOLESCENT; ADULT; ATP-BINDING CASSETTE TRANSPORTERS; CHILD; DISEASE PROGRESSION; FEMALE; FLUORESCEIN ANGIOGRAPHY; FOLLOW-UP STUDIES; GEOGRAPHIC ATROPHY; HUMANS; MACULAR DEGENERATION; MALE; MIDDLE AGED; MUTATION; OPTICAL IMAGING; PROSPECTIVE STUDIES; RESEARCH DESIGN; RETINA; RETINAL PIGMENT EPITHELIUM; RETROSPECTIVE STUDIES; TOMOGRAPHY, OPTICAL COHERENCE; VISUAL ACUITY; VISUAL FIELD TESTS; VISUAL FIELDS; YOUNG ADULT;

EID: 84954357666     PISSN: 01616420     EISSN: 15494713     Source Type: Journal    
DOI: 10.1016/j.ophtha.2015.12.009     Document Type: Article

References (48)

1. K. Stargardt Ueber familiaere, progressive Degeneration in der Makulagegend des Auges Graefes Arch Clin Exp Ophthalmol 71 1909 534 549
2. R. Allikmets, N. Singh, H. Sun, and et al. A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy Nat Genet 15 3 1997 236 246
3. N.V. Palejwala, M.J. Gale, R.F. Clark, and et al. Insights into autosomal dominant Stargardt-like macular dystrophy through multimodality diagnostic imaging Retina 36 2016 119 130 http://www.ncbi.nlm.nih.gov/pubmed/?term=Palejwala+NV%2C+Gale+MJ%2C+Clark+RF
4. Y. Rotenstreich, G.A. Fishman, and R.J. Anderson Visual acuity loss and clinical observations in a large series of patients with Stargardt disease Ophthalmology 110 2003 1151 1158
5. V. Miraldi Utz, R.G. Coussa, M.J. Marino, and et al. Predictors of visual acuity and genotype-phenotype correlates in a cohort of patients with Stargardt disease Br J Ophthalmol 98 2014 513 518
6. K. Fujinami, J. Zernant, R.K. Chana, and et al. Clinical and molecular characteristics of childhood-onset Stargardt disease Ophthalmology 122 2015 326 334
7. R.C. Eagle Jr., A.C. Lucier, V.B. Bernardino Jr., and M. Yanoff Retinal pigment epithelial abnormalities in fundus flavimaculatus: a light and electron microscopic study Ophthalmology 87 1980 1189 1200
8. C.D. Birnbach, M. Jarvelainen, D.E. Possin, and A.H. Milam Histopathology and immunocytochemistry of the neurosensory retina in fundus flavimaculatus Ophthalmology 101 1994 1211 1219
9. J.R. Sparrow, E. Gregory-Roberts, K. Yamamoto, and et al. The bisretinoids of retinal pigment epithelium Prog Retin Eye Res 31 2012 121 135
10. K. Fujinami, N. Lois, A.E. Davidson, and et al. A longitudinal study of Stargardt disease: clinical and electrophysiologic assessment, progression, and genotype correlations Am J Ophthalmol 155 2013 1075 1088 e13
11. A.V. Cideciyan, M. Swider, T.S. Aleman, and et al. ABCA4 disease progression and a proposed strategy for gene therapy Hum Mol Genet 18 2009 931 941
12. A.V. Cideciyan, M. Swider, S.B. Schwartz, and et al. Predicting progression of ABCA4-associated retinal degenerations based on longitudinal measurements of the leading disease front Invest Ophthalmol Vis Sci 56 2015 5946 5955
13. T.R. Burke, S.H. Tsang, J. Zernant, and et al. Familial discordance in Stargardt disease Mol Vis 18 2012 227 233
14. A.V. Cideciyan, M. Swider, T.S. Aleman, and et al. Reduced-illuminance autofluorescence imaging in ABCA4-associated retinal degenerations J Opt Soc Am A Opt Image Sci Vis 24 2007 1457 1467
15. Kuehlewein L, Hairi A, Ho A, et al. Comparison of manual and semiautomated fundus autofluorescence analysis of macular atrophy in Stargardt disease phenotype. Retina http://www.ncbi.nlm.nih.gov/pubmed/?term=Comparison+of+manual+and+semiautomated+fundus 2015 Nov 18. [Epub ahead of print].
16. Early Treatment Diabetic Retinopathy Study design and baseline patient characteristics. ETDRS report number 7 Ophthalmology 98 5 Suppl 1991 741 756
17. E.Y. Chew, J. Kim, R.D. Sperduto, and et al. Evaluation of the age-related eye disease study clinical lens grading system AREDS report no. 31 Ophthalmology 117 2010 2112 2119 e3
18. D.L. McCulloch, M.F. Marmor, M.G. Brigell, and et al. ISCEV Standard for full-field clinical electroretinography (2015 update) Doc Ophthalmol 130 2015 1 12
19. B. Chen, C. Tosha, M.B. Gorin, and S. Nusinowitz Analysis of autofluorescent retinal images and measurement of atrophic lesion growth in Stargardt disease Exp Eye Res 91 2010 143 152
20. A. Deckert, S. Schmitz-Valckenberg, J. Jorzik, and et al. Automated analysis of digital fundus autofluorescence images of geographic atrophy in advanced age-related macular degeneration using confocal scanning laser ophthalmoscopy (cSLO) BMC Ophthalmol 5 2005 8
21. L. Dandona, and R. Dandona Revision of visual impairment definitions in the International Statistical Classification of Diseases BMC Med 4 2006 7
22. G.A. Fishman, S.G. Jacobson, K.R. Alexander, and et al. Outcome measures and their application in clinical trials for retinal degenerative diseases: outline, review, and perspective Retina 25 2005 772 777
23. D.A. Thompson, R.R. Ali, E. Banin, and et al. Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium Invest Ophthalmol Vis Sci 56 2015 918 931
24. S. Lambertus, R.A. van Huet, N.M. Bax, and et al. Early-onset Stargardt disease: phenotypic and genotypic characteristics Ophthalmology 122 2015 335 344
25. R.W. Beck, M.G. Maguire, N.M. Bressler, and et al. Visual acuity as an outcome measure in clinical trials of retinal diseases Ophthalmology 114 2007 1804 1809
26. K. Ratnam, J. Carroll, T.C. Porco, and et al. Relationship between foveal cone structure and clinical measures of visual function in patients with inherited retinal degenerations Invest Ophthalmol Vis Sci 54 2013 5836 5847
27. F. Testa, P. Melillo, V. Di Iorio, and et al. Macular function and morphologic features in juvenile Stargardt disease: longitudinal study Ophthalmology 121 2014 2399 2405
28. K.G. Csaky, E.A. Richman, and F.L. Ferris 3rd Report from the NEI/FDA Ophthalmic Clinical Trial Design and Endpoints Symposium Invest Ophthalmol Vis Sci 49 2008 479 489
29. E.Y. Chew, T. Clemons, J.P. SanGiovanni, and et al. The Age-Related Eye Disease Study 2 (AREDS2): study design and baseline characteristics (AREDS2 report number 1) Ophthalmology 119 2012 2282 2289
30. J.S. Sunness, E. Margalit, D. Srikumaran, and et al. The long-term natural history of geographic atrophy from age-related macular degeneration: enlargement of atrophy and implications for interventional clinical trials Ophthalmology 114 2007 271 277
31. S. Bearelly, and S.W. Cousins Fundus autofluorescence imaging in age-related macular degeneration and geographic atrophy Adv Exp Med Biol 664 2010 395 402
32. S. Schmitz-Valckenberg, A. Bindewald-Wittich, J. Dolar-Szczasny, and et al. Correlation between the area of increased autofluorescence surrounding geographic atrophy and disease progression in patients with AMD Invest Ophthalmol Vis Sci 47 2006 2648 2654
33. F.G. Holz, C. Bellman, S. Staudt, and et al. Fundus autofluorescence and development of geographic atrophy in age-related macular degeneration Invest Ophthalmol Vis Sci 42 2001 1051 1056
34. W.T. Wong, W. Kam, D. Cunningham, and et al. Treatment of geographic atrophy by the topical administration of OT-551: results of a phase II clinical trial Invest Ophthalmol Vis Sci 51 2010 6131 6139
35. A.A. Khanifar, D.E. Lederer, J.H. Ghodasra, and et al. Comparison of color fundus photographs and fundus autofluorescence images in measuring geographic atrophy area Retina 32 2012 1884 1891
36. K. Fujinami, N. Lois, R. Mukherjee, and et al. A longitudinal study of Stargardt disease: quantitative assessment of fundus autofluorescence, progression, and genotype correlations Invest Ophthalmol Vis Sci 54 2013 8181 8190
37. V.A. McBain, J. Townend, and N. Lois Progression of retinal pigment epithelial atrophy in Stargardt disease Am J Ophthalmol 154 2012 146 154
38. Strauss RW, Munoz BM, Wolfson Y, et al. Assessment of estimated retinal atrophy progression in Stargardt macular dystrophy using spectral-domain optical coherence tomography. Br J Ophthalmol 2015 Nov 14. http://dx.doi.org/10.1136/bjophthalmol-2015-307035. [Epub ahead of print].
39. G. Staurenghi, S. Sadda, U. Chakravarthy, and R.F. Spaide Proposed lexicon for anatomic landmarks in normal posterior segment spectral-domain optical coherence tomography: the IN∗OCT consensus Ophthalmology 121 2014 1572 1578
40. M.A. Lazow, D.C. Hood, R. Ramachandran, and et al. Transition zones between healthy and diseased retina in choroideremia (CHM) and Stargardt disease (STGD) as compared to retinitis pigmentosa (RP) Invest Ophthalmol Vis Sci 52 2011 9581 9590
41. P. Stalmans, M.S. Benz, A. Gandorfer, and et al. Enzymatic vitreolysis with ocriplasmin for vitreomacular traction and macular holes N Engl J Med 367 2012 606 615
42. N.L. Gomes, V.C. Greenstein, J.N. Carlson, and et al. A comparison of fundus autofluorescence and retinal structure in patients with Stargardt disease Invest Ophthalmol Vis Sci 50 2009 3953 3959
43. A.V. Cideciyan, M. Swider, T.S. Aleman, and et al. Macular function in macular degenerations: repeatability of microperimetry as a potential outcome measure for ABCA4-associated retinopathy trials Invest Ophthalmol Vis Sci 53 2012 841 852
44. P. Charbel Issa, E. Troeger, R. Finger, and et al. Structure-function correlation of the human central retina PLoS One 5 2010 e12864
45. G.A. Fishman, M. Farber, B.S. Patel, and D.J. Derlacki Visual acuity loss in patients with Stargardt's macular dystrophy Ophthalmology 94 1987 809 814
46. A. Galor, and D.J. Lee Effects of smoking on ocular health Curr Opin Ophthalmol 22 2011 477 482
47. F. Delori, J.P. Greenberg, R.L. Woods, and et al. Quantitative measurements of autofluorescence with the scanning laser ophthalmoscope Invest Ophthalmol Vis Sci 52 2011 9379 9390
48. A.V. Cideciyan, S.G. Jacobson, T.S. Aleman, and et al. In vivo dynamics of retinal injury and repair in the rhodopsin mutant dog model of human retinitis pigmentosa Proc Natl Acad Sci U S A 102 2005 5233 5238