title: Comparison of outcomes from a phase 3 study of age-related macular degeneration with a matched, observational cohort.
abstract: OBJECTIVE
  To compare outcomes of intravitreal therapy from an observational study cohort with those of participants receiving treatment in the Minimally Classic/Occult Trial of the Anti-VEGF Antibody Ranibizumab (MARINA) for the treatment of neovascular age-related macular degeneration (wet AMD).
  
  
  METHODS
  Database observational study. Participants in the observational cohort were chosen to match demographic features and entry criteria of the treatment group from MARINA. Outcomes over 12 months were compared.
  
  
  METHODS
  Eight hundred twenty-one anti-vascular endothelial growth factor (anti-VEGF)-naïve eyes treated with ranibizumab with 12 months or more of follow-up were included in the total Fight Retinal Blindness! (FRB-All) cohort, whereas a subset of this cohort of 401 eyes who were matched to the MARINA treatment group were included as the FRB-MARINA cohort.
  
  
  METHODS
  Intravitreal ranibizumab therapy of 0.5 mg for wet AMD.
  
  
  METHODS
  Visual acuity (VA) in logarithm of the minimum angle of resolution (logMAR) letters and treatments given were recorded continuously and anonymously in an electronic database for 12 months. Locally weighted scatterplot smoothing (LOESS) regression was used to plot change in visual acuity data over the course of 12 months for both the FRB-All cohort and the FRB-MARINA cohort, whereas results from the MARINA trial were taken from the published study report.
  
  
  METHODS
  Change in VA in logMAR letters over 12 months, treatment, and visit intensity.
  
  
  RESULTS
  Mean visual acuity improvement after 12 months in FRB-MARINA (+5.5 letters) was similar to that of the 0.5-mg group from MARINA (+7.2 letters). Improvement in FRB-ALL was slightly less (+4.9 letters). Mean treatment effect compared with the MARINA control group was similar for the MARINA treated group (+17.6 letters) and the FRB-MARINA cohort (+15.9 letters). A mean of 7.3 injections in 12 months was received by the observational cohorts.
  
  
  CONCLUSIONS
  Similarity of mean VA improvement in the matched observational cohort with that of the phase 3 clinical trial suggests that these results can be achieved in real-world clinical practice with a modified treatment regimen.
authors.affiliation: The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, Australia. Electronic address: mark.gillies@sydney.edu.au.
authors.forename: Mark C
authors.initials: MC
authors.lastname: Gillies
authors.affiliation: The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, Australia.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: Walton
authors.affiliation: Marsden Eye Specialists, Parramatta, Australia.
authors.forename: Jennifer J
authors.initials: JJ
authors.lastname: Arnold
authors.affiliation: Lions Eye Institute, Center for Ophthalmology and Vision Science, University of Western Australia, Australia.
authors.forename: Ian L
authors.initials: IL
authors.lastname: McAllister
authors.affiliation: School of Public Health, University of Sydney, Sydney, Australia.
authors.forename: Judy M
authors.initials: JM
authors.lastname: Simpson
authors.affiliation: Retina Associates, Chatswood, Australia.
authors.forename: Alex P
authors.initials: AP
authors.lastname: Hunyor
authors.affiliation: Department of Ophthalmology, Centre for Eye Research Australia, University of Melbourne, Royal Victorian Eye and Ear Hospital, Melbourne, Australia.
authors.forename: Robyn
authors.initials: R
authors.lastname: Guymer
authors.affiliation: Academic Unit of Ophthalmology, Australian National University, Canberra, Australia.
authors.forename: Rohan W
authors.initials: RW
authors.lastname: Essex
authors.affiliation: University of Western Australia, Department of Population Health, Perth, Australia.
authors.forename: Nigel
authors.initials: N
authors.lastname: Morlet
authors.affiliation: The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, Australia; Department of Ophthalmology, University Hospital Zurich, University of Zurich, Zurich, Switzerland.
authors.forename: Daniel
authors.initials: D
authors.lastname: Barthelmes
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use; D005500:Follow-Up Studies; D058449:Intravitreal Injections; D000069579:Ranibizumab; D016896:Treatment Outcome; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors; D014792:Visual Acuity / Q000502:physiology; D057135:Wet Macular Degeneration / Q000188:drug therapy / Q000503:physiopathology; D017326:Clinical Trials, Phase III as Topic; D016032:Randomized Controlled Trials as Topic; D015337:Multicenter Studies as Topic
chemical_list: D020533:Angiogenesis Inhibitors; D061067:Antibodies, Monoclonal, Humanized; D000069579:Ranibizumab; D042461:Vascular Endothelial Growth Factor A; C467484:VEGFA protein, human