Lifetime outcomes of anti-vascular endothelial growth factor treatment for neovascular age-related macular degeneration
Open accessAbstract
Importance: Neovascular age-related macular degeneration (nAMD), the largest single cause of irreversible severe vision loss in high-income countries, can now be treated with vascular endothelial growth factor (VEGF) inhibitors, but to our knowledge, no data on lifetime outcomes are available. Objective: To determine visual acuity (VA) outcomes of anti-VEGF treatment for nAMD in both eyes for patients' remaining lifetime. Design, Setting, and Participants: Multistate modeling using real-world cohort data of 3192 patients with nAMD (>67 000 visits) treated in routine eye clinics in Australia, New Zealand, and Switzerland. Data were analyzed between 2007 and 2015. Exposures: Intravitreal anti-VEGF treatment at the treating physician's discretion and prospective data collection in standardized registry. Main Outcomes and Measures: Visual acuity in both eyes over the remaining lifetime. Results: For the mean remaining lifetime of 11 years, an estimated 12% (n = 371; 95% CI, 345-400) of the sample retained driving VA and an estimated 15% (n = 463; 95% CI, 434-495) reading VA in at least 1 eye. At that time, an estimated 82% of the sample (n = 2629; 95% CI, 2590-2660) had dropped out. Younger age at baseline and more injections during the first year of treatment were associated with better long-term outcomes. Conclusions and Relevance: Anti-VEGF treatment was associated with preserved useful visual acuity in almost 20% of patients over their average remaining lifetime. More than 80% of patients will cease treatment over that time, having likely experienced a deterioration of vision beforehand. This is a remarkable outcome compared with outcomes without intervention, which lead to legal blindness within 3 years of disease onset in 80% of those affected. These findings underline the public health necessity of providing anti-VEGF treatment to persons in need.
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The current nAMD therapeutic paradigm comes with a tremendous treatment burden of repeated intravitreal injections for the lifetime of the patient.
“The current nAMD therapeutic paradigm comes with a tremendous treatment burden of repeated intravitreal injections for the lifetime of the patient.6,24”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [24]
The Abstract describes regular intravitreal anti-VEGF injections and says treatment is long term, often continuing until it is no longer beneficial or the patient dies. But the Discussion reports that more than 80% of patients are no longer being treated toward the end of their remaining life expectancy, so the source does not support injections continuing for every patient’s lifetime. (LLM, conf=0.88)
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Khanani, A. M., Boyer, D. S., Wykoff, C. C., Regillo, C. D., Busbee, B. G., Pieramici, D., Danzig, C. J., Joondeph, B. C., Major, J. C., Turpcu, A., et al. (2024) · eClinicalMedicine · doi:10.1016/j.eclinm.2023.102394 · Cited by 76 · see citing works
“6,24 Prior to receiving ixo-vec, patients in this study required a mean of nearly 10 annualised injections.”
Background
- Authors
- Finger, R. P., Puth, M.-T., Schmid, M., Barthelmes, D., Guymer, R. H., & Gillies, M.
- Affiliations
- University of Bonn (DE)
- The University of Sydney (AU)
- The University of Melbourne (AU)
- Corresponding author
- Finger, Robert P.
- Venue
- JAMA Ophthalmology
- Publisher
- American Medical Association (AMA)
- Published
- 2020-12-01
- Volume
- 138
- Issue
- 12
- Pages
- 1234
- ISSN
- 2168-6165 (ISSN-L)
- PMC
- PMC7563682
- OpenAlex
- W3092667339
- Cited by
- 34
- References
- 25
- Citations by year
- 2026: 9 · 2025: 6 · 2024: 6 · 2023: 8 · 2021: 5
- FWCI
- 2.58
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- JAMA Ophthalmology (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Zurich Open Repository and Archive (University of Zurich) (submittedVersion)
- Topics
- Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
- Health Sciences › Medicine › Ophthalmology › Ocular Diseases and Behçet’s Syndrome
- Health Sciences › Medicine › Radiology, Nuclear Medicine and Imaging › Retinal Imaging and Analysis
- Keywords
- neovascular age-related macular degeneration, anti-VEGF therapy, visual acuity, remaining lifetime, treatment discontinuation, legal blindness, intravitreal injection, age at baseline, multi-state models
- MeSH terms
- Bevacizumab, Ranibizumab, Aged, Aged, 80 and over, Automobile Driving, Female, Humans, Male, Markov Chains, Middle Aged, Reading, Retrospective Studies, Visual Acuity, Treatment Outcome, Choroidal Neovascularization, Angiogenesis Inhibitors, Vascular Endothelial Growth Factor A, Wet Macular Degeneration, Intravitreal Injections
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