Elicit: Clinical Outcomes of Aflibercept in AMD and DME
Clinical Outcomes of Aflibercept in AMD and DME
What clinical outcomes support aflibercept treatment of wet AMD and diabetic macular edema?
Aflibercept treatment for wet AMD and diabetic macular edema is supported by visual acuity improvements equivalent to ranibizumab and superior to laser photocoagulation, significant anatomical improvements including central retinal thickness reductions and diabetic retinopathy severity improvements, clinically meaningful quality of life gains, and a safety profile comparable to other anti-VEGF agents with the advantage of less frequent dosing.
Abstract
Aflibercept treatment for wet age-related macular degeneration produces visual acuity outcomes equivalent to ranibizumab, with both treatments showing similar mean BCVA changes (within 0.5 letters) and approximately 32% of patients gaining ≥15 letters at one year. Anatomical outcomes were also similar, with comparable proportions achieving dry retina (RR 1.06, 95% CI 0.98 to 1.14) and equivalent choroidal neovascularization area reductions. For diabetic macular edema, aflibercept demonstrated statistically significant superiority over laser photocoagulation, with mean BCVA gains of 10.5-13.6 letters compared to -0.5 to 1.2 letters for laser (p<0.0001), and central retinal thickness reductions of 183-195 μm versus 66-73 μm for laser (p<0.0001). At 148 weeks, 35.8-42.9% of aflibercept-treated eyes gained ≥15 letters versus 13.6-18.9% with laser. Aflibercept showed non-inferiority to bevacizumab (mean improvement 15.0 versus 14.0 letters, p=0.37) and better anatomical outcomes than ranibizumab in real-world practice (central subfield thickness reduction -114.4 μm versus -87.8 μm, p<0.01). Quality of life improved meaningfully in both conditions, with NEI VFQ-25 total score gains of +6.11 points in DME and clinically significant improvements across multiple subscales in wet AMD. The safety profile was comparable to other anti-VEGF agents, with serious systemic adverse events similar to ranibizumab (RR 0.99, 95% CI 0.79 to 1.25) and no new safety signals identified in long-term use. The every-8-weeks maintenance dosing regimen after initial loading reduced treatment burden compared to monthly regimens while maintaining efficacy.
Methods
We analyzed 10 sources from an initial pool of 200, using 8 screening criteria. Each paper was reviewed for 8 key aspects that mattered most to the research question.
Paper search
We performed a semantic search across over 138 million academic papers from the Elicit search engine, which includes all of Semantic Scholar and OpenAlex.
We ran this query: “What clinical outcomes support aflibercept treatment of wet AMD and diabetic macular edema?”
The search returned 200 total results from Elicit. We retrieved 200 papers most relevant to the query for screening.
Results
Characteristics of included studies
The systematic review included 10 sources examining aflibercept treatment for wet age-related macular degeneration (AMD) and diabetic macular edema (DME). These comprised randomized controlled trials, observational studies, and systematic reviews.
| Study | Full text retrieved? | Study Type | Sample Size | Patient Population | Baseline Characteristics | Comparator | Follow-up Duration |
|---|---|---|---|---|---|---|---|
| J. Heier et al., 2012 | Yes | RCT | 2419 patients | Wet AMD | Active, subfoveal CNV | Ranibizumab | 52 weeks |
| J. M. Ruiz-Moreno, 2015 | No | Phase III RCT | 461/402 patients | DME | Not specified | Laser | 52 weeks |
| J. Heier et al., 2016 | No | Phase III RCT | 872 eyes | DME | Central-involved DME | Laser | 148 weeks |
| Chirag Jhaveri et al., 2022 | No | RCT | 312 eyes (158 aflibercept) | DME | Moderate vision loss (VA 24-69 letters) | Bevacizumab first | 2 years |
| You-Xin Chen et al., 2020 | No | Phase III RCT | 127 eyes per group | DME | Not specified | Laser | 52 weeks |
| Pierre-Henry Gabrielle et al., 2022 | No | Retrospective | 534 eyes (267 aflibercept) | DME | Moderate (VA ≤68) and mild (VA ≥69) impairment | Ranibizumab | 3 years |
| S. Sarwar et al., 2016 | No | Cochrane review | 2457 participants | Wet AMD | Treatment-naive, active subfoveal CNV | Ranibizumab | 1-2 years |
| J. Carrasco et al., 2021 | Yes | Meta-analysis | 40-2506 patients/eyes | Wet AMD | Mean age 78.62 years, baseline BCVA 57.73 letters | None | 2 years |
| M. Yuzawa et al., 2015 | No | Phase III RCT | 2419 patients (607 aflibercept) | Wet AMD | Treatment-naive, exudative AMD | Ranibizumab | 52 weeks |
| J. Garweg et al., 2019 | No | Phase IV single-arm | 553 patients | DME | Mean BCVA 61.5 letters, mean CRT 464.81 μm | None | 52 weeks |
The studies represented diverse designs, with five focusing on wet AMD and five on DME. Most studies were randomized controlled trials, with comparators including ranibizumab, bevacizumab, and laser photocoagulation. Follow-up durations ranged from 52 weeks to 3 years. Only two studies had full texts available for detailed extraction.
Effects
Visual Acuity Outcomes in Wet AMD
Aflibercept demonstrated substantial and sustained visual acuity improvements in wet AMD across multiple studies. In the VIEW trials comparing aflibercept to ranibizumab, all aflibercept regimens were within 0.5 letters of ranibizumab for mean change in BCVA, demonstrating clinical equivalence. At one year, the mean change in BCVA was similar between aflibercept and ranibizumab groups (mean difference -0.15 ETDRS letters, 95% CI -1.47 to 1.17). This similarity persisted at two years.
| Study | Time Point | Mean BCVA Change | Proportion Gaining ≥15 Letters | Proportion Achieving ≥70 Letters | Statistical Significance |
|---|---|---|---|---|---|
| S. Sarwar et al., 2016 | 1 year | -0.15 letters vs ranibizumab (95% CI -1.47 to 1.17) | ~32% (RR 0.97, 95% CI 0.85 to 1.11) | Not reported | High-quality evidence |
| S. Sarwar et al., 2016 | 2 years | 7.2 letters | ~31% (RR 0.98, 95% CI 0.85 to 1.12) | Not reported | High-quality evidence |
| J. Carrasco et al., 2021 | 2 years | +4.49 letters | Not reported | 47.39% | Not specified |
| M. Yuzawa et al., 2015 | 52 weeks | Similar improvements in NEI VFQ-25 | Not reported | Not reported | Similar to ranibizumab |
The real-world meta-analysis reported a mean BCVA of 62.55 ETDRS letters at 2 years, with 47.39% of patients achieving visual acuity of ≥70 letters.
Visual Acuity Outcomes in Diabetic Macular Edema
Aflibercept demonstrated consistent superiority over laser photocoagulation and non-inferiority to other anti-VEGF agents in DME.
| Study | Time Point | Aflibercept Regimen | Mean BCVA Gain | Proportion Gaining ≥15 Letters | Comparator BCVA Gain | p-value |
|---|---|---|---|---|---|---|
| J. M. Ruiz-Moreno, 2015 | 52 weeks | 2q4 (VISTA) | 12.5 letters | Significant vs laser | 0.2 letters (laser) | p<0.0001 |
| J. M. Ruiz-Moreno, 2015 | 52 weeks | 2q8 (VISTA) | 10.7 letters | Significant vs laser | 0.2 letters (laser) | p<0.0001 |
| J. M. Ruiz-Moreno, 2015 | 52 weeks | 2q4 (VIVID) | 10.5 letters | Significant vs laser | 1.2 letters (laser) | p<0.0001 |
| J. Heier et al., 2016 | 148 weeks | 2q4 (VISTA) | 10.4 letters | 42.9% | 1.4 letters (laser) | p<0.0001 |
| You-Xin Chen et al., 2020 | 52 weeks | 2q4 | +13.6 letters | 43.3% | -0.5 letters (laser) | p<0.0001 |
Long-term outcomes remained robust. At 148 weeks, mean BCVA gains with aflibercept were significantly higher than laser control. The proportion of eyes gaining ≥15 letters at 148 weeks ranged from 35.8% to 42.9% for aflibercept versus 13.6% to 18.9% for laser control.
Anatomical Outcomes
Aflibercept produced significant anatomical improvements in both wet AMD and DME.
| Study | Time Point | Aflibercept CRT Change (2q4) | Aflibercept CRT Change (2q8) | Comparator CRT Change | p-value |
|---|---|---|---|---|---|
| J. M. Ruiz-Moreno, 2015 | 52 weeks (VISTA) | -185.9 μm | -183.1 μm | -73.3 μm (laser) | p<0.0001 |
| J. M. Ruiz-Moreno, 2015 | 52 weeks (VIVID) | -195.0 μm | -192.4 μm | -66.2 μm (laser) | p<0.0001 |
| Pierre-Henry Gabrielle et al., 2022 | 3 years | N/A | -114.4 μm (95% CI -134.4 to -94.3) | -87.8 μm (ranibizumab) | p<0.01 |
The real-world comparison to ranibizumab showed aflibercept had significantly better anatomical outcomes.
Vision-Related Quality of Life
Quality of life assessments demonstrated clinically meaningful improvements with aflibercept treatment.
| Quality of Life Measure | Baseline Score | Week 52 Change | Clinical Significance |
|---|---|---|---|
| NEI VFQ-25 total score | 70.12 | +6.11 (SD 11.46) | Clinically meaningful |
| Near activities subscale | Not specified | +11.37 (SD 18.01) | More pronounced than distance |
| Distance activities subscale | Not specified | +7.33 (SD 17.32) | Clinically meaningful |
| Better-seeing eye | Not specified | +7.74 (SD 13.59) | Comprehensive improvement |
| Worse-seeing eye | Not specified | +5.48 (SD 9.70) | Clinically meaningful |
Safety Profile
The safety profile of aflibercept was generally favorable. Serious systemic adverse events were similar between aflibercept and ranibizumab (RR 0.99, 95% CI 0.79 to 1.25), while the risk of serious ocular adverse events was lower in the aflibercept group (RR 0.62, 95% CI 0.36 to 1.07).
| Study | Serious Ocular Adverse Events | Treatment-Emergent Adverse Events | Deaths | Safety Conclusion |
|---|---|---|---|---|
| J. M. Ruiz-Moreno, 2015 | Similar across groups | Similar ocular and nonocular events | Not reported | Comparable to laser |
| J. Heier et al., 2016 | Cataract: 3.1% | Consistent with known profile | Not reported | Known safety profile maintained |
| J. Garweg et al., 2019 | Endophthalmitis (0.5%) | 53.6% overall; 26.8% ocular | 5 (0.9%), not treatment-related | Consistent with known profile |
Treatment Regimens
Aflibercept was administered at a dose of 2 mg across all studies. Two primary dosing regimens emerged:
- Every 4 weeks regimen (2q4): Monthly injections throughout the treatment period.
- Every 8 weeks regimen (2q8): 3-5 initial monthly loading doses followed by maintenance dosing every 8 weeks.
The eight-week dosing regimen represented reduced treatment requirements compared to monthly dosing regimens.
Comparative Effectiveness
Aflibercept demonstrated non-inferiority to ranibizumab in wet AMD and superiority to laser photocoagulation in DME, while showing comparable effectiveness to bevacizumab.
References
- J. Heier, David M. Brown, V. Chong, et al. (2012)
- Chirag Jhaveri, A. Glassman, F. Ferris, et al. (2022)
- You-Xin Chen, et al. (2020)
- J. M. Ruiz-Moreno (2015)
- J. Heier, et al. (2016)
- Pierre-Henry Gabrielle, et al. (2022)
- S. Sarwar, et al. (2016)
- J. Carrasco, et al. (2021)
- M. Yuzawa, et al. (2015)
- J. Garweg, et al. (2019)