scholarly journals Endpoints selection in registration clinical trials and the needs of real-world clinical practice with the example of anti-VEGF therapy in neovascular age-related macular degeneration

2021 ◽  
Vol 14 (1) ◽  
pp. 25-33
Author(s):  
Sergey Yu. Astakhov ◽  
Svyatoslav L. Plavinskii
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Macular Degeneration ◽  
Real World ◽  
New Technologies ◽  
Age Related Macular Degeneration ◽  
Type I ◽  
Regulatory Requirements ◽  
Anti Vegf ◽  
Age Related

The issues of endpoints selection for regulatory requirements and real-world clinical practice using the example of anti-VEGF therapy in neovascular age-related macular degeneration (nAMD) are discussed in the article. New technologies (optical coherent tomography) introduction are shown to change clinical practice but not regulatory requirements on the endpoints. In the same time for regulatory purpose clinical trials design is changed from superiority to non-inferiority. The changes in the approach to primary endpoint selection are not anticipated due to regulators conservatism but there is a requirement to the comparison with best treatment alternative (i.e. same class comparator in case of anti-VEGF therapy) due to ethical reasons. To satisfy real-world clinicians need, the secondary endpoints are analyzed, but multiple testing problem appears. Statistical methods developed in recent years allow using specified comparison to be made without inflating Type I error. HAWK and HARRIER clinical trials demonstrated an example how superiority of brolucizumab over aflibercet on anatomical endpoints was reliably found.

scholarly journals Anti-VEGF therapy of neovascular age-related macular degeneration: from randomized trials to routine clinical practice

2019 ◽  
Vol 12 (2) ◽  
pp. 97-105 ◽  
Author(s):  
R. R. Fayzrakhmanov
Keyword(s):  
Clinical Practice ◽  
Macular Degeneration ◽  
Randomized Clinical Trials ◽  
High Reliability ◽  
Functional Results ◽  
Routine Clinical Practice ◽  
Age Related Macular Degeneration ◽  
Treatment Protocols ◽  
Anti Vegf ◽  
Age Related

Antivasoproliferative therapy is a revolutionary trend in the treatment of neovascular age-related macular degeneration (nAMD), as it is aimed at blocking growth factors of the newly formed vessels. Currently, two anti-VEGF drugs are registered for ophthalmological use, and the search for new molecules is only gaining momentum. Studying new approaches to treatment and developing innovative drugs, modern medicine relies on the data from international randomized clinical trials (RCT). The fact that this direction attracts much scientific interest is explained by high reliability of the data obtained with RCTs. However, when analyzing the effectiveness of anti-VEGF therapy in patients with nAMD in routine clinical practice, many ophthalmologists are confronted with discrepancies between the expected morphological and functional results as predicted by RCTs and those obtained in reality. It is thus important to bear in mind that RCTs simply determine how the dynamics of morphofunctional parameters should look like, whilst the ideal result is only achievable through correct implementation of therapeutic strategy in real clinical practice. The results obtained in the practice of any specialist wholly depend on how carefully the prescribed treatment protocols are followed by doctors and patients, how burdensome the treatment is, as well as on the potential of the medication. This literary review offers a comparative analysis of the results achieved by using anti-VEGF drugs (ranibizumab and aflibercept) obtained in key RCTs and in routine clinical treatment of nAMD patients.

scholarly journals Treatment patterns in patients with age-related macular degeneration and diabetic macular edema: A real-world claims analysis in Dubai

PLoS ONE ◽  
2021 ◽  
Vol 16 (7) ◽  
pp. e0254569
Author(s):  
Igor Kozak ◽  
Avinash Gurbaxani ◽  
Ammar Safar ◽  
Prasan Rao ◽  
Amal Masalmeh ◽  
...  
Keyword(s):  
Macular Degeneration ◽  
Macular Edema ◽  
Diabetic Macular Edema ◽  
Real World ◽  
Treatment Patterns ◽  
Age Related Macular Degeneration ◽  
Treatment Frequency ◽  
Anti Vegf ◽  
Age Related

Objectives To characterize the pattern of approved anti-vascular endothelial growth factor (VEGF) treatments among patients with neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME) in the United Arab Emirates (UAE). Method This was a retrospective, nonrandomized, observational cohort analysis of the Dubai Real-world Claims Database with a 360-day follow-up period. Adult patients diagnosed with nAMD or DME treated with ranibizumab or aflibercept for the first time were included. The primary objective was to evaluate anti-VEGF treatment patterns with respect to the proportion of patients receiving ranibizumab and aflibercept for nAMD and DME separately. Results Of the 451 patients included in the final study cohort, 83.6% and 16.4% had a diagnosis of DME (ranibizumab: 48.5%; aflibercept: 51.5%) and nAMD (ranibizumab: 40.5%; aflibercept: 59.5%), respectively, at baseline. Treatment frequency of ranibizumab/aflibercept was similar for nAMD (mean: 2.4/2.9 injections; p = 0.2389) with fewer injections in the ranibizumab cohort for DME (mean: 1.9/2.5 injections; p = 0.0002). Most patients received ≤3 anti-VEGF injections during the 360-day follow-up period. The time between consecutive treatments was large (nAMD: 73.6 days/10.5 weeks; DME: 80.5 days/11.5 weeks). Approximately 10%–13.5% of patients switched their anti-VEGF therapy. Most patients (83.8%) had a diabetes diagnosis during the follow-up period. Conclusions This real-world study provides an initial understanding of anti-VEGF treatment patterns in patients with nAMD and DME in the UAE. Treatment frequency of the 2 anti-VEGF agents assessed was similar in both patient populations. Both treatments were infrequently administered with large dosing intervals.

scholarly journals Choroidal neovascularization activity and structure by optical coherence tomography angiography in age related macular degeneration

2021 ◽  
Vol 6 (6-1) ◽  
pp. 12-18
Author(s):  
M. A. Kovalevskaya ◽  
O. A. Pererva
Keyword(s):  
Macular Degeneration ◽  
Choroidal Neovascularization ◽  
Vascular System ◽  
Developed Countries ◽  
Age Related Macular Degeneration ◽  
Control Group ◽  
Type I ◽  
Neovascular Amd ◽  
Anti Vegf ◽  
Age Related

Background. In economically developed countries, age-related macular degeneration (AMD) is the leading cause of visual disability among the population of the older age group. The main criterion for the anti-VEGF treatment of neovascular AMD is the activity of choroidal neovascularization (CNV), which is determined by its confi guration. The search for optimal criteria for quantifying the state of the macular region in order to decide on the appointment of anti-VEGF therapy continues.Aim: improving the effi ciency of diagnosis and treatment of AMD based on the assessment of the configuration of vascular system on the “Key to Diagnosis II” platform.Material and methods. The study included 341 patients: 64 % (218 patients, 267 eyes) with non-neovascular AMD, 36 % (123 patients, 174 eyes) – with neovascular AMD. 56 patients (58 eyes) had active type I CNV. Group 1A – active CNV before treatment (9 patients, 9 eyes), group 1B – non-active CNV after treatment with antiVEGF (9 patients, 9 eyes); control group – 10 patients (10 eyes) without AMD. Analysis of OCT-angio images of choriocapillaries included the isolation of CNV, its area, fractal dimension (Df) and the complexity of the vascular system (CVS) counting.Results. Group 1A: Df – 1.5871 ± 0.05, CVS – 2.29 ± 0.29, area – 11734 ± 4866; group 1B: Df – 1.6462 ± 0.08, CVS – 1.65 ± 0.18, area – 6797 ± 3818; control: Df – 1.9167 ± 0.06, CVS – 1, area – 0. Significant differences were found for CVS (p = 0.0003). Df correlates with the CNV area (p = 0.7) and is probably an unreliable parameter due to incomplete visualization of active CNV.Conclusions. CVS is a quantitative biomarker for determining the activity of type 1 CNV in patients with AMD and can serve as a parameter for convolutional neural networks training for automated analysis of OCT angiography images based on the “Key to Diagnosis II” platform

Visual Acuity Outcomes in Patients Receiving Frequent Treatment of Neovascular Age-Related Macular Degeneration in Clinical Practice

2020 ◽  
pp. 247412642096089
Author(s):  
Andrew A. Moshfeghi ◽  
John D. Pitcher ◽  
Genevieve Lucas ◽  
Nick Boucher ◽  
Namrata Saroj
Keyword(s):  
Visual Acuity ◽  
Clinical Practice ◽  
Macular Degeneration ◽  
Age Related Macular Degeneration ◽  
First Year ◽  
Treatment Frequency ◽  
Anti Vegf ◽  
Age Related ◽  
Endothelial Growth Factor ◽  
Change In Mean

Purpose: This work evaluates dosing frequency with intravitreal antivascular endothelial growth factor (anti-VEGF) agents over 2 years and visual acuity (VA) outcomes in neovascular age-related macular degeneration (nAMD). Methods: This retrospective analysis assesses electronic medical record data (Vestrum Health treatment and outcomes database) of newly diagnosed nAMD in patients who were initiated on intravitreal anti-VEGF treatment at US clinical sites. Eyes were divided into 2 injection frequency subcohorts (≤ 6 or > 6 injections/y); treatment frequency and change in mean VA (Early Treatment Diabetic Retinopathy Study letters) were evaluated. Results: Overall, 8127 of 213 824 eyes met inclusion criteria in year 1 and 4968 in year 2. During year 1, 77% of the eyes received more than 6 injections (n = 6287), the majority of which received injections at the same frequency during year 2. Mean VA gain from baseline at year 1 was lower in the ≤ 6 than > 6 injections/y subcohort (2.2 vs 6.5, P < .001). Decrease in mean VA from the end of year 1 to year 2 was significantly greater for eyes administered 6 or fewer injections in year 2 than those that received more frequent injections, irrespective of the frequency of injections in the first year. Conclusions: In routine clinical practice, most eyes with nAMD that completed at least 1 year of follow-up were treated with more than 6 injections of anti-VEGF agents during the first year of treatment, resulting in better VA gains than eyes treated less frequently during the same period.

scholarly journals Comparing Effectiveness of Three Different Anti-VEGF Treatment Regimens for Neovascular Age-Related Macular Degeneration: Two Years’ Real-World Clinical Outcomes

2021 ◽  
Vol Volume 15 ◽  
pp. 1703-1713
Author(s):  
Faye Horner ◽  
Peck Lin Lip ◽  
Bashar R Mohammed ◽  
William Fusi-Rubiano ◽  
Eesha Gokhale ◽  
...  
Keyword(s):  
Macular Degeneration ◽  
Clinical Outcomes ◽  
Real World ◽  
Age Related Macular Degeneration ◽  
Treatment Regimens ◽  
Anti Vegf ◽  
Age Related
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