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      Demographics of patients receiving Intravitreal anti-VEGF treatment in real-world practice: healthcare research data versus randomized controlled trials

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          Abstract

          Background

          While randomized controlled trials (RCTs) are based on strict inclusion/exclusion criteria, non-interventional studies (NISs) might provide additional information to guide management in patients more representative to the real-world setting. The aim of this study was to compare baseline characteristics of patients receiving intravitreal treatment in the NIS OCEAN with those from published RCTs.

          Methods

          The ongoing OCEAN study enrolled patients treated with ranibizumab for neovascular age-related macular degeneration (nAMD), diabetic macular oedema (DME) or branch/central retinal vein occlusion (B/CRVO). Baseline patient characteristics were compared by indication within the OCEAN cohort. Furthermore, the characteristics were set in reference to those of published RCTs in the same indications. Confidence intervals (CIs) were calculated and assessed for statistically significant differences as indicated by non-overlapping CIs.

          Results

          Patient characteristics in the NIS OCEAN were evaluated for 3,614 patients with nAMD, 1,211 with DME, 204 with BRVO and 121 with CRVO. Between these groups, significant differences in mean age, gender distributions, and mean baseline VA were seen, reflecting known differences between the indications.

          Compared to the patient characteristics of published RCTs (trials selected by literature search: nAMD: 13 RCTs, DME: 9, RVO: 5), the OCEAN patients’ mean age was significantly higher in every indication. The gender distributions across the trials were comparable, with only few differences between OCEAN and the RCTs. Regarding the mean baseline VA, notable differences were found in nAMD and in DME, with VA significantly higher in some RCTs and lower in others.

          Conclusions

          The described differences underline the complementarity of NISs and RCTs. OCEAN covers a broader spectrum and more variability of patients than do RCTs. As baseline values may have impact on the treatment response (ceiling effect), there is an ongoing need for research in all patient subgroups. Country-specific assessments of patient populations can better reflect the real-world situation. NISs can deliver insights that RCTs may not, as NISs can include non-typical patients, patients with comorbidities, a broader age spectrum and patients of various disease stages.

          Trial registration

          The NIS OCEAN was registered on www.clinicaltrials.gov (identifier: NCT02194803).

          Electronic supplementary material

          The online version of this article (doi:10.1186/s12886-017-0401-y) contains supplementary material, which is available to authorized users.

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          Most cited references34

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          Randomized trial evaluating ranibizumab plus prompt or deferred laser or triamcinolone plus prompt laser for diabetic macular edema.

          Michael Elman, Lloyd Paul Aiello, Roy Beck … (2010)
          Evaluate intravitreal 0.5 mg ranibizumab or 4 mg triamcinolone combined with focal/grid laser compared with focal/grid laser alone for treatment of diabetic macular edema (DME). Multicenter, randomized clinical trial. A total of 854 study eyes of 691 participants with visual acuity (approximate Snellen equivalent) of 20/32 to 20/320 and DME involving the fovea. Eyes were randomized to sham injection + prompt laser (n=293), 0.5 mg ranibizumab + prompt laser (n=187), 0.5 mg ranibizumab + deferred (> or =24 weeks) laser (n=188), or 4 mg triamcinolone + prompt laser (n=186). Retreatment followed an algorithm facilitated by a web-based, real-time data-entry system. Best-corrected visual acuity and safety at 1 year. The 1-year mean change (+/-standard deviation) in the visual acuity letter score from baseline was significantly greater in the ranibizumab + prompt laser group (+9+/-11, P<0.001) and ranibizumab + deferred laser group (+9+/-12, P<0.001) but not in the triamcinolone + prompt laser group (+4+/-13, P=0.31) compared with the sham + prompt laser group (+3+/-13). Reduction in mean central subfield thickness in the triamcinolone + prompt laser group was similar to both ranibizumab groups and greater than in the sham + prompt laser group. In the subset of pseudophakic eyes at baseline (n=273), visual acuity improvement in the triamcinolone + prompt laser group appeared comparable to that in the ranibizumab groups. No systemic events attributable to study treatment were apparent. Three eyes (0.8%) had injection-related endophthalmitis in the ranibizumab groups, whereas elevated intraocular pressure and cataract surgery were more frequent in the triamcinolone + prompt laser group. Two-year visual acuity outcomes were similar to 1-year outcomes. Intravitreal ranibizumab with prompt or deferred laser is more effective through at least 1 year compared with prompt laser alone for the treatment of DME involving the central macula. Ranibizumab as applied in this study, although uncommonly associated with endophthalmitis, should be considered for patients with DME and characteristics similar to those in this clinical trial. In pseudophakic eyes, intravitreal triamcinolone + prompt laser seems more effective than laser alone but frequently increases the risk of intraocular pressure elevation. Copyright 2010 American Academy of Ophthalmology. Published by Elsevier Inc. All rights reserved.
          Article 0 comments Cited 424 times – based on 0 reviews     Review now
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            Intravitreal aflibercept injection for neovascular age-related macular degeneration: ninety-six-week results of the VIEW studies.

            Ursula Schmidt-Erfurth, Peter K Kaiser, Jean-François Korobelnik … (2014)
            To determine efficacy and safety of intravitreal aflibercept in patients with neovascular age-related macular degeneration (AMD) during a second year of variable dosing after a first-year fixed-dosing period. Two randomized, double-masked, active-controlled, phase 3 trials. Two thousand four hundred fifty-seven patients with neovascular AMD. From baseline to week 52, patients received 0.5 mg intravitreal ranibizumab every 4 weeks (Rq4), 2 mg aflibercept every 4 weeks (2q4), 0.5 mg aflibercept every 4 weeks (0.5q4), or 2 mg aflibercept every 8 weeks (2q8) after 3 monthly injections. During weeks 52 through 96, patients received their original dosing assignment using an as-needed regimen with defined retreatment criteria and mandatory dosing at least every 12 weeks. Proportion of eyes at week 96 that maintained best-corrected visual acuity (BCVA; lost <15 letters from baseline); change from baseline in BCVA. Proportions of eyes maintaining BCVA across treatments were 94.4% to 96.1% at week 52 and 91.5% to 92.4% at week 96. Mean BCVA gains were 8.3 to 9.3 letters at week 52 and 6.6 to 7.9 letters at week 96. Proportions of eyes without retinal fluid decreased from week 52 (60.3% to 72.4%) to week 96 (44.6% to 54.4%), and more 2q4 eyes were without fluid at weeks 52 and 96 than Rq4 eyes (difference of 10.4% [95% confidence interval {CI}, 4.9-15.9] and 9.0% [95% CI, 3.0-15.1]). Patients received on average 16.5, 16.0, 16.2, and 11.2 injections over 96 weeks and 4.7, 4.1, 4.6, and 4.2 injections during weeks 52 through 96 in the Rq4, 2q4, 0.5q4, and 2q8 groups, respectively. The number of injections during weeks 52 through 96 was lower in the 2q4 and 2q8 groups versus the Rq4 group (differences of -0.64 [95% CI, -0.89 to -0.40] and -0.55 [95% CI, -0.79 to -0.30]; P < 0.0001, post hoc analysis). Incidences of Antiplatelet Trialists' Collaboration-defined arterial thromboembolic events were similar across groups (2.4% to 3.8%) from baseline to week 96. All aflibercept and ranibizumab groups were equally effective in improving BCVA and preventing BCVA loss at 96 weeks. The 2q8 aflibercept group was similar to ranibizumab in visual acuity outcomes during 96 weeks, but with an average of 5 fewer injections. Small losses at 96 weeks in the visual and anatomic gains seen at 52 weeks in all arms were in the range of losses commonly observed with variable dosing. Copyright © 2014 American Academy of Ophthalmology. Published by Elsevier Inc. All rights reserved.
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              Randomized, double-masked, sham-controlled trial of ranibizumab for neovascular age-related macular degeneration: PIER Study year 1.

              Carl Regillo, David M Brown, Prema Abraham … (2008)
              To evaluate the efficacy and safety of ranibizumab administered monthly for three months and then quarterly in patients with subfoveal choroidal neovascularization (CNV) secondary to age-related macular degeneration (AMD). Phase IIIb, multicenter, randomized, double-masked, sham injection-controlled trial in patients with predominantly or minimally classic or occult with no classic CNV lesions. Patients were randomized 1:1:1 to 0.3 mg ranibizumab (n = 60), 0.5 mg ranibizumab (n = 61), or sham (n = 63) treatment groups. The primary efficacy endpoint was mean change from baseline visual acuity (VA) at month 12. Mean changes from baseline VA at 12 months were -16.3, -1.6, and -0.2 letters for the sham, 0.3 mg, and 0.5 mg groups, respectively (P < or = .0001, each ranibizumab dose vs sham). Ranibizumab arrested CNV growth and reduced leakage from CNV. However, the treatment effect declined in the ranibizumab groups during quarterly dosing (e.g., at three months the mean changes from baseline VA had been gains of 2.9 and 4.3 letters for the 0.3 mg and 0.5 mg doses, respectively). Results of subgroups analyses of mean change from baseline VA at 12 months by baseline age, VA, and lesion characteristics were consistent with the overall results. Few serious ocular or nonocular adverse events occurred in any group. Ranibizumab administered monthly for three months and then quarterly provided significant VA benefit to patients with AMD-related subfoveal CNV and was well tolerated. The incidence of serious ocular or nonocular adverse events was low.
              Article 0 comments Cited 205 times – based on 0 reviews     Review now
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                Author and article information

                Contributors
                F. Ziemssen:
                ORCID: http://orcid.org/0000-0002-3873-0581
                +49 7071 29 84761 , focke.ziemssen@med.uni-tuebingen.de
                N. Feltgen: nicolas.feltgen@med.uni-goettingen.de
                FG. Holz: Frank.Holz@ukb.uni-bonn.de
                R. Guthoff: rainer.guthoff@med.uni-duesseldorf.de
                A. Ringwald: andreas.ringwald@klinikumdo.de
                T. Bertelmann: thomas.bertelmann@novartis.com
                A. Wiedon: annette.wiedon@novartis.com
                C. Korb: christina.korb@unimedizin-mainz.de
                Journal
                Journal ID (nlm-ta): BMC Ophthalmol
                Journal ID (iso-abbrev): BMC Ophthalmol
                Title: BMC Ophthalmology
                Publisher: BioMed Central (London )
                ISSN (Electronic): 1471-2415
                Publication date (Electronic): 19 January 2017
                Publication date PMC-release: 19 January 2017
                Publication date Collection: 2017
                Volume: 17
                Electronic Location Identifier: 7
                Affiliations
                [1 ]ISNI 0000 0001 2190 1447, GRID grid.10392.39, Centre for Ophthalmology, , Eberhard-Karls-University Tuebingen, ; Schleichstr. 12, Tuebingen, 72076 Germany
                [2 ]University Eye Hospital Goettingen, Goettingen, Germany
                [3 ]ISNI 0000 0001 2240 3300, GRID grid.10388.32, Department of Ophthalmology, , University of Bonn, ; Bonn, Germany
                [4 ]ISNI 0000 0001 2176 9917, GRID grid.411327.2, University Eye Hospital, , Heinrich-Heine-University Duesseldorf, ; Duesseldorf, Germany
                [5 ]ISNI 0000 0001 2172 9288, GRID grid.5949.1, Klinikum Dortmund, , University of Muenster, ; Muenster, Germany
                [6 ]ISNI 0000 0001 2364 4210, GRID grid.7450.6, Department of Ophthalmology, and Novartis Pharma GmbH, , Georg-August-University Goettingen, ; Nuremberg, Germany
                [7 ]Novartis Pharma GmbH, Nuremberg, Germany
                [8 ]GRID grid.410607.4, Department of Ophthalmology, , University Medical Center Mainz, ; Mainz, Germany
                Author information
                http://orcid.org/0000-0002-3873-0581
                Article
                Publisher ID: 401
                DOI: 10.1186/s12886-017-0401-y
                PMC ID: 5244516
                PubMed ID: 28103831
                SO-VID: 2ab75fbe-7504-4064-a11d-22c2b1713c02
                Copyright © © The Author(s). 2017
                License:

                Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License ( http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver ( http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

                History
                Date received : 9 July 2016
                Date accepted : 10 January 2017
                Funding
                Funded by: FundRef http://dx.doi.org/10.13039/100008792, Novartis Pharma;
                Categories
                Subject: Research Article
                Custom metadata
                issue-copyright-statement © The Author(s) 2017

                ScienceOpen disciplines: Ophthalmology & Optometry
                Keywords: namd,dme,rvo,non-interventional study,demographic characteristics,anti-vegf,epidemiology,ceiling effect
                Data availability:
                ScienceOpen disciplines: Ophthalmology & Optometry
                Keywords: namd, dme, rvo, non-interventional study, demographic characteristics, anti-vegf, epidemiology, ceiling effect

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