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Discontinuation and loss to follow-up rates in clinical trials of intravitreal anti-vascular endothelial growth factor injections

  • Retinal Disorders
  • Published:
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Abstract

Purpose

Clinical trials are often designed to include homogenous, highly specific patient populations with many resources to reduce patient dropout. Results may not translate to real-world settings. We evaluated discontinuation and loss to follow-up (LTFU) rates in clinical trials of anti-vascular endothelial growth factor (anti-VEGF) injections for diabetic macular edema (DME), age-related macular degeneration (AMD), and retinal vein occlusion (RVO).

Methods

Retrospective meta-epidemiological study. The authors queried ClinicalTrials.gov for all completed trials of anti-VEGF injections for DME, AMD, or RVO. Of 658 trials identified, 582 were excluded for being non-interventional, <100 patients, terminating early, or missing study results. The remaining 76 trials of 27,823 patients were analyzed for discontinuation and LTFU rates.

Results

Mean discontinuation rate was 12.44% (SD 8.12%, range 0–54.12%), with higher rates among control (18.87%) than treatment arms (10.78%, p = .006). Mean LTFU rate was 1.84% (SD 1.78%, range 0–7.76%), with no differences by disease, treatment type, or treatment frequency.

Conclusion

Discontinuation rates of major intravitreal anti-VEGF clinical trials were highly variable, suggesting even trials struggle with overall patient retention. Though trial LTFU rates were low, real-world outcomes may differ due to higher reported LTFU rates, which should be considered when extrapolating trial results to clinical practice.

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Availability of data and materials

Data obtained from publicly available clinical trial records from ClinicalTrials.gov.

Code availability

Available upon request.

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Authors and Affiliations

  1. Department of Ophthalmology, Byers Eye Institute, Stanford School of Medicine, Palo Alto, CA, USA

    Tatiana R. Rosenblatt, Nadim Rayess, Ahmad Al-Moujahed & Prithvi Mruthyunjaya

  2. California Retina Vitreous Associates, Mountain View, CA, USA

    Rahul N. Khurana

Authors
  1. Tatiana R. Rosenblatt
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  2. Nadim Rayess
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  3. Ahmad Al-Moujahed
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  4. Rahul N. Khurana
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  5. Prithvi Mruthyunjaya
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All the authors contributed sufficiently to this manuscript to qualify as authors. Specific author contributions are available upon request.

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Correspondence to Prithvi Mruthyunjaya.

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The Stanford University Institutional Review Board determined that this study was exempt from requiring IRB approval as it does not meet the definition of human subject research.

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Rosenblatt, T.R., Rayess, N., Al-Moujahed, A. et al. Discontinuation and loss to follow-up rates in clinical trials of intravitreal anti-vascular endothelial growth factor injections. Graefes Arch Clin Exp Ophthalmol 260, 93–100 (2022). https://doi.org/10.1007/s00417-021-05246-5

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  • DOI: https://doi.org/10.1007/s00417-021-05246-5

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