Estimated Utility of the Short-term Assessment of Glaucoma Progression Model in Clinical Practice
| Autor: | Luke J. Saunders, Christopher Bowd, Akram Belghith, Eunice Williams-Steppe, Felipe A. Medeiros, Huiyuan Hou, Keri Dirkes, Robert N. Weinreb, James A. Proudfoot, Tess Acera, Sasan Moghimi, Linda M. Zangwill |
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| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Male
Retinal Ganglion Cells medicine.medical_specialty genetic structures Glaucoma 01 natural sciences Cohort Studies 03 medical and health sciences Nerve Fibers 0302 clinical medicine Ophthalmology medicine Humans 0101 mathematics Stage (cooking) Intraocular Pressure Aged Original Investigation business.industry Clinical study design 010102 general mathematics medicine.disease Clinical trial Clinical Practice Sample size determination Disease Progression 030221 ophthalmology & optometry Optic nerve Visual Field Tests Female sense organs business Glaucoma Open-Angle Tomography Optical Coherence Follow-Up Studies Cohort study |
| Zdroj: | JAMA Ophthalmol |
| Popis: | Importance Clinical trials of glaucoma therapies focused on protecting the optic nerve have required large sample sizes and lengthy follow-up to detect clinically relevant change due to its slow rate of progression. Whether shorter trials may be possible with more frequent testing and use of rate of change as the end point warrants further investigation. Objective To describe the design for the Short-term Assessment of Glaucoma Progression (STAGE) model and provide guidance on sample size and power calculations for shorter clinical trials. Design, Setting, and Participants A cohort study of patients with mild, moderate, or advanced open-angle glaucoma recruited from the Diagnostic Innovations in Glaucoma Study at the University of California, San Diego. Enrollment began in May 2012 with follow-up for every 3 months for 2 years after baseline examination. Follow-up was concluded in September 2016. Data were analyzed from July 2019 to January 2021. Visual fields (VF) and optic coherence tomography (OCT) scans were obtained at baseline and for 2 years with visits every 3 months. Exposures Glaucoma was defined as glaucomatous appearing optic discs classified by disc photographs in at least 1 eye and/or repeatable VF damage at baseline. Main Outcomes and Measures Longitudinal rates of change in retinal nerve fiber layer (RNFL) thickness and VF mean deviation (MD) are estimated in study designs of varying length and observation frequency. Power calculations as functions of study length, observation frequency, and sample size were performed. Results In a total referred sample of 97 patients with mild, moderate, or advanced glaucoma (mean [SD] age, 69 [11.4] years; 50 [51.5%] were female; 19 [19.6%]), over the 2-year follow-up, the mean VF 24-2 MD slope was −0.32 dB/y (95% CI, −0.43 to −0.21 dB/y) and the mean RNFL thickness slope was −0.54 μm/y (95% CI, −0.75 to −0.32 μm/y). Sufficient power (80%) to detect similar group differences in the rate of change in both outcomes was attained with total follow-up between 18 months and 2 years and fewer than 300 total participants. Conclusions and Relevance In this cohort study, results from the STAGE model with reduction of the rate of progression as the end point, frequent testing, and a moderate effect size, suggest that clinical trials to test efficacy of glaucoma therapy can be completed within 18 months of follow-up and with fewer than 300 participants. |
| Databáze: | OpenAIRE |
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