June 2013
Volume 54, Issue 15
Free
ARVO Annual Meeting Abstract  |   June 2013
Health utility in the Multicenter Uveitis Steroid Treatment Trial
Author Affiliations & Notes
  • Elizabeth Sugar
    Biostatistics, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
    Center for Clinical Trials, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
  • Alyce Burke
    Epidemiology, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
    Center for Clinical Trials, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
  • Lea Drye
    Epidemiology, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
    Center for Clinical Trials, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
  • Janet Holbrook
    Epidemiology, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
    Center for Clinical Trials, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
  • John Kempen
    Ocular Inflammation Service, The University of Pennsylvania, Philadelphia, PA
    Center for Clinical Epidemiology and Biostatistics, Department of Biostatistics and Epidemiology, The University of Pennsylvania, Philadelphia, PA
  • Jennifer Thorne
    Epidemiology, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
    Ophthalmology, The Johns Hopkins University School of Medicine, Baltimore, MD
  • Kevin Frick
    Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD
  • Footnotes
    Commercial Relationships Elizabeth Sugar, None; Alyce Burke, None; Lea Drye, None; Janet Holbrook, None; John Kempen, Alcon (C), Allergan (C), Clearside (C), Can-Fite (C), Lux Biosciences (C), Xoma (C), NEI/NIH (F), FDA (F), Research to Prevent Blindness (F), Mackall Foundation (F), EyeGate Pharma (F), University of Pennsylvania (E); Jennifer Thorne, Allergan (C), XOMA (C), Santen (C); Kevin Frick, Center for Applied Value Analysis (C), National Association for Eye and Vision Research (C)
  • Footnotes
    Support None
Investigative Ophthalmology & Visual Science June 2013, Vol.54, 5934. doi:
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      Elizabeth Sugar, Alyce Burke, Lea Drye, Janet Holbrook, John Kempen, Jennifer Thorne, Kevin Frick, ; Health utility in the Multicenter Uveitis Steroid Treatment Trial. Invest. Ophthalmol. Vis. Sci. 2013;54(15):5934.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose: Cost-effectiveness is usually evaluated by comparing treatment cost with a corresponding gain in a patient-centered outcome like health utility. We estimated change in a measure of health utility (EQ-5D) in participants randomized to receive treatment with fluocinolone acetonide implant versus systemic therapy for active or recently active non-infectious intermediate, posterior, or panuveitis as a first step.

Methods: Data from the first 3 years following randomization for individuals enrolled in the Multicenter Uveitis Steroid Treatment (MUST) Trial were evaluated. The follow-up time period was based on the expected lifetime of the implant. The EuroQol EQ-5D questionnaire was scored at 6-month intervals. Linear regression models were fit using generalized estimating equations to account for repeated measurements while adjusting for disease stratum (intermediate v. posterior/panuveitis). Contrasts were used to construct the quality adjusted life years (QALYs), a weighted sum of the EQ-5D at all of the visits. The weighting discounted years 2 and 3 by dividing by 1.03 and 1.032, respectively. Separate analyses were performed for participants with unilateral and bilateral disease due to the large expected discrepancy in upfront cost.

Results: 255 individuals were randomized (31 unilateral and 224 bilateral). The differences in QALYs between the treatment groups were 0.057 (95% CI: -0.035 to 0.148, p = 0.12) for those with bilateral disease and 0.128 (95% CI: -0.033 to 0.290, p = 0.22) for those with unilateral disease, favoring the implant. For unilateral uveitis, there was a slight increase in EQ-5D from baseline (Range: 0.060 to 0.096) among implant patients at each visit that was significant for all visits except the 3 year visit. For bilateral uveitis, there was a slight but not statistically significant decline in EQ-5D from baseline (Range: -0.200 to -0.055) among systemic patients at each visit. There were no detectable trends in the change from baseline among bilateral implant or unilateral systemic participants.

Conclusions: The differences in QALYs by treatment assignment were not statistically significant for either disease cohort. However, the magnitude of the observed values is large enough that the potential for the implant treatment to be cost-effective remains, depending upon the total cost, despite its larger upfront cost.

Keywords: 746 uveitis-clinical/animal model • 669 quality of life • 466 clinical (human) or epidemiologic studies: treatment/prevention assessment/controlled clinical trials  
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