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Elizabeth Sugar, Alyce Burke, Lea Drye, Janet Holbrook, John Kempen, Jennifer Thorne, Kevin Frick, Multicenter Uveitis Steroid Treatment (MUST) Trial Research Group; Health utility in the Multicenter Uveitis Steroid Treatment Trial. Invest. Ophthalmol. Vis. Sci. 2013;54(15):5934.
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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.
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.
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.
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.
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