Utility Assessment of Moderate to Severe Thyroid Eye Disease Health States

Abstract

ImportanceThyroid eye disease (TED) results in varying degrees of proptosis and diplopia negatively affecting quality of life (QoL), producing possibly substantial visual changes, disfigurement, and disability.ObjectiveTo determine the association of varying TED severities with QoL in a non-TED population by assessing health state utility scores.Design, Setting, and ParticipantsThis qualitative study, conducted from April 20, 2020, to April 29, 2021, assessed health states for active, moderate-severe TED, and values were elicited using time trade-off methods. Six health states of varying severity were determined from 2 placebo-controlled clinical trials (171 patients with TED and clinical activity score ≥4, ±diplopia/proptosis) and refined using interviews with US patients with TED (n = 6). Each health state description was validated by interviews with additional TED patient advocates (n = 3) and physician experts (n = 3). Health state descriptions and a QOL questionnaire were piloted and administered to a general population. Visual analog scales (VASs) were also administered to detect concurrence of the findings.Main Outcomes and MeasuresTED health state utility scores and whether they differ from one another were assessed using Shapiro-Wilk, Kruskal-Wallis, pairwise Wilcoxon rank sum, and paired t tests.ResultsA total of 111 participants completed time trade-off interviews. The mean (SD) utility value was 0.44 (0.34). The lowest (worse) mean utility value was observed in the most severe disease state (constant diplopia/large proptosis) with 0.30 (95% CI, 0.24-0.36), followed by constant diplopia/small proptosis (0.34; 95% CI, 0.29-0.40), intermittent or inconstant diplopia/large proptosis (0.43; 95% CI, 0.36-0.49), no diplopia/large proptosis (0.46; 95% CI, 0.40-0.52), and intermittent or inconstant diplopia/small proptosis (0.52; 95% CI, 0.45-0.58). The highest (best) mean value, 0.60 (95% CI, 0.54-0.67), was observed for the least severe disease state (no diplopia/small proptosis).Conclusions and RelevanceThese findings suggest that patients with active, moderate-severe TED may have substantial disutility, with increasing severity of proptosis/diplopia more likely to have detrimental associations with QoL. These health state scores may provide a baseline for determining QoL improvement in these TED health states (utility gains) treated with new therapies.

How patients experience thyroid eye disease

“…TED is associated with excess co-morbidity and mortality, as well as early retirement and loss of productivity (10)(11)(12)(13)(14). Decreased quality of life (QoL) results from physical manifestations and their negative impact on mental health (15,16). The wide-ranging emotional toll of TED frequently includes anxiety and depression, both of which can persist in chronic disease (3,15,17,18).…”

“…Relatively little recent evidence informs the current experiences, perspectives, and QoL of individuals living with TED (9,16,20,22). An overarching question to be considered was the degree to which disease-related reductions in QoL widely attributed to early, active TED might become less problematic after the transition of patients to chronic, non-progressive disease.…”

Efficacy and Safety of Teprotumumab in Patients With Thyroid Eye Disease of Long Duration and Low Disease Activity

“…Thyroid eye disease (TED), also known as Graves orbitopathy, is an autoimmune disease manifesting retro-orbital fat and muscle inflammation and tissue expansion with progressive signs and symptoms of strabismus, diplopia, and proptosis, resulting in significant disability and morbidity ( 1 ). Historically, it has been described as a biphasic disease with initial acute or dynamic inflammation of orbital tissues (pain, redness, edema) that crescendos followed by a diminution and stabilization of inflammation with continued tissue expansion and fibrosis often referred to as the chronic phase.…”

Insight Into Quality of Life in Patients With Moderate to Severe Thyroid Eye Disease

“…Determining, therefore, utility values for uncommon eye diseases, which have a prevalence of something like 0.1% in a general population, is a worthy goal. In this issue of JAMA Ophthalmology , Smith et al report on their endeavors to measure utility in thyroid eye disease (TED), a condition that involves chronic mild to severe symptoms of diplopia and proptosis.…”