Presentation (CTI)

OBJECTIVES: Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune disease associated with severe relapses and long-term disability [1]. Long-term health-related quality of life (HRQoL) data, particularly EQ-5D utility values, remain limited [2-3]. The study aims to evaluate utility values over time with Staralizumab treatment using data from the SAkuraMoon trial [4].
METHODS: Pooled patient-level data from the SAkuraMoon trial were mapped to three EQ-5D-3L value sets: UK 2023 [5], Canada 2012 [6], and Korea 2009 [7]. Two linear mixed-effects models were used to estimate utility (1) Visit-based model: Utility was modeled as a function of study visit, with baseline utility included as a covariate to evaluate its impact on follow-up scores. (2) Expanded Disability Status Scale (EDSS) utility model: A repeated measures model was used, adjusting for time-varying EDSS scores categorized into (≤2.0, 2.5-3.5, 4.0-4.5, 5.0-6.0, >6.0), based on study eligibility criteria and the limited data available on higher EDSS scores.
RESULTS: Mean descriptive utility scores at the end of the study were: Canada 0.766 (95% CI: 0.758-0.774), Korea 0.818 (95% CI: 0.812-0.825), and UK 0.787 (95% CI: 0.779-0.795). In the visit-based model (UK tariff), all post-baseline visits demonstrated significant utility improvements: At the end of the study, Week 456 vs baseline +0.05 (95% CI: 0.018-0.087). Baseline utility was a significant predictor of follow-up utility, with an estimate of 0.65 (95% CI: 0.522-0.768). In the EDSS-adjusted model, utility decreased as disability increased, with the greatest decrement seen in EDSS >6.0 (-0.211, 95% CI: -0.279 to -0.142).
CONCLUSIONS: EQ-5D utilities have increased significantly during long-term Satralizumab treatment. Patients who had higher baseline utility sustained better HRQoL outcomes, while disease progression, measured by EDSS, was associated with reduced utility. These findings underscore the importance of early intervention in preserving HRQoL in NMOSD.