Patient preferences for neuromyelitis Optica spectrum disorder (NMOSD) treatments

Abstract

INTRODUCTION: Benefit-risk profiles and mode/frequency of administration vary among the four US-approved treatments for anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4-Ab+ NMOSD), but no published studies have reported treatment preferences among patients with NMOSD in the USA. Therefore, the objective of this study was to quantify preferences and predict treatment choices among patients with AQP4-Ab+ NMOSD in the USA.

METHODS: A cross-sectional, web-based discrete choice experiment survey was administered to patients with self-reported AQP4-Ab+ NMOSD. Respondents evaluated hypothetical AQP4-Ab+ NMOSD treatment profile pairs defined by efficacy, safety, process-related attributes, and administration mode/frequency. Data were analyzed using a random-parameters logit model to estimate conditional relative importance of attributes, minimum acceptable benefit, and predicted treatment choice in pairwise treatment profile (ravulizumab-like, eculizumab-like, inebilizumab-like, and satralizumab-like) comparisons. Preference heterogeneity was investigated by exploratory subgroup analysis using interaction terms in the regression analysis.

RESULTS: The 255 survey completers (mean ± standard deviation [SD] age, 41.4 ± 13.7 years) averaged 6.6 ± 5.4 years since diagnosis, and 63.5% identified as female and 46.7% as Black or African American. Rituximab was the most common treatment (34.1% of respondents), and 15.7%, 9.4%, or 7.1% of respondents were receiving eculizumab, inebilizumab, or satralizumab, respectively. Respondents placed greater importance on reducing chance of relapse within the first year of treatment compared with other attributes such as reducing administration frequency from every 2 weeks to every 8 weeks (Q8W); safety attributes (risks of serious opportunistic or recurrent infection, elevated liver enzymes, and meningococcal infection) were rated similar to each other in importance. Pairwise comparisons favored the ravulizumab-like profile (67.8-87.7%) over the other three treatment profiles (12.3-32.2%). Preferences did not vary by age, disease duration, disease impact, or relapses in the past 12 months, but subgroups defined by current treatment type demonstrated different (p = 0.066) preferences. Respondents receiving intravenous/subcutaneous injections alone or with oral immunosuppressive therapy/corticosteroid treatments placed greater importance on reducing chance of relapse versus those receiving oral treatments alone, but they were not more likely to select a Q24W treatment over a Q8W treatment.

CONCLUSIONS: Respondents with AQP4-Ab+ NMOSD placed the highest importance on reducing chance of relapse, placed a high importance on reduced administration frequency, and rated the safety attributes (avoiding treatments with a risk of meningococcal infection, risk of elevated liver enzymes, and risk of other infections) as comparable to each other but less influential than relapse prevention. Respondents were more likely to select a ravulizumab-like profile over comparators. These findings can inform shared decision-making in selecting treatments.