Patient Preferences for Treatment Features in Neuromyelitis Optica Spectrum Disorder (NMOSD): Results From a Discrete Choice Experiment (DCE)
Justin Abbatemarco1, Jeffrey Yu2, Martin Kleman2, Kelley Myers3, Christine Poulos3, Devon Conway1
1Cleveland Clinic, 2Alexion, AstraZeneca Rare Disease, 3RTI Health Solutions
Objective:
To quantify preferences and predict treatment choices between ravulizumab and other approved treatments (eculizumab, inebilizumab, satralizumab) among US adults with anti-aquaporin-4 antibody-positive (AQP4-Ab+) NMOSD.
Background:
Benefit-risk profiles and mode/frequency of administration vary among the 4 US-approved NMOSD treatments. No published studies have reported treatment preferences among US patients.
Design/Methods:
A cross-sectional, web-based DCE survey was administered to US adults with self-reported AQP4-Ab+ NMOSD. Respondents evaluated hypothetical NMOSD treatment profile pairs defined by efficacy, risk, process-related attributes, and administration mode/frequency. Data were analyzed using a random parameters logit model to estimate conditional relative attribute importance, minimum acceptable benefit, and predicted treatment choice in pairwise treatment profile (ravulizumab-like, eculizumab-like, inebilizumab-like, satralizumab-like) comparisons. Preference heterogeneity was investigated by exploratory subgroup analysis.
Results:
The 255 survey completers (mean±SD age, 41.4±13.7y) averaged 6.6±5.4y since diagnosis, 64% identified as female, and 15.7%/9.4%/7.1% were receiving eculizumab/inebilizumab/satralizumab. Respondents placed greatest importance on reducing chance of relapse within the first year of treatment, followed by reducing administration frequency from every 2 weeks (Q2W) to Q8W; the 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 3 treatment profiles (12.3%-32.2%). Preferences did not vary across subgroups defined 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 (with or without oral treatments) placed more weight on reducing chance of relapse versus those receiving oral treatments alone but were not more likely to select a Q24W treatment over a Q8W treatment.
Conclusions:
Patients with AQP4-Ab+ NMOSD placed the highest value on reducing relapse risk and were more likely to select a ravulizumab-like profile over comparators. These findings can inform shared decision-making in selecting treatments.
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