Patient Preferences for Treatment Features in Neuromyelitis Optica Spectrum Disorder (NMOSD)

Abstract

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. Disclosure: Dr. Abbatemarco has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EMD Serono. Dr. Abbatemarco has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon. Dr. Abbatemarco has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech . Dr. Abbatemarco has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics, Inc.. Dr. Abbatemarco has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. The institution of Dr. Abbatemarco has received research support from Horizon. Dr. Yu has received personal compensation for serving as an employee of Alexion. Dr. Yu has stock in AstraZeneca. Mr. Kleman has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Mr. Kleman has stock in AstraZeneca. Dr. Myers has nothing to disclose. Ms. Poulos has nothing to disclose. Dr. Conway has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Pharmaceuticals. Dr. Conway has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for AstraZeneca. Dr. Conway has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bristol Myers Squibb. Dr. Conway has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TG Therapeutics. Dr. Conway has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Conway has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. The institution of Dr. Conway has received research support from Novartis. The institution of Dr. Conway has received research support from BMS. The institution of Dr. Conway has received research support from Biogen. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.