Abstract Details

Title Indirect Comparison Analysis of FDA-Approved Treatment Options for Adults with Aquaporin-4 Immunoglobulin G-positive Neuromyelitis Optica Spectrum Disorder

Topic Autoimmune Neurology

Presentation(s) P15 - Poster Session 15 (5:30 PM-6:30 PM)

Poster/Presentation
Number 9-007

Background Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune disease targeting the central nervous system that can cause irreversible damage to the spinal cord, optic nerves, brainstem, and brain. As several therapies are approved, a comparison of their relative treatment effects would facilitate the individual patient treatment selection process.

Objective

To perform an indirect treatment comparison (ITC) on the efficacy of all FDA-approved treatments in adult patients with aquaporin-4 immunoglobulin G-positive (AQP4-IgG+) NMOSD using published data from randomized controlled trials (RCTs).

Design/Methods A Bayesian network meta-analysis (NMA) was performed to estimate the relative treatment effects between eculizumab, inebilizumab, and satralizumab based on RCT data. Analyses were performed for clinically relevant subpopulations based on 3 treatment networks (monotherapy, combination therapy, mixed mono-combination therapy). For time-to-first relapse, the NMA used a regression model with a contrast-based normal likelihood for the log hazard ratio (HR) and corresponding standard error of each trial (or comparison) in the network. The NMA was performed under the assumption of proportional hazards. Relative treatment effects were expressed as HRs, which is standard for an ITC.

Results Time-to-first relapse was the only outcome measure reported across all clinical trials. When evaluating time-to-first relapse, patients on eculizumab monotherapy were 90% less likely to experience a first relapse compared with satralizumab monotherapy (HR: 0.10, 95% credible interval [Crl]: 0.01, 0.65) and 89% less likely to experience a first relapse than with inebilizumab monotherapy (HR: 0.11, 95% CrI: 0.02, 0.68). Patients treated with eculizumab ± immunosuppressants (IST) were 76% less likely to experience a first relapse when compared with satralizumab ± IST (HR: 0.24, 95% CrI: 0.06, 0.98).

Conclusions Using available RCT data, NMA results showed that eculizumab monotherapy and eculizumab ± IST demonstrated greater efficacy in prolonging time-to-first relapse when compared with either satralizumab or inebilizumab in adults with AQP4-IgG+ NMOSD.

Authors/Disclosures
Dean M. Wingerchuk, MD, FAAN (Mayo Clinic) PRESENTER	Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Imcyse. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TTY Biopharm. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reistone Biopharm. Dr. Wingerchuk has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Dr. Wingerchuk 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. Wingerchuk has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bristol Meyer Squibb. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. Wingerchuk has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AstraZeneca. Dr. Wingerchuk has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Wingerchuk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abcuro. Dr. Wingerchuk has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wolters Kluwers.
Sean J. Pittock, MD (Mayo Clinic Dept of Neurology)	Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech, Inc.. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sage Therapeutics, Inc.. The institution of Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prime Therapeutics. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche/Genentech. The institution of Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. The institution of Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MedImmune/Viela Bio. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arialys Therapeutics. The institution of Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche/Genentech. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB, Inc. Dr. Pittock has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman/LaRoche AG. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genetech. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for F. Hofman/LaRoche. The institution of Dr. Pittock has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Pittock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Arialys. The institution of Dr. Pittock has received research support from Grifols. The institution of Dr. Pittock has received research support from NIH. The institution of Dr. Pittock has received research support from Viela Bio/MedImmune/Horizon. The institution of Dr. Pittock has received research support from Alexion Pharmaceuticals. The institution of Dr. Pittock has received research support from F. Hoffman/LaRoche/Genentech. The institution of Dr. Pittock has received research support from NovelMed. The institution of Dr. Pittock has received research support from AstaZeneca. Dr. Pittock has received intellectual property interests from a discovery or technology relating to health care. Dr. Pittock has received intellectual property interests from a discovery or technology relating to health care. Dr. Pittock has received publishing royalties from a publication relating to health care.
Michael Levy, MD, PhD, FAAN (Massachusetts General Hospital/Harvard Medical School)	Dr. Levy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Pharma. Dr. Levy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB Pharma. Dr. Levy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Levy has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Levy 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. Levy 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. Levy has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. Dr. Levy has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Various law firms. The institution of Dr. Levy has received research support from National Institutes Health.
Kazuo Fujihara, MD (Tohoku University, School of Medicine)	Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck Boipharma. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Japan Tobacco. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubishi-Tanabe. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai/Roche. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon. The institution of Dr. Fujihara has received research support from Ministry of Health, Welfare and Labor of Japan. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Novartis. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving as a speaker, chair, etc with Biogen. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Mitsubishi-Tanabe. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a apeaker, chair, etc with Chugai/Roche. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker, chair, etc with Alexion. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a chair, speaker with Asahi Kasei Medical. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker with Eisai. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker, chair with Teijin.
Ichiro Nakashima, MD, PhD (Department of Neurology, Tohoku Medical and Pharmaceutical University Hospital)	Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Novartis. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Chugai. The institution of Dr. Nakashima has received research support from LSI Medience.
Friedemann Paul (Charite Universitatsmedizin in Berlin)	The institution of Dr. Paul has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe PC (MTPC). The institution of Dr. Paul has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Aarhus University. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Paul has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Genzyme. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CELGENE. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Bayer. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for ACRELION. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Viela Bio. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizter. Dr. Paul has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Merck Serono. The institution of Dr. Paul has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Springer.
Adrian Kielhorn	Adrian Kielhorn has received personal compensation for serving as an employee of Alexion. Adrian Kielhorn has stock in Alexion.
	No disclosure on file
Imran Tanvir	Imran Tanvir has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Imran Tanvir has received stock or an ownership interest from Alexion Pharmaceuticals.
	No disclosure on file