Indirect Comparison Analysis of United States Food and Drug Administration-Approved Treatment Options for Adults with Aquaporin-4 Immunoglobulin G-Positive Neuromyelitis Optica Spectrum Disorder
Author(s)
Wingerchuk D1, Pittock SJ2, Levy M3, Fujihara K4, Nakashima I5, Paul F6, Kielhorn A7, Royston M8, Tanvir I8, Zhang I9
1Mayo Clinic, Phoenix, AZ, USA, 2Mayo Clinic, Rochester, MN, USA, 3Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA, 4Fukushima Medical University, Koriyama, Japan, 5Tohoku Medical and Pharmaceutical University, Sendai, Japan, 6Charité-Universitätsmedizin Berlin, Berlin, Germany, 7Alexion Pharmaceuticals, Norwell, MA, USA, 8Alexion Pharmaceuticals, Boston, MA, USA, 9PRECISIONheor, San Francisco, CA, USA
INTRODUCTION: Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune disease that attacks the central nervous system and can cause irreversible damage. In light of several approved treatment options, a comparison of relative treatment effects would facilitate the treatment selection process. OBJECTIVES: The objective of this study was to perform an indirect treatment comparison (ITC) on the efficacy of all United States Food and Drug Administration-approved treatments (eculizumab, inebilizumab, and satralizumab) in adults with aquaporin-4 immunoglobulin G-positive (AQP4+) NMOSD using published data from randomized controlled trials (RCTs). METHODS: A Bayesian network meta-analysis (NMA) was performed to estimate the relative treatment effects between eculizumab, inebilizumab, and satralizumab based on data extracted from RCTs. Analyses were performed for clinically relevant subpopulations based on 3 treatment networks (monotherapy, combination therapy, and mono-combination therapy). For time-to-first relapse, the NMA was performed using a regression model with a contrast-based normal likelihood for the log hazard ratio (HR) and corresponding standard error for each trial (or comparison) in the network. Relative treatment effects were expressed as HRs, which is standard for an ITC. RESULTS: Time-to-first relapse was the only outcome measure shared across all RCTs. In the monotherapy network, patients on eculizumab were 90% less likely to experience a first relapse compared with satralizumab (HR: 0.10, 95% credible interval [Crl]: 0.01, 0.65) and 89% less likely to relapse than with inebilizumab (HR: 0.11, 95% CrI: 0.02, 0.68). In addition, patients treated with eculizumab ± immunosuppressant therapy (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 for treating adults with AQP4+ NMOSD.
Conference/Value in Health Info
2021-11, ISPOR Europe 2021, Copenhagen, Denmark
Value in Health, Volume 24, Issue 12, S2 (December 2021)
Code
POSA8
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment, Comparative Effectiveness or Efficacy
Disease
Neurological Disorders