Relapse, Non-Relapse and Treatment Outcomes in a Real-World Neuromyelitis Optica Spectrum Disorder Cohort
Philippe-Antoine Bilodeau1, Mattia Wruble2, Sathya Narasimhan3, Danielle Kei Pua2, Farrah Mateen4, Michael Levy5, Shamik Bhattacharyya2
1Mass General Brigham/Harvard Medical School, 2Brigham and Women's Hospital, 3Baylor College of Medicine, 4Massachusetts General Hospital, 5Massachusetts General Hospital/Harvard Medical School
Objective:
Characterize relapse and non-relapse hospitalizations and evaluate the efficacy and safety of rituximab, eculizumab, inebilizumab, satralizumab, mycophenolate mofetil (MMF), and azathioprine.
Background:
NMOSD is characterized by severe relapses. There is a lack of comparative efficacy and safety studies comparing rituximab, MMF and azathioprine to eculizumab, inebilizumab and satralizumab.
Design/Methods:
Retrospective cohort study of n=180 NMOSD patients at Mass General Brigham. A negative binomial analysis was used to assess relapse and non-relapse hospitalization rates. Cox proportional hazards models were used to assess relapse-free survival, and logistic regression to assess predictors of disability.
Results:
664 relapses were assessed. Predictors of mRS 3-5 on admission were age at relapse over 60 (OR 13.31, 95% CI 1.89-93.76) and number of prior relapses (OR 1.29, 95% CI 1.04-1.60) but not being on a DMT during relapse. 37% of relapses requiring admission were associated with adverse events, and 22% had an infectious complication. Rituximab showed a relapse-free survival probability of 63.1% at 10 years, while no relapses were observed on eculizumab, inebilizumab, or satralizumab. Rituximab had an ARR of 0.135 (95% CI 0.09-0.188), compared to 0.00004 (0.000-0.023) for eculizumab, 0.00007 (0.000-0.037) for inebilizumab, 0.00007 (0.00-0.048) for satralizumab, 0.26 (0.133-0.420) for MMF, and 0.83 (0.262-1.82) for azathioprine. 315 non-relapse hospitalizations were analyzed. Infections were the most common cause of admission (53%), shock and respiratory failure were common complications and mortality rate was 5%. The composite endpoint of relapse or serious infectious adverse events (AEs) showed better survival probabilities for eculizumab (83% at 5 years), inebilizumab (75% at 3 years), and satralizumab (90% at 5 years) than rituximab (52% at 5 years), MMF (32% at 5 years), and azathioprine (8% at 5 years).
Conclusions:
Eculizumab, inebilizumab, and satralizumab demonstrated better efficacy than rituximab, MMF, and azathioprine. Non-relapse hospitalizations were common, primarily driven by infections, and associated with substantial morbidity.
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