Rethinking Treatment Strategies in NMOSD Based on Safety Failures and Emerging Therapies: Shamik Bhattacharyya, MD

WATCH TIME: 5 minutes

"Our concept of what constitutes failure might need to evolve into a combination of efficacy failure and safety failure. That could be a more comprehensive way to think about it."

There are currently 4 FDA-approved therapies for aquaporin-4-antibody seropositive neuromyelitis optica spectrum disorder (NMOSD): satralizumab (Enspryng; Genentech), eculizumab (Soliris; Alexion), ravulizumab (Ultomiris; Alexion), and inebilizumab (Uplizna; Amgen). Additionally, rituximab, mycophenolate mofetil (MMF), and azathioprine (AZA) remain commonly used treatments. A recent study presented at the 2024 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, September 18-20, in Copenhagen, Denmark, evaluated the efficacy and safety of these therapies in 107 patients with NMOSD, predominantly women (82.2%), with a median follow-up of 9.8 years. Among 280 relapses analyzed, 65.3% required hospitalization, and 83.5% were associated with new MRI T2 lesions.

The study reported varying failure rates, defined as at least 1 relapse on treatment, with rituximab at 40%, MMF at 59%, and AZA at 75%. Notably, no relapses occurred with satralizumab, while eculizumab and inebilizumab each reported one relapse during early treatment. Non-relapse hospitalizations were common, with infections accounting for 55.7% of cases, predominantly in patients on rituximab (57.9%). Overall, these findings highlighted the need for careful consideration of both efficacy and safety when selecting NMOSD therapies.

During the Congress, senior author Shamik Bhattacharyya, MD, a neurologist at Brigham and Women’s Hospital, expanded on these findings in an interview with NeurologyLive^®. He discussed the critical balance clinicians must strike between efficacy and safety when deciding to switch NMOSD therapies, particularly in light of recurrent infections and treatment-related failures. Bhattacharyya emphasized the importance of robust observational studies to better capture real-world outcomes across diverse patient populations, which often differ from clinical trial cohorts. Additionally, he highlighted how evolving diagnostic criteria for multiple sclerosis could improve early diagnosis and prompt treatment initiation, ultimately optimizing patient outcomes.

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REFERENCES
1. Bilodeau PA, Narasimhan S, Pua DK, et al. Comparative Effectiveness and Safety of Disease-modifying Treatments (DMTs) in a Real-World Neuromyelitis Optica Spectrum Disorder Cohort (NMOSD). Presented at: 2024 ECTRIMS; September 18-20; Copenhagen, Denmark. Abstract P022.