Comparative Analysis Insights on Neuromyelitis Optica Spectrum Disorder Treatments: Shamik Bhattacharyya, MD

WATCH TIME: 4 minutes

"We are very often as a field focused on relapses, and perhaps not enough on infection hospitalizations or other serious complications of therapy."

Currently, there are 4 FDA-approved treatments for aquaporin-4-antibody seropositive neuromyelitis optica spectrum disorder (NMOSD): satralizumab (Enspryng; Genentech), eculizumab eculizumab (Soliris; Alexion), ravulizumab (Ultomiris; Alexion), and inebilizumab (Uplizna; Amgen). Besides these therapies, rituximab (RTX), mycophenolate mofetil (MMF) and azathioprine (AZA) are also commonly used to treat the disease. At the 2024 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, September 18-20, in Copenhagen, Denmark, a new study presented assessed the efficacy and safety of both off- and on-label therapies in the treatment of patients with NMOSD.¹

In this analysis of patients with NMOSD (n = 107), investigators collected data from the Mass General Brigham system (mean age at diagnosis, 45.3 years [SD, 15.9]; AQP4 positive, 93.4%, women, 82.2%; White 63.6%) as well as defined relapses as new signs and symptoms consistent with a core NMOSD phenotype. Authors also included all non-relapse hospitalizations and noted that the median follow-up was 9.8 years (IQR 8.1). Among the total 280 relapses analyzed by researchers, 65.3% of them were reported as requiring hospitalization and 83.5% of the relapses with available MRI had a new T2 lesion.

Conducted senior author Shamik Bhattacharyya, MD, and colleagues, 85 patients were given rituximab, compared with 27 patients for MMF, 12 patients for AZA, 4 patients for satralizumab, 5 patients for inebilizumab, and 9 patients for eculizumab. The median treatment duration was 6.5 years (IQR 7.5) for MMF, 5.8 years (IQR 6.4) for rituximab, 1.05 years (IQR 3.69) for AZA, 2.1 years (IQR 2.74) for eculizumab, 3.0 years (IQR 0.13) for inebilizumab and 1.2 years (IQR 2.3) for satralizumab. The failure rate, which was defined as at least 1 relapse on treatment, was reported by investigators as 40% for rituximab, 59% for MMF and 75% for AZA.

In the analysis, the most common treatments at relapse were rituximab (30.6%), MMF (23.2%) and AZA (11.0%). Researchers also reported no relapses observed with patients on satralizumab, 1 relapse reported on eculizumab 1 week after starting treatment, and 1 relapse reported on inebilizumab prior to completing the initial load. In 183 non-relapse hospitalizations recorded, 55.7% were because of infections and 57.9% were reported while on rituximab treatment, compared with 23.2% for MMF and less than 1% for satralizumab, inebilizumab, and eculizumab.

During the Congress, Bhattacharyya, a neurologist at Brigham and Women's Hospital, sat down with NeurologyLive^® in an interview to discuss how relapse rates in NMOSD differed between rituximab and the newer FDA-approved therapies. He also talked about the factors that may contribute to the 30% failure rate of rituximab in preventing NMOSD relapses. Furthermore, Bhattacharyya, who also serves as an associate professor of neurology at Harvard Medical School, spoke about the barriers that might exist for patients who would switch from rituximab to newer NMOSD therapies.

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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.