Comparative effectiveness and safety of DMTs in NMOSD

In a real-world study, the treatment failure rate and toxicity of rituximab as a treatment of neuromyelitis optica spectrum disorder (NMOSD) were higher than previously reported. While the failure rate for rituximab was 30%, there were no failures on satralizumab, eculizumab, ravulizumab, and inebilizumab.

There are 4 FDA-approved treatments for aquaporin-4 (AQP4)-positive NMOSD: satralizumab, eculizumab, ravulizumab, and inebilizumab. Traditionally, mycophenolate mofetil, azathioprine, and especially rituximab have been used as first-line immunosuppressive treatments for NMOSD; these are still commonly prescribed. On rituximab, relapse failure rates for patients with NMOSD of up to 35% have been reported [1]. Dr Philippe-Antoine Bilodeau (Brigham and Women’s Hospital, MA, USA) and colleagues aimed to establish the comparative effectiveness and safety of NMOSD therapies in a cohort of their own patients [2].

The researchers set up a retrospective analysis of treatment outcomes in 178 patients, who had a mean age at diagnosis of 45.3 years, were mostly AQP4-positive (86.5%), women (83.7%), and White (62.9%); 128 patients received rituximab, 40 received mycophenolate mofetil, 14 azathioprine, 12 satralizumab, 12 eculizumab, and 9 inebilizumab. The median follow-up was 9.8 years.

Despite suppressed B cells, rituximab had a failure rate (≥1 relapse while on therapy) of 30%, while the new FDA-approved therapies had no failures. Higher doses of rituximab were not more protective. There was a high burden of non-relapse hospitalisations, primarily due to infection. Of patients on rituximab, 28.7% had an infection-related hospitalisation. There was a signal towards lower rates of infection-related hospitalisations on eculizumab versus rituximab. The researchers added that conducting a multicentre analysis would help to further validate these results, especially the outcomes on rituximab.

1. Barreras P, et al. Neurology. 2022;99(22):e2504-16.
2. Bilodeau P-A, et al. Comparative effectiveness and safety of disease-modifying treatments (DMTs) in a real-world neuromyelitis optica spectrum disorder cohort (NMOSD). P022, ECTRIMS 2024, 18–20 September, Copenhagen, Denmark.

 

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Posted on 18 November 202418 November 2024