High-Efficacy Therapies Reduce Clinical and Radiological Events More Effectively than Traditional Treatments in Neuromyelitis Optica Spectrum Disorder
Tatum Moog1, Alexander Smith1, Morgan McCreary1, Katy Burgess1, Darin Okuda1
1Neurology, The University of Texas Southwestern Medical Center
Objective:

To compare differences in clinical and radiological outcomes between high-efficacy and traditional therapies in people with neuromyelitis optica spectrum disorder (NMOSD).

Background:

NMOSD is a neurological autoimmune condition characterized by recurring attacks and permanent disability. It is unknown if treatment with high-efficacy agents is better than traditionally used medications.

Design/Methods:
A retrospective analysis of people with NMOSD exposed to traditional treatment, high-efficacy treatment, or both was conducted. Therapies classified as high-efficacy included eculizumab, inebilizumab, satralizumab, etc. Traditional therapies were defined as azathioprine, methotrexate, mycophenolate mofetil, etc. Comparisons of clinical/radiological events while exposed to traditional or high-efficacy therapy were performed by evaluating the number of events related to MRI advancement and location of progression (brain or spinal cord), relapses, and hospitalizations. A Poisson regression model was constructed, and high-efficacy treatments were determined to affect any of the three outcomes of interest if the corresponding 95% credible interval did not contain zero.
Results:
The study cohort included 189 people with NMOSD fulfilling International Panel Criteria (Aquaporin-4 IgG+: 161) with 94 (80 female; median disease duration (range) of 6.75 years (y) (0.77-21.28)) exposed only to high-efficacy therapy, 32 (28 female; median disease duration of 10.04y (0.37-17.36)) exposed only to traditional therapy, and 63 (52 female; median disease duration of 7.61y (0.23-22.11)) exposed to both. High-efficacy treatments reduced the event rate with MRI advancement by 65.70% (95% Credible Interval=[-86.47%, -15.63%]), the relapse rate by 99.82% (95% Credible Interval=[-99.92%, -99.59%]), and the hospitalization rate by 99.33% (95% Credible interval=[-99.64%, -98.78%]). A 15-fold increase in the odds of MRI advancement involving the spinal cord (95% credible interval=[2.34, 287.15]) was observed during treatment with a traditional medication versus with a high-efficacy agent.
Conclusions:
The availability of newer treatments maximizes the opportunity to reduce permanent neurological disability over traditionally used medications in both newly diagnosed and established people with NMOSD.
10.1212/WNL.0000000000202012