Abstract Details

Title Comparative Effectiveness and Safety of Disease-modifying Treatments (DMTs) in a Real-World Neuromyelitis Optica Spectrum Disorder Cohort (NMOSD)

Topic Autoimmune Neurology

Presentation(s) P2 - Poster Session 2 (2:45 PM-3:45 PM)

Poster/Presentation
Number 003

Objective Determine treatment effectiveness and safety outcomes in a cohort of NMOSD patients.

Background In addition to the 4 FDA-approved treatments for AQP4-positive NMOSD (satralizumab, eculizumab, ravulizumab, and inebilizumab), rituximab (RTX), mycophenolate mofetil (MMF) and azathioprine (AZA) are commonly used to treat NMOSD. The comparative effectiveness and safety of these therapies in real-world cohorts remains unclear.

Design/Methods Retrospective analysis of treatment outcomes in patients with NMOSD at Mass General Brigham. Relapses were defined as new signs and symptoms consistent with a core NMOSD phenotype. All non-relapse hospitalizations were included.

Results 107 patients were included. The majority were AQP4 positive (93.4%), female (82.2%) and white (63.6%). 480 relapses were analyzed. 83.5% of relapses with available MRI had a new T2 lesion. 85 patients received RTX, compared to 27 for MMF, 12 for AZA, 4 for satralizumab, 5 for inebilizumab and 9 for eculizumab. Median treatment duration (years) was 6.5 (IQR 7.5) for MMF, 5.8 (IQR 6.4) for RTX, 1.05 (IQR 3.69) for AZA, 2.1 (IQR 2.74) for eculizumab, 3.0 (IQR 0.13) for inebilizumab and 1.2 (IQR 2.3) for satralizumab. The failure rate was 40% for RTX, 59% for MMF and 75% for AZA. The CD19 CD20 count were 0 for 10/11 and 7/7 relapses on RTX. ALC was <1.5 x 10³ cells/μL for 11/13 relapses on MMF. There were no relapses on satralizumab, one relapse on eculizumab 1 week after starting treatment and one relapse on inebilizumab prior to completing the initial load. Out of 183 non-relapse hospitalizations, 55.7% were due to infections and 57.9% occurred while on RTX, compared to 23.2% for MMF and <1% for satralizumab, inebilizumab and eculizumab.

Conclusions A significant proportion of relapses occurred on RTX despite suppressed B-cells and on MMF despite lymphopenia. Most non-relapse hospitalizations occurred on RTX, while very few occurred on satralizumab, inebilizumab, and eculizumab.

Authors/Disclosures
Philippe-Antoine Bilodeau, MD (Massachusetts General Hospital) PRESENTER	Dr. Bilodeau has nothing to disclose.
Sathya S. Narasimhan, MBBS (Baylor College of Medicine)	Dr. Narasimhan has nothing to disclose.
Danielle Kei Pua, MD (Westchester Medical Center)	Dr. Pua has nothing to disclose.
Mattia Wruble, MD	The institution of Dr. Wruble has received research support from Alexion. The institution of Dr. Wruble has received research support from Roche.
Yihan Zhang (Brigham and Women's Hospital, Harvard Medical School)	No disclosure on file
Farrah J. Mateen, MD, PhD, FAAN (Northwestern University Department of Neurology)	Dr. Mateen has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Mateen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Mateen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Mateen has received research support from Genentech. The institution of Dr. Mateen has received research support from Amgen. The institution of Dr. Mateen has received research support from TG Therapeutics. Dr. Mateen has received intellectual property interests from a discovery or technology relating to health care.
Michael Levy, MD, PhD, FAAN (Massachusetts General Hospital/Harvard Medical School)	Dr. Levy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Pharma. Dr. Levy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB Pharma. Dr. Levy has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Levy has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Levy has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon. Dr. Levy has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Levy has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. Dr. Levy has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Various law firms. The institution of Dr. Levy has received research support from National Institutes Health.
Shamik Bhattacharyya, MD, FAAN (Brigham and Women's Hospital)	Dr. Bhattacharyya has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NeuroLambda. Dr. Bhattacharyya has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Bhattacharyya has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Bhattacharyya has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. Dr. Bhattacharyya has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Continuum. Dr. Bhattacharyya has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wiley. Dr. Bhattacharyya has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Merck. The institution of Dr. Bhattacharyya has received research support from Alexion Pharmaceuticals. The institution of Dr. Bhattacharyya has received research support from National Institute of Health. The institution of Dr. Bhattacharyya has received research support from UCB. The institution of Dr. Bhattacharyya has received research support from Genentech. Dr. Bhattacharyya has received publishing royalties from a publication relating to health care. Dr. Bhattacharyya has received publishing royalties from a publication relating to health care.

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