Comparative Effectiveness and Safety of Disease-modifying Treatments (DMTs) in a Real-World Neuromyelitis Optica Spectrum Disorder Cohort (NMOSD)
Year: 2024
Type: article
Study design: Observational Study
Abstract: 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.
Source: Neurology
Language: English
Subfield: Pathology and Forensic Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
OpenAlex ID: W4403206788