Satralizumab Treatment in Adult Patients With AQP4-IgG–Seropositive Neuromyelitis Optica Spectrum Disorder: A Case Series
Hesham Abboud1, Brian Steingo2, Diana Vargas3, Julie Patel4, Nancy Nealon5, Mary Alissa Willis6, Yang Mao-Draayer7, Dmitry Khaitov8, Jose Avila Ornelas9, Adnan Subei10, Clifford Reed11, Lisa Ferayorni12, Xavier Laucirica12, Shervin Gholizadeh12
1University Hospitals Cleveland Medical Center, 2Infinity Clinical Research, 3Department of Neurology, Emory University School of Medicine, 4Department of Neurology and Department of Pharmacy, Emory University School of Medicine and Emory University Hospital Midtown, 5Department of Neurology, Weill Cornell Medicine, 6Department of Neurology, University of Mississippi Medical Center, 7Department of Neurology, University of Michigan School of Medicine, 8Lehigh Valley Health Network, 9HIMA Caguas Multiple Sclerosis Center, 10Memorial Healthcare System, 11Department of Neurology, Reading Hospital, 12Genentech, Inc.
Objective:
To describe the response to satralizumab in adult patients with aquaporin 4 autoantibody–positive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD) in clinical practice.
Background:
NMOSD is a rare autoimmune neuroinflammatory disease that primarily affects the optic nerves and spinal cord and can lead to sensory/motor impairment, vision loss and permanent neurological disability. The US FDA approved satralizumab for use in adult patients with AQP4-IgG+ NMOSD in 2020, but real-world data are limited.
Design/Methods:
Case information for patients with AQP4-IgG+ NMOSD who had received satralizumab for ≥6 months was obtained from US healthcare providers; collection of cases is ongoing. Patient characteristics, exam findings, diagnostic tests, treatment response and reported satralizumab-related adverse effects were recorded.
Results:
To date, 16 patients aged 23-74 years and of multiple races and ethnicities are included. Most patients are female; approximately half have autoimmune comorbidities. NMOSD disease duration ranged from <1-14 years; symptoms included optic neuritis and transverse myelitis. More than half of patients received immunosuppressants other than corticosteroids (most commonly mycophenolate mofetil) or disease-modifying therapies (most commonly rituximab) for NMOSD before satralizumab, with reasons for switch including treatment intolerance and inadequate disease control. To date, patients received satralizumab for 7-21 months, either as monotherapy or in combination with immunosuppressants. Fifteen patients were relapse free with satralizumab, despite reports of confirmed or suspected relapses with previous treatments in some patients. Overall, patients maintained disease control, with few adverse events reported (leukopenia being the most common). No patients permanently discontinued satralizumab due to ineffectiveness or intolerance.
Conclusions:
In this ongoing retrospective case series, patients with NMOSD receiving satralizumab either as monotherapy or in combination with immunosuppressants in clinical practice had a beneficial response compared with previous treatments, and satralizumab was well tolerated. These outcomes align with the efficacy and safety outcomes with satralizumab in Phase 3 SAkura clinical trials.