Satralizumab Treatment in Adult Patients With AQP4-IgG–Seropositive Neuromyelitis Optica Spectrum Disorder Switching From Other Therapies: A Case Series

Abstract

Purpose : Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune neuroinflammatory disease that primarily affects the optic nerves and spinal cord and can lead to sensory and motor impairment, vision loss and permanent neurological disability. Satralizumab was approved for use in adults with aquaporin 4 autoantibody–positive (AQP4-IgG+) NMOSD in 2020, but real-world data are limited. This case series describes patients with AQP4-IgG+ NMOSD who received satralizumab following treatment with biologics or conventional immunosuppressive therapies (ISTs).

Methods : Case information for patients with AQP4-IgG+ NMOSD who received satralizumab for ≥6 months was obtained from US healthcare providers; case collection is ongoing. Patient characteristics, examination findings, treatment response and reported satralizumab-related adverse events were recorded. Patients who received biologics or ISTs (other than corticosteroids) before satralizumab were included.

Results : To date, 11 patients aged 23-69 years are included; 5 self-identified as Black/African American, 4 as White and 2 as Hispanic. Nine patients are female; ~50% have autoimmune comorbidities. NMOSD disease duration ranged from 3-14 years; symptoms included optic neuritis and transverse myelitis. Eight patients had received rituximab (2 with concomitant mycophenolate mofetil [MMF]), 2 MMF alone and 1 eculizumab as the last NMOSD treatment prior to satralizumab, with reasons for satralizumab initiation including treatment intolerance and inadequate disease control. To date, patients received satralizumab for 14-84 months. Eight patients received satralizumab monotherapy, and 3 received satralizumab in combination with MMF. Ten 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 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 III SAkura clinical trials.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.