Successful Natalizumab Treatment of Two Female Individuals With Susac Syndrome

Abstract

Background

Susac syndrome is a rare autoimmune endotheliopathy that affects the central nervous system, retina, and inner ear, characterized by encephalopathy, branch retinal artery occlusions, and sensorineural hearing loss. Due to the heterogeneity of its presentation, early diagnosis, and treatment remain challenging.

Objective/Methods

To evaluate the clinical outcomes and radiological responses in two patients with Susac syndrome treated with natalizumab in an off-label therapeutic approach, clinical assessments and serial magnetic resonance imaging (MRI) were performed over a follow-up period of up to 22 months to monitor disease progression and treatment response.

Results

Both patients demonstrated clinical stabilization with reduced MRI and retinal angiography disease activity. Treatment was well tolerated, and no significant adverse events were reported during observation.

Discussion

Natalizumab may constitute a potential off-label therapeutic for Susac syndrome. Further studies are warranted to assess its efficacy and safety in this rare condition.