A prospective real‐world study of the efficacy and safety of aducanumab in China: Focus on early‐onset and autosomal dominant Alzheimer's disease

INTRODUCTION: Aducanumab is the first anti-amyloid therapy used for Alzheimer's disease (AD) in China. METHODS: This 12-month, single-center, real-world study enrolled 12 participants with early AD receiving aducanumab 10 mg/kg every 4 weeks (ChiCTR2200066153). The primary outcomes were changes in Clinical Dementia Rating-Sum of Boxes (CDR-SB) scores at 12 months. Secondary outcomes included amyloid clearance, brain structure measures, and biomarker assessments. RESULTS: No amyloid-related imaging abnormalities occurred. The mean CDR-SB score increased by 0.88 at 12 months, with cortical and hippocampal atrophy, enlarged choroid plexus, and ventricular volumes. Two autosomal dominant AD patients exhibited transient amyloid burden elevation at 6 months and subsequent reduction at 12 months, alongside increased serum glial fibrillary acidic protein (GFAP) levels. In the remaining 10 patients, the mean amyloid clearance reached -34.93 Centiloids, alongside decreased serum GFAP levels. DISCUSSION: Aducanumab showed good tolerability and favorable biological outcomes, with different responses in autosomal dominant mutation carriers.