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

Abstract 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.