Discordant cerebrospinal fluid and positron emission tomography amyloid biomarkers in an APP mutation carrier presenting corticobasal syndrome

携带 APP 基因突变并出现皮质基底节综合征的患者的脑脊液和正电子发射断层扫描淀粉样蛋白生物标志物不一致

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Abstract

INTRODUCTION: While amyloid cerebrospinal fluid (CSF) and positron emission tomography (PET) biomarkers are considered interchangeable indicators of Alzheimer's disease (AD) pathology, biomarker discrepancies can occur but remain poorly characterized. METHODS: We evaluated (18)F-florbetapir amyloid PET, (18)F-Florzolotau PET (tau pathology), magnetic resonance imaging (MRI) findings, and CSF biomarkers in a 59-year-old man carrying the pathogenic APP p.K687Q mutation, who presented with possible corticobasal syndrome. RESULTS: CSF analysis revealed reduced amyloid beta (Aβ)(1-42) (503.44 pg/mL) and Aβ(1-42)/Aβ(1-40) ratio (0.044), indicating amyloid pathology. Conversely, (18)F-florbetapir PET was visually negative (standardized uptake value ratio [SUVR] 0.97; -11.8 Centiloids). (18)F-Florzolotau PET demonstrated AD-typical tau deposition, whereas MRI revealed extensive white matter hyperintensities, enlarged perivascular spaces, and a temporal microbleed. DISCUSSION: The observed discordance suggests that CSF and PET amyloid biomarkers can diverge in certain patients. Potential mechanisms include polymorphic Aβ fibrils lacking (18)F-florbetapir binding sites, excess non-fibrillar aggregates, low fibril density, or contributions from cerebral amyloid angiopathy. HIGHLIGHTS: CSF Aβ and (18)F-florbetapir PET findings showed a mismatch in a patient with an APP mutation. Amyloid pathology should not be excluded despite negative (18)F-florbetapir PET findings. Mismatch may reflect altered ligand binding or fibril structural variants. Comorbid cerebral amyloid angiopathy may contribute to biomarker discrepancies.

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