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Blood-based biomarkers have unique value for the development of treatments because they are easily available and relatively cheap.
Frontotemporal lobe degeneration (FTLD) produces behavioral, cognitive, language, and movement disorders, which damage the quality of life of patients and caregivers more severely than other forms of dementia.
About 20-30% of FTLD cases are familial, of which about 60% are caused by autosomal dominant mutations in three genes: chromosome 9 open reading frame 72 (C9orf72), protogranular protein (GRN) and Microtubule-associated protein tau (MAPT).
Because of these mutations, several therapies are preparing to start clinical trials for familial FTLD (f-FTLD).
Due to the low incidence of f-FTLD and the lack of good clinical endpoints to monitor the severity of the disease and treatment response, it is challenging to plan such studies.
Neurofilament light chain (NfL) is a sensitive marker of neurodegeneration.
Compared with Alzheimer's disease and healthy controls, CSF NfL in patients with FTLD is elevated, and its concentration is related to disease severity, cognitive function, and disease progression.
In multiple sclerosis and spinal muscular atrophy, the concentration of CSF NfL will return to normal after effective treatment, which indicates that NfL is very sensitive to the treatment effect.
Serum NfL is elevated in FTLD, and its concentration is associated with brain atrophy in symptomatic carriers of FTLD pathogenic mutations.
Therefore, it is reasonable to assume that plasma NfL can identify asymptomatic carriers of f-FTLD mutations, and their risk of developing symptomatic diseases is high.
Plasma NfL can identify asymptomatic carriers of f-FTLD mutations, and their risk of progression to symptomatic diseases is high.
Therefore, Julio C.
Rojas of the University of Los Angeles, San Francisco, et al.
explored differences in baseline plasma NfL related to phenotype, genotype, and disease severity, and whether it can predict disease progression in two independent cohorts.
They used Simoa to measure baseline plasma NfL concentrations in the original cohort (n = 277) and validation cohort (n = 297).
Use the CDR® dementia staging tool and the National Alzheimer's Disease Coordination Center FTLD module (CDR®+NACC-FTLD) in the behavior and language fields to treat C9orf72, GRN and MAPT mutation carriers and non-carriers in the same family according to the severity of the disease The degree [asymptomatic, prodromal and complete phenotype] is classified.
Finally, a linear mixed-effects model was used to link NfL with clinical variables.
They found that in both cohorts, asymptomatic mutation carriers had a higher baseline NfL than non-progressive mutations.
Plasma NfL can distinguish symptomatic and asymptomatic mutation carriers or precursor diseases.
Plasma NfL can distinguish symptomatic and asymptomatic mutation carriers or precursor diseases.
A higher baseline NfL is associated with a poor longitudinal CDR®+NACC-FTLD total score, neuropsychological function, and atrophy, and has nothing to do with genotype or disease severity, including asymptomatic mutation carriers.
Based on two independent cohort studies, plasma NfL FTLD can determine asymptomatic carriers of disease-causing mutation, the risk of disease progression in the short term, is to choose the prevention of a potential tool for clinical trial subjects.
Original source: neurology.
org/content/early/2021/04/07/WNL.
0000000000011848" target="_blank" rel="noopener">Plasma Neurofilament Light for Prediction of Disease Progression in Familial Frontotemporal Lobar Degeneration
neurology.
org/content/early/2021/04/07/WNL.
0000000000011848" target="_blank" rel="noopener">Plasma Neurofilament Light for Prediction of Disease Progression in Familial Frontotemporal Lobar Degeneration
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