Introduction-We created global rating scoring rules for the CDR® plus NACC FTLD to detect and track early frontotemporal lobar degeneration (FTLD) and to conduct clinical trials in FTLD.Methods-The CDR plus NACC FTLD rating was applied to 970 sporadic and familial participants from the baseline visit of Advancing Research and Treatment in Frontotemporal Lobar Degeneration (ARTFL)/Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects (LEFFTDS). Each of the eight domains of the CDR plus NACC FTLD was equally weighed in determining the global score. An interrater reliability study was completed for 40 participants.Results-The CDR plus NACC FTLD showed very good interrater reliability. It was especially useful in detecting clinical features of mild non-fluent/agrammatic variant primary progressive aphasia participants.Discussion-The global CDR plus NACC FTLD score could be an attractive outcome measure for clinical trials in symptomatic FTLD, and may be useful in natural history studies and clinical trials in FTLD spectrum disorders.
Introduction:Behavior/Comportment/Personality (BEHAV) and Language (LANG) domains were added to the Clinical Dementia Rating (CDR®) for improving evaluation of frontotemporal lobar degeneration (FTLD) patients (CDR® plus NACC FTLD).
Methods:We analyzed the CDR® plus NACC FTLD among participants from the baseline visit of the ARTFL/LEFFTDS Consortium.
Introduction:
Identifying clinical measures that track disease in the earliest
stages of frontotemporal lobar degeneration (FTLD) is important for clinical
trials. Familial FTLD provides a unique paradigm to study early FTLD.
Executive dysfunction is a clinically relevant hallmark of FTLD and may be a
marker of disease progression.
Methods:
Ninety-three mutation carriers with no symptoms or
minimal/questionable symptoms (
MAPT
, n = 31;
GRN
,n = 28;
C9orf72
,n = 34; Clinical
Dementia Rating scale plus NACC FTLD Module <1) and 78 noncarriers
enrolled through Advancing Research and Treatment in Frontotemporal Lobar
Degeneration/Longitudinal Evaluation of Familial Frontotemporal Dementia
Subjects studies completed the Executive Abilities: Measures and Instruments
for Neurobehavioral Evaluation and Research (NIH-EXAMINER) and the UDS
neuropsychological battery. Linear mixed-effects models were used to
identify group differences in cognition at baseline and longitudinally. We
examined associations between cognition, clinical functioning, and magnetic
resonance imaging volumes.
Results:
NIH-EXAMINER scores detected baseline and differences in slopes
between carriers and noncarriers, even in carriers with a baseline Clinical
Dementia Rating scale plus NACC FTLD Module = 0. NIH-EXAMINER declines were
associated with worsening clinical symptoms and brain volume loss.
Discussion:
The NIH-EXAMINER is sensitive to cognitive changes in presymptomatic
familial FTLD and is a promising surrogate endpoint.
IntroductionIt is important to establish the natural history of familial frontotemporal lobar degeneration (f‐FTLD) and provide clinical and biomarker data for planning these studies, particularly in the asymptomatic phase.MethodsThe Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects protocol was designed to enroll and follow at least 300 subjects for more than at least three annual visits who are members of kindreds with a mutation in one of the three most common f‐FTLD genes—microtubule‐associated protein tau, progranulin, or chromosome 9 open reading frame 72.ResultsWe present the theoretical considerations of f‐FTLD and the aims/objectives of this protocol. We also describe the design and methodology for evaluating and rating subjects, in which detailed clinical and neuropsychological assessments are performed, biofluid samples are collected, and magnetic resonance imaging scans are performed using a standard protocol.DiscussionThese data and samples, which are available to interested investigators worldwide, will facilitate planning for upcoming disease‐modifying therapeutic trials in f‐FTLD.
INTRODUCTION:Behavioral variant Frontotemporal Dementia (bvFTD) may present sporadically or due to an autosomal dominant mutation. Characterization of both forms will improve understanding of the generalizability of assessments and treatments.METHODS: 135 sporadic (s-bvFTD; mean age 63.3 years; 34% female) and 99 familial (f-bvFTD; mean age 59.9; 48% female) bvFTD participants were identified. F-bvFTD cases included 43 with known or presumed C9orf72 expansions, 28 with known or presumed MAPT mutations, 14 with known GRN mutations, and 14 with a strong family history of FTD but no identified mutation.RESULTS: F-bvFTD were younger and had earlier age of onset. S-bvFTD had higher total NPI-Q scores due to more frequent endorsement of depression and irritability.DISCUSSION: Familial and sporadic bvFTD cases are clinically similar, suggesting the generalizability of novel biomarkers, therapies, and clinical tools developed in either form to the other.
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