The Cortical Basal ganglia Functional Scale (CBFS): Development and preliminary validation

To develop a patient/care-giver reported scale capable of easily and reliably assessing functional disability in 4 repeat tauopathies (4RTs). 4R tauopathies including progressive supranuclear palsy, corticobasal degeneration and a subset of frontotemporal dementias manifest a range of overlapping cl...

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Vydáno v:	Parkinsonism & related disorders Ročník 79; s. 121 - 126
Hlavní autoři:	Lang, Anthony E., Stebbins, Glenn T., Wang, Ping, Jabbari, Edwin, Lamb, Ruth, Morris, Huw, Boxer, Adam L., Boxer (PI), A., Boeve, B., Dickerson, B., Grossman, M., Litvan, I., Ljubenkov, P., Pantelyat, A., Rojas-Martinez, J., Tartaglia, M.-C., Wills, A.-M., Morris (PI), H., Amar, K., Capps, E., Carey, G., Church, A., Critchley, P., Ghosh, B., Houlden, H., Hu, M., Kobylecki, C., Massey, L., Molloy, S., Nath, U., Pavese, N., Rowe, J.B.
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	England Elsevier Ltd 01.10.2020
Témata:	4 repeat tauopathies Clinical rating scales Corticobasal degeneration Corticobasal Degeneration - diagnosis Delphi Technique Female Frontotemporal Dementia - diagnosis Humans Longitudinal Studies Male Middle Aged Outcome measures Patient Reported Outcome Measures Patient reported outcomes Progressive supranuclear palsy Psychometrics - instrumentation Psychometrics - methods Psychometrics - standards Reproducibility of Results Severity of Illness Index Supranuclear Palsy, Progressive - diagnosis Outcome measures Clinical rating scales Progressive supranuclear palsy 4 repeat tauopathies Corticobasal degeneration Patient reported outcomes
ISSN:	1353-8020, 1873-5126, 1873-5126
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Popis
Shrnutí:	To develop a patient/care-giver reported scale capable of easily and reliably assessing functional disability in 4 repeat tauopathies (4RTs). 4R tauopathies including progressive supranuclear palsy, corticobasal degeneration and a subset of frontotemporal dementias manifest a range of overlapping clinical phenotypes. No available rating scale is capable of evaluating the functional impact of these complex disorders. A multi-staged modified Delphi process was used to propose, evaluate and rank potential scale items providing content validity ratios. Staged cognitive pretesting involving input from examiners, patients and caregivers was followed by validation testing in patients participating in the 4R Tauopathy Neuroimaging Initiative or the PROgressive Supranuclear Palsy CorTico-Basal Syndrome MSA Longitudinal Study. Clinimetric properties were examined using classical test theory and item response methods, assessing data quality, reliability, construct validity, convergent validity and known-group validity. The resultant Cortical Basal ganglia Functional Scale (CBFS) included questions on Motor Experiences in Daily Living (14 items) and Non-Motor Experiences of Daily Living (17 items). Reliability was acceptable for internal consistency, test-retest stability, item discrimination, item-scaling thresholds and item-fit. Examination of construct validity revealed a parsimonious two-factor solution, and concurrent validity demonstrated significant correlations between the CBFS and other measures of disease severity and functional impairment. The CBFS significantly discriminated between all diagnostic groups and controls (all AUCs>90). The CBFS scores demonstrated sensitivity to change over a 12 month follow-up in patients with probable 4RTs. The CBFS is a patient/care-giver reported outcome measure with excellent clinimetric properties that captures disability correlated with motor, cognitive and psychiatric impairments. •There is considerable clinical and pathogenic overlap between the 4R tauopathies (PSP, CBD).•There is no available rating scale capable of evaluating the functional impact of the complex disorders that constitute the 4R tauopathies.•Disease modifying trials directed at common pathogenic factors will require scales that evaluate the complex clinical phenotypes.•Patient/caregiver reported outcomes are increasing emphasized in drug development.•The CBFS is a newly developed scale using patient/caregiver reported information that was designed to address these unmet needs.
Bibliografie:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23
ISSN:	1353-8020 1873-5126 1873-5126
DOI:	10.1016/j.parkreldis.2020.08.021