The Progressive Supranuclear Palsy Clinical Deficits Scale.
clinical rating scales
outcome measures
power calculation
progressive supranuclear palsy
Journal
Movement disorders : official journal of the Movement Disorder Society
ISSN: 1531-8257
Titre abrégé: Mov Disord
Pays: United States
ID NLM: 8610688
Informations de publication
Date de publication:
04 2020
04 2020
Historique:
received:
19
08
2019
revised:
04
12
2019
accepted:
06
12
2019
pubmed:
18
1
2020
medline:
28
4
2021
entrez:
18
1
2020
Statut:
ppublish
Résumé
There is currently no undisputed, validated, clinically meaningful measure for deficits in the broad spectrum of PSP phenotypes. To develop a scale to monitor clinical deficits in patients with PSP across its broad phenotypes. The Progressive Supranuclear Palsy Clinical Deficits Scale was conceptualized to cover seven clinical domains (Akinesia-rigidity, Bradyphrenia, Communication, Dysphagia, Eye movements, Finger dexterity, and Gait & balance), each scored from 0 to 3 (no, mild, moderate, or severe deficits). User guidelines were developed to standardize its application. Progressive Supranuclear Palsy Clinical Deficits Scale scores were collected in patients fulfilling the MDS-PSP diagnostic criteria in two independent, multicenter, observational studies, both cross-sectionally (exploratory DescribePSP cohort; confirmatory ProPSP cohort) and longitudinally (12-months' follow-up, both cohorts). Cognitive pretesting demonstrated easy scale utility. In total, 164 patients were scored (70.4 ± 7.6 years; 62% males, 35% variant phenotypes). Mean Progressive Supranuclear Palsy Clinical Deficits Scale completion time was 4 minutes. The Progressive Supranuclear Palsy Clinical Deficits Scale total score correlated with existing scales (e.g., Progressive Supranuclear Palsy Rating Scale: R = 0.88; P < 0.001). Individual Progressive Supranuclear Palsy Clinical Deficits Scale items correlated well with similar constructs in existing scales. Internal consistency (Cronbach's alpha: 0.75), inter-rater reliability (0.96), and test-retest stability (0.99) were acceptable. The PSP-CDS showed significant 12-month change (baseline, 8.6 ± 3.6; follow-up: 10.8 ± 3.6; annualized difference: 3.4 ± 3.4; n = 49; P < 0.0001). Sample sizes required per arm for a two-arm, 1-year follow-up therapeutic trial to detect 50% change in Progressive Supranuclear Palsy Clinical Deficits Scale progression was estimated to be 65 (two-sided, two-sample t test). The Progressive Supranuclear Palsy Clinical Deficits Scale is a rapidly completed, clinimetrically sound scale for clinical care and research involving PSP. © 2020 International Parkinson and Movement Disorder Society.
Sections du résumé
BACKGROUND
There is currently no undisputed, validated, clinically meaningful measure for deficits in the broad spectrum of PSP phenotypes.
OBJECTIVE
To develop a scale to monitor clinical deficits in patients with PSP across its broad phenotypes.
METHODS
The Progressive Supranuclear Palsy Clinical Deficits Scale was conceptualized to cover seven clinical domains (Akinesia-rigidity, Bradyphrenia, Communication, Dysphagia, Eye movements, Finger dexterity, and Gait & balance), each scored from 0 to 3 (no, mild, moderate, or severe deficits). User guidelines were developed to standardize its application. Progressive Supranuclear Palsy Clinical Deficits Scale scores were collected in patients fulfilling the MDS-PSP diagnostic criteria in two independent, multicenter, observational studies, both cross-sectionally (exploratory DescribePSP cohort; confirmatory ProPSP cohort) and longitudinally (12-months' follow-up, both cohorts).
RESULTS
Cognitive pretesting demonstrated easy scale utility. In total, 164 patients were scored (70.4 ± 7.6 years; 62% males, 35% variant phenotypes). Mean Progressive Supranuclear Palsy Clinical Deficits Scale completion time was 4 minutes. The Progressive Supranuclear Palsy Clinical Deficits Scale total score correlated with existing scales (e.g., Progressive Supranuclear Palsy Rating Scale: R = 0.88; P < 0.001). Individual Progressive Supranuclear Palsy Clinical Deficits Scale items correlated well with similar constructs in existing scales. Internal consistency (Cronbach's alpha: 0.75), inter-rater reliability (0.96), and test-retest stability (0.99) were acceptable. The PSP-CDS showed significant 12-month change (baseline, 8.6 ± 3.6; follow-up: 10.8 ± 3.6; annualized difference: 3.4 ± 3.4; n = 49; P < 0.0001). Sample sizes required per arm for a two-arm, 1-year follow-up therapeutic trial to detect 50% change in Progressive Supranuclear Palsy Clinical Deficits Scale progression was estimated to be 65 (two-sided, two-sample t test).
CONCLUSION
The Progressive Supranuclear Palsy Clinical Deficits Scale is a rapidly completed, clinimetrically sound scale for clinical care and research involving PSP. © 2020 International Parkinson and Movement Disorder Society.
Types de publication
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
650-661Investigateurs
Thomas Gasser
(T)
Andreas Hermann
(A)
Günter Höglinger
(G)
Matthias Höllerhage
(M)
Okka Kimmich
(O)
Thomas Klockgether
(T)
Johannes Levin
(J)
Gerrit Machetanz
(G)
Antje Osterrath
(A)
Carla Palleis
(C)
Johannes Prudlo
(J)
Annika Spottke
(A)
Daniela Berg
(D)
Katrin Bürk
(K)
Joseph Claßen
(J)
Carsten Eggers
(C)
Andrea Greuel
(A)
Max-Joseph Grimm
(MJ)
Lennard Hermann
(L)
Vassilena Iankova
(V)
Klaus Jahn
(K)
Wolfgang Jost
(W)
Martin Klietz
(M)
Andrea Kühn
(A)
Franz Marxreiter
(F)
Steffen Paschen
(S)
Monika Poetter-Nerger
(M)
Marie-Therese Preisl
(MT)
Lisa Prilop
(L)
Lars Tönges
(L)
Claudia Trenkwalder
(C)
Tobias Warnecke
(T)
Florian Wegner
(F)
Jürgen Winkler
(J)
Angelo Antonini
(A)
Kailash P P
(KP)
Adam L L
(AL)
Carlo Colosimo
(C)
Yaroslau Compta
(Y)
Jean-Christophe Corvol
(JC)
Lawrence I I
(LI)
Günter U Höglinger
(GU)
Anthony E E
(AE)
Irene Litvan
(I)
Huw R R
(HR)
Christer Nilsson
(C)
Alexander Pantelyat
(A)
Gesine Respondek
(G)
Maria Stamelou
(M)
Commentaires et corrections
Type : CommentIn
Type : CommentIn
Informations de copyright
© 2020 International Parkinson and Movement Disorder Society.
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