Long-term progression of clinician-reported and gait performance outcomes in hereditary spastic paraplegias.
clinical outcome assessment
clinician-reported outcomes
gait analysis
hereditary spastic paraplegias
performance outcomes
Journal
Frontiers in neuroscience
ISSN: 1662-4548
Titre abrégé: Front Neurosci
Pays: Switzerland
ID NLM: 101478481
Informations de publication
Date de publication:
2023
2023
Historique:
received:
21
05
2023
accepted:
22
08
2023
medline:
9
10
2023
pubmed:
9
10
2023
entrez:
9
10
2023
Statut:
epublish
Résumé
Hereditary spastic paraplegias (HSPs) are a heterogeneous group of neurodegenerative diseases in which little is known about the most appropriate clinical outcome assessments (COAs) to capture disease progression. The objective of this study was to prospectively determine disease progression after 4.5 years of follow-up with different clinician-reported (ClinRO) and gait performance outcomes (PerFOs). Twenty-six HSP patients (15 SPG4, 5 SPG7, 4 SPG5, 2 SPG3A) participated in this single-center cohort study in which the ClinRO: Spastic Paraplegia Rating Scale; and the PerFOs: 10-meters walking test and timed-up and go (TUG), at self-selected and maximal walking speeds; Locomotor Rehabilitation Index; and 6-min walking test were performed at baseline and after 1.5 (18 patients) and 4.5 (13 patients) years. In the 3-year interval between the second and third assessments, significant progressions were only found in PerFOs, while in the overall 4.5 years of follow-up, both PerFOs and ClinROs presented significant progressions. The progression slopes of COAs modeled according to the disease duration allowed the estimation of the annual progression of the outcomes and sample size estimations for future clinical trials of interventions with different effect sizes. TUG at maximal walking speed was the only COA capable of differentiating subjects with a worse compared to a stable/better impression of change and would require the smallest sample size if chosen as the primary endpoint of a clinical trial. These findings indicate that both performance and clinician-reported outcomes can capture long-term progression of HSPs, with some PerFOs presenting greater sensitivity to change. The presented data are paramount for planning future disease-modifying and symptomatic therapy trials for this currently untreatable group of diseases.
Identifiants
pubmed: 37811319
doi: 10.3389/fnins.2023.1226479
pmc: PMC10556702
doi:
Types de publication
Journal Article
Langues
eng
Pagination
1226479Informations de copyright
Copyright © 2023 Cubillos Arcila, Dariva Machado, Martins, Leotti, Schüle, Peyré-Tartaruga and Saute.
Déclaration de conflit d'intérêts
The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
Références
J Neurol. 2015 May;262(5):1285-8
pubmed: 25808501
Cerebellum. 2022 Jun;21(3):350-357
pubmed: 34244902
J Am Geriatr Soc. 1991 Feb;39(2):142-8
pubmed: 1991946
Brain. 1996 Oct;119 ( Pt 5):1487-96
pubmed: 8931574
Muscle Nerve. 2013 Sep;48(3):357-68
pubmed: 23674289
Neurology. 2006 Aug 8;67(3):430-4
pubmed: 16894103
Lancet. 1983 May 21;1(8334):1151-5
pubmed: 6133167
Brain. 2017 Dec 1;140(12):3112-3127
pubmed: 29126212
Brain. 2018 Dec 1;141(12):3331-3342
pubmed: 30476002
Neuropathol Appl Neurobiol. 2004 Dec;30(6):576-84
pubmed: 15540998
Mov Disord. 2021 Jul;36(7):1654-1663
pubmed: 33595142
Age Ageing. 2003 May;32(3):315-20
pubmed: 12720619
BMC Geriatr. 2014 Feb 01;14:14
pubmed: 24484314
Neurology. 2022 Jun 6;:
pubmed: 35667840
Front Mol Biosci. 2021 Nov 26;8:690899
pubmed: 34901147
Neuromuscul Disord. 2015 Jan;25(1):5-13
pubmed: 25497400
Lancet Neurol. 2019 Dec;18(12):1136-1146
pubmed: 31377012
Nat Nanotechnol. 2021 Jun;16(6):630-643
pubmed: 34059811
Clin Genet. 2023 May;103(5):580-584
pubmed: 36537231
Lancet. 2017 Sep 23;390(10101):1489-1498
pubmed: 28728956
Control Clin Trials. 1989 Dec;10(4):407-15
pubmed: 2691207
Am J Respir Crit Care Med. 2002 Jul 1;166(1):111-7
pubmed: 12091180
N Engl J Med. 2010 Apr 15;362(15):1396-406
pubmed: 20393176
Neurol Genet. 2023 Jan 10;9(1):e200052
pubmed: 36636734
Front Neurosci. 2020 Feb 21;14:111
pubmed: 32153352
Ann Neurol. 2016 Apr;79(4):646-58
pubmed: 26856398
Psychiatry (Edgmont). 2007 Jul;4(7):28-37
pubmed: 20526405
J Geriatr Phys Ther. 2013 Apr-Jun;36(2):74-7
pubmed: 22874880
Neurol Genet. 2022 Mar 30;8(2):e664
pubmed: 35372684
J Rehabil Med. 2015 Feb;47(2):147-53
pubmed: 25325386
Sci Rep. 2021 Nov 15;11(1):22248
pubmed: 34782662
Clin Neurol Neurosurg. 2021 Oct;209:106888
pubmed: 34455170
Eur J Paediatr Neurol. 2019 Jan;23(1):165-170
pubmed: 30449663
Arq Neuropsiquiatr. 2016 Jun;74(6):489-94
pubmed: 27332075