Disease severity and progression in progressive supranuclear palsy and multiple system atrophy

validation of the NNIPPS - PARKINSON PLUS SCALE

Christine A M Payan, François Viallet, Bernhard G Landwehrmeyer, Anne-Marie Bonnet, Michel Borg, Franck Durif, Lucette Lacomblez, Frédéric Bloch, Marc Verny, Jacques Fermanian, Yves Agid, Albert C Ludolph, Peter N Leigh, Gilbert Bensimon, on behalf of the NNIPPS Study Group, Carl Counsell

Research output: Contribution to journalArticle

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Abstract

Background

The Natural History and Neuroprotection in Parkinson Plus Syndromes (NNIPPS) study was a large phase III randomized placebo-controlled trial of riluzole in Progressive Supranuclear Palsy (PSP, n = 362) and Multiple System Atrophy (MSA, n = 398). To assess disease severity and progression, we constructed and validated a new clinical rating scale as an ancillary study.

Methods and Findings

Patients were assessed at entry and 6-montly for up to 3 years. Evaluation of the scale's psychometric properties included reliability (n = 116), validity (n = 760), and responsiveness (n = 642). Among the 85 items of the initial scale, factor analysis revealed 83 items contributing to 15 clinically relevant dimensions, including Activity of daily Living/Mobility, Axial bradykinesia, Limb bradykinesia, Rigidity, Oculomotor, Cerebellar, Bulbar/Pseudo-bulbar, Mental, Orthostatic, Urinary, Limb dystonia, Axial dystonia, Pyramidal, Myoclonus and Tremor. All but the Pyramidal dimension demonstrated good internal consistency (Cronbach α≥0.70). Inter-rater reliability was high for the total score (Intra-class coefficient = 0.94) and 9 dimensions (Intra-class coefficient = 0.80–0.93), and moderate (Intra-class coefficient = 0.54–0.77) for 6. Correlations of the total score with other clinical measures of severity were good (rho≥0.70). The total score was significantly and linearly related to survival (p<0.0001). Responsiveness expressed as the Standardized Response Mean was high for the total score slope of change (SRM = 1.10), though higher in PSP (SRM = 1.25) than in MSA (SRM = 1.0), indicating a more rapid progression of PSP. The slope of change was constant with increasing disease severity demonstrating good linearity of the scale throughout disease stages. Although MSA and PSP differed quantitatively on the total score at entry and on rate of progression, the relative contribution of clinical dimensions to overall severity and progression was similar.

Conclusions

The NNIPPS-PPS has suitable validity, is reliable and sensitive, and therefore is appropriate for use in clinical studies with PSP or MSA.
Original languageEnglish
Article numbere22293
Number of pages12
JournalPloS ONE
Volume6
Issue number8
DOIs
Publication statusPublished - 4 Aug 2011

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Progressive Supranuclear Palsy
Multiple System Atrophy
paralysis
atrophy
disease course
disease severity
Disease Progression
Hypokinesia
Riluzole
Factor analysis
Rigidity
rating scales
natural history
Dystonia
placebos
Tremor
clinical trials
Activities of Daily Living
Natural History
Psychometrics

Cite this

Disease severity and progression in progressive supranuclear palsy and multiple system atrophy : validation of the NNIPPS - PARKINSON PLUS SCALE. / Payan, Christine A M; Viallet, François; Landwehrmeyer, Bernhard G; Bonnet, Anne-Marie; Borg, Michel; Durif, Franck; Lacomblez, Lucette; Bloch, Frédéric; Verny, Marc; Fermanian, Jacques; Agid, Yves; Ludolph, Albert C; Leigh, Peter N; Bensimon, Gilbert; on behalf of the NNIPPS Study Group ; Counsell, Carl.

In: PloS ONE, Vol. 6, No. 8, e22293, 04.08.2011.

Research output: Contribution to journalArticle

Payan, CAM, Viallet, F, Landwehrmeyer, BG, Bonnet, A-M, Borg, M, Durif, F, Lacomblez, L, Bloch, F, Verny, M, Fermanian, J, Agid, Y, Ludolph, AC, Leigh, PN, Bensimon, G, on behalf of the NNIPPS Study Group & Counsell, C 2011, 'Disease severity and progression in progressive supranuclear palsy and multiple system atrophy: validation of the NNIPPS - PARKINSON PLUS SCALE', PloS ONE, vol. 6, no. 8, e22293. https://doi.org/10.1371/journal.pone.0022293
Payan, Christine A M ; Viallet, François ; Landwehrmeyer, Bernhard G ; Bonnet, Anne-Marie ; Borg, Michel ; Durif, Franck ; Lacomblez, Lucette ; Bloch, Frédéric ; Verny, Marc ; Fermanian, Jacques ; Agid, Yves ; Ludolph, Albert C ; Leigh, Peter N ; Bensimon, Gilbert ; on behalf of the NNIPPS Study Group ; Counsell, Carl. / Disease severity and progression in progressive supranuclear palsy and multiple system atrophy : validation of the NNIPPS - PARKINSON PLUS SCALE. In: PloS ONE. 2011 ; Vol. 6, No. 8.
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abstract = "BackgroundThe Natural History and Neuroprotection in Parkinson Plus Syndromes (NNIPPS) study was a large phase III randomized placebo-controlled trial of riluzole in Progressive Supranuclear Palsy (PSP, n = 362) and Multiple System Atrophy (MSA, n = 398). To assess disease severity and progression, we constructed and validated a new clinical rating scale as an ancillary study.Methods and FindingsPatients were assessed at entry and 6-montly for up to 3 years. Evaluation of the scale's psychometric properties included reliability (n = 116), validity (n = 760), and responsiveness (n = 642). Among the 85 items of the initial scale, factor analysis revealed 83 items contributing to 15 clinically relevant dimensions, including Activity of daily Living/Mobility, Axial bradykinesia, Limb bradykinesia, Rigidity, Oculomotor, Cerebellar, Bulbar/Pseudo-bulbar, Mental, Orthostatic, Urinary, Limb dystonia, Axial dystonia, Pyramidal, Myoclonus and Tremor. All but the Pyramidal dimension demonstrated good internal consistency (Cronbach α≥0.70). Inter-rater reliability was high for the total score (Intra-class coefficient = 0.94) and 9 dimensions (Intra-class coefficient = 0.80–0.93), and moderate (Intra-class coefficient = 0.54–0.77) for 6. Correlations of the total score with other clinical measures of severity were good (rho≥0.70). The total score was significantly and linearly related to survival (p<0.0001). Responsiveness expressed as the Standardized Response Mean was high for the total score slope of change (SRM = 1.10), though higher in PSP (SRM = 1.25) than in MSA (SRM = 1.0), indicating a more rapid progression of PSP. The slope of change was constant with increasing disease severity demonstrating good linearity of the scale throughout disease stages. Although MSA and PSP differed quantitatively on the total score at entry and on rate of progression, the relative contribution of clinical dimensions to overall severity and progression was similar.ConclusionsThe NNIPPS-PPS has suitable validity, is reliable and sensitive, and therefore is appropriate for use in clinical studies with PSP or MSA.",
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T2 - validation of the NNIPPS - PARKINSON PLUS SCALE

AU - Payan, Christine A M

AU - Viallet, François

AU - Landwehrmeyer, Bernhard G

AU - Bonnet, Anne-Marie

AU - Borg, Michel

AU - Durif, Franck

AU - Lacomblez, Lucette

AU - Bloch, Frédéric

AU - Verny, Marc

AU - Fermanian, Jacques

AU - Agid, Yves

AU - Ludolph, Albert C

AU - Leigh, Peter N

AU - Bensimon, Gilbert

AU - on behalf of the NNIPPS Study Group

AU - Counsell, Carl

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N2 - BackgroundThe Natural History and Neuroprotection in Parkinson Plus Syndromes (NNIPPS) study was a large phase III randomized placebo-controlled trial of riluzole in Progressive Supranuclear Palsy (PSP, n = 362) and Multiple System Atrophy (MSA, n = 398). To assess disease severity and progression, we constructed and validated a new clinical rating scale as an ancillary study.Methods and FindingsPatients were assessed at entry and 6-montly for up to 3 years. Evaluation of the scale's psychometric properties included reliability (n = 116), validity (n = 760), and responsiveness (n = 642). Among the 85 items of the initial scale, factor analysis revealed 83 items contributing to 15 clinically relevant dimensions, including Activity of daily Living/Mobility, Axial bradykinesia, Limb bradykinesia, Rigidity, Oculomotor, Cerebellar, Bulbar/Pseudo-bulbar, Mental, Orthostatic, Urinary, Limb dystonia, Axial dystonia, Pyramidal, Myoclonus and Tremor. All but the Pyramidal dimension demonstrated good internal consistency (Cronbach α≥0.70). Inter-rater reliability was high for the total score (Intra-class coefficient = 0.94) and 9 dimensions (Intra-class coefficient = 0.80–0.93), and moderate (Intra-class coefficient = 0.54–0.77) for 6. Correlations of the total score with other clinical measures of severity were good (rho≥0.70). The total score was significantly and linearly related to survival (p<0.0001). Responsiveness expressed as the Standardized Response Mean was high for the total score slope of change (SRM = 1.10), though higher in PSP (SRM = 1.25) than in MSA (SRM = 1.0), indicating a more rapid progression of PSP. The slope of change was constant with increasing disease severity demonstrating good linearity of the scale throughout disease stages. Although MSA and PSP differed quantitatively on the total score at entry and on rate of progression, the relative contribution of clinical dimensions to overall severity and progression was similar.ConclusionsThe NNIPPS-PPS has suitable validity, is reliable and sensitive, and therefore is appropriate for use in clinical studies with PSP or MSA.

AB - BackgroundThe Natural History and Neuroprotection in Parkinson Plus Syndromes (NNIPPS) study was a large phase III randomized placebo-controlled trial of riluzole in Progressive Supranuclear Palsy (PSP, n = 362) and Multiple System Atrophy (MSA, n = 398). To assess disease severity and progression, we constructed and validated a new clinical rating scale as an ancillary study.Methods and FindingsPatients were assessed at entry and 6-montly for up to 3 years. Evaluation of the scale's psychometric properties included reliability (n = 116), validity (n = 760), and responsiveness (n = 642). Among the 85 items of the initial scale, factor analysis revealed 83 items contributing to 15 clinically relevant dimensions, including Activity of daily Living/Mobility, Axial bradykinesia, Limb bradykinesia, Rigidity, Oculomotor, Cerebellar, Bulbar/Pseudo-bulbar, Mental, Orthostatic, Urinary, Limb dystonia, Axial dystonia, Pyramidal, Myoclonus and Tremor. All but the Pyramidal dimension demonstrated good internal consistency (Cronbach α≥0.70). Inter-rater reliability was high for the total score (Intra-class coefficient = 0.94) and 9 dimensions (Intra-class coefficient = 0.80–0.93), and moderate (Intra-class coefficient = 0.54–0.77) for 6. Correlations of the total score with other clinical measures of severity were good (rho≥0.70). The total score was significantly and linearly related to survival (p<0.0001). Responsiveness expressed as the Standardized Response Mean was high for the total score slope of change (SRM = 1.10), though higher in PSP (SRM = 1.25) than in MSA (SRM = 1.0), indicating a more rapid progression of PSP. The slope of change was constant with increasing disease severity demonstrating good linearity of the scale throughout disease stages. Although MSA and PSP differed quantitatively on the total score at entry and on rate of progression, the relative contribution of clinical dimensions to overall severity and progression was similar.ConclusionsThe NNIPPS-PPS has suitable validity, is reliable and sensitive, and therefore is appropriate for use in clinical studies with PSP or MSA.

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