Primary lateral sclerosis (PLS) functional rating scale: PLS-specific clinimetric scale

the PLSFRS study group

doi:10.1002/mus.26765

Primary lateral sclerosis (PLS) functional rating scale: PLS-specific clinimetric scale

the PLSFRS study group

Neurology

Research output: Contribution to journal › Article › peer-review

17 Scopus citations

Abstract

Introduction: Our research aim was to develop a novel clinimetric scale sensitive enough to detect disease progression in primary lateral sclerosis (PLS). Methods: A prototype of the PLS Functional Rating Scale (PLSFRS) was generated. Seventy-seven participants with PLS were enrolled and evaluated at 21 sites that comprised the PLSFRS study group. Participants were assessed using the PLSFRS, Neuro-Quality of Life (QoL), Schwab-England Activities of Daily Living (ADL), and the Clinical Global Impression of Change scales. Participants completed telephone assessments at 12, 24, and 48 weeks after enrollment. Results: The PLSFRS demonstrated internal consistency as well as intrarater, interrater, telephone test-retest reliability, and construct validity. Significant changes in disease progression were detected at 6 and 12 months; changes measured by the PLSFRS vs the ALSFRS-R were significantly higher. Discussion: The PLSFRS is a valid tool to assess the natural history of PLS in a shorter study period.

Original language	English (US)
Pages (from-to)	163-172
Number of pages	10
Journal	Muscle and Nerve
Volume	61
Issue number	2
DOIs	https://doi.org/10.1002/mus.26765
State	Published - Feb 1 2020

Bibliographical note

Funding Information:
H.M. has received funding from the NIH, the CDC, the MDA, the ALS Association, the MNDA, the SPF, Tsumura, Cytokinetics, Mitsubishi‐Tanabe; funding for clinical trials from Cytokinetics; and has been on the advisory boards for Mitsubishi‐Tanabe, Biohaven, and Daihippon‐Sumitomo. Z.S. has served as a consultant for Biohaven; received research support from Biohaven, Mallinckrodt, and Sanofi; and receives a stipend from Wiley as Editor and Chief for Muscle & Nerve . S.P. has received research grants from Target ALS, the Salah Foundation, the Spastic Paraplegia Foundation, the ALS Association, ALS Finding a Cure, the American Academy of Neurology, Amylyx Therapeutics, and Revalesio Corporation; advisory board fees from Roche; and honoraria from Oakstone Publishing and McGraw‐Hill Education. M.K.F. has received support from the intramural program of NINDS, NIH. E.P.P. has received clinical trial and research funding from the NIH/CDC and the ALS Association; consulting fees from Avanir Pharmaceuticals, Biohaven Pharmaceuticals, Cytokinetics, ITF Pharma, MT Pharma America, and Otsuka America. J.A.M.F. has received support for clinical trials from Mallinckrodt Pharmaceuticals. D.H. has received speaker/consultant fees from Biohaven Pharmaceuticals, and research grant support from Cytokinetics, Mallinckrodt Pharmaceuticals, Orion Pharma, Amylyx Therapeutics, Revance Therapeutics, the MDA, the ALS Association, and the Neurologix Foundation. C.N.F. has received research funding from the Department of Veterans Affairs Office of Research and Development (IK2CX001595–02), Mallinckrodt ARD, and Amylyx Pharmaceuticals. B.O. has received grants from Target ALS, NINDS, Biogen, Genentech, Cytokinetics, Orion, Eisai, and consulting/advisory board fees from Mitsubishi Tanabe, Medicinova, and Biohaven. T.H.‐P. has received consultant and advisory board fees from Mitsubishi Tanabe Pharma, Cytokinetics, ITF, and Biohaven. N.M. has received consultant/advisory board fees from Biogen, Clene Nanomedicine, Apellis, Brainstorm, Orion Pharma; DSMC from Orphazyme; and grant support from the ALS Association, the Department of Defense ALSRP, the NINDS, and the Maryland Stem Cell Research Fund. N.J. has received grants from the NIH, NIDDR; clinical trials funding from Novartis, Acceleron, Mitsubishi‐Tanabe; and speaker/consultant fees from Biogen. D.W. has been a consultant for MT Pharma and Sarepta Therapeutics. The remaining authors declare no conflicts of interest.

Funding Information:
information Spastic Paraplegia Foundation (research grant); David Marren and familyThe authors are grateful to the participants and their families for being part of this clinical study. We thank the study funders for their contributions to the development of the PLSFRS scale. Georgia Christodoulou, MA, University of Southern California, assisted with the preparation of the manuscript and editing.

Publisher Copyright:
© 2019 Wiley Periodicals, Inc.

Keywords

MND
PLS
PLSFRS
PUMND
clinical trials
clinimetric scale

Access

10.1002/mus.26765

OpenUrl availability

Full text

Cite this

@article{7a17d0d4c7494f4b90baf43c88ad7202,

title = "Primary lateral sclerosis (PLS) functional rating scale: PLS-specific clinimetric scale",

abstract = "Introduction: Our research aim was to develop a novel clinimetric scale sensitive enough to detect disease progression in primary lateral sclerosis (PLS). Methods: A prototype of the PLS Functional Rating Scale (PLSFRS) was generated. Seventy-seven participants with PLS were enrolled and evaluated at 21 sites that comprised the PLSFRS study group. Participants were assessed using the PLSFRS, Neuro-Quality of Life (QoL), Schwab-England Activities of Daily Living (ADL), and the Clinical Global Impression of Change scales. Participants completed telephone assessments at 12, 24, and 48 weeks after enrollment. Results: The PLSFRS demonstrated internal consistency as well as intrarater, interrater, telephone test-retest reliability, and construct validity. Significant changes in disease progression were detected at 6 and 12 months; changes measured by the PLSFRS vs the ALSFRS-R were significantly higher. Discussion: The PLSFRS is a valid tool to assess the natural history of PLS in a shorter study period.",

keywords = "MND, PLS, PLSFRS, PUMND, clinical trials, clinimetric scale",

author = "{the PLSFRS study group} and Hiroshi Mitsumoto and Codruta Chiuzan and Madison Gilmore and Yuan Zhang and Zachary Simmons and Sabrina Paganoni and Kisanuki, {Yasushi Y.} and Lorne Zinman and Omar Jawdat and Eric Sorenson and Floeter, {Mary Kay} and Pioro, {Erik P.} and {Fernandes Filho}, {J. Americo M.} and Daragh Heitzman and Fournier, {Christina Nicole} and Bjorn Oskarsson and Terry Heiman-Patterson and Nicholas Maragakis and Nanette Joyce and Ghazala Hayat and Sharon Nations and Stephen Scelsa and David Walk and Lauren Elman and Jonathan Hupf and Brittany McHale",

note = "Funding Information: H.M. has received funding from the NIH, the CDC, the MDA, the ALS Association, the MNDA, the SPF, Tsumura, Cytokinetics, Mitsubishi‐Tanabe; funding for clinical trials from Cytokinetics; and has been on the advisory boards for Mitsubishi‐Tanabe, Biohaven, and Daihippon‐Sumitomo. Z.S. has served as a consultant for Biohaven; received research support from Biohaven, Mallinckrodt, and Sanofi; and receives a stipend from Wiley as Editor and Chief for Muscle & Nerve . S.P. has received research grants from Target ALS, the Salah Foundation, the Spastic Paraplegia Foundation, the ALS Association, ALS Finding a Cure, the American Academy of Neurology, Amylyx Therapeutics, and Revalesio Corporation; advisory board fees from Roche; and honoraria from Oakstone Publishing and McGraw‐Hill Education. M.K.F. has received support from the intramural program of NINDS, NIH. E.P.P. has received clinical trial and research funding from the NIH/CDC and the ALS Association; consulting fees from Avanir Pharmaceuticals, Biohaven Pharmaceuticals, Cytokinetics, ITF Pharma, MT Pharma America, and Otsuka America. J.A.M.F. has received support for clinical trials from Mallinckrodt Pharmaceuticals. D.H. has received speaker/consultant fees from Biohaven Pharmaceuticals, and research grant support from Cytokinetics, Mallinckrodt Pharmaceuticals, Orion Pharma, Amylyx Therapeutics, Revance Therapeutics, the MDA, the ALS Association, and the Neurologix Foundation. C.N.F. has received research funding from the Department of Veterans Affairs Office of Research and Development (IK2CX001595–02), Mallinckrodt ARD, and Amylyx Pharmaceuticals. B.O. has received grants from Target ALS, NINDS, Biogen, Genentech, Cytokinetics, Orion, Eisai, and consulting/advisory board fees from Mitsubishi Tanabe, Medicinova, and Biohaven. T.H.‐P. has received consultant and advisory board fees from Mitsubishi Tanabe Pharma, Cytokinetics, ITF, and Biohaven. N.M. has received consultant/advisory board fees from Biogen, Clene Nanomedicine, Apellis, Brainstorm, Orion Pharma; DSMC from Orphazyme; and grant support from the ALS Association, the Department of Defense ALSRP, the NINDS, and the Maryland Stem Cell Research Fund. N.J. has received grants from the NIH, NIDDR; clinical trials funding from Novartis, Acceleron, Mitsubishi‐Tanabe; and speaker/consultant fees from Biogen. D.W. has been a consultant for MT Pharma and Sarepta Therapeutics. The remaining authors declare no conflicts of interest. Funding Information: information Spastic Paraplegia Foundation (research grant); David Marren and familyThe authors are grateful to the participants and their families for being part of this clinical study. We thank the study funders for their contributions to the development of the PLSFRS scale. Georgia Christodoulou, MA, University of Southern California, assisted with the preparation of the manuscript and editing. Publisher Copyright: {\textcopyright} 2019 Wiley Periodicals, Inc.",

year = "2020",

month = feb,

day = "1",

doi = "10.1002/mus.26765",

language = "English (US)",

volume = "61",

pages = "163--172",

journal = "Muscle and Nerve",

issn = "0148-639X",

publisher = "John Wiley and Sons Inc.",

number = "2",

}

TY - JOUR

T1 - Primary lateral sclerosis (PLS) functional rating scale

T2 - PLS-specific clinimetric scale

AU - the PLSFRS study group

AU - Mitsumoto, Hiroshi

AU - Chiuzan, Codruta

AU - Gilmore, Madison

AU - Zhang, Yuan

AU - Simmons, Zachary

AU - Paganoni, Sabrina

AU - Kisanuki, Yasushi Y.

AU - Zinman, Lorne

AU - Jawdat, Omar

AU - Sorenson, Eric

AU - Floeter, Mary Kay

AU - Pioro, Erik P.

AU - Fernandes Filho, J. Americo M.

AU - Heitzman, Daragh

AU - Fournier, Christina Nicole

AU - Oskarsson, Bjorn

AU - Heiman-Patterson, Terry

AU - Maragakis, Nicholas

AU - Joyce, Nanette

AU - Hayat, Ghazala

AU - Nations, Sharon

AU - Scelsa, Stephen

AU - Walk, David

AU - Elman, Lauren

AU - Hupf, Jonathan

AU - McHale, Brittany

N1 - Funding Information: H.M. has received funding from the NIH, the CDC, the MDA, the ALS Association, the MNDA, the SPF, Tsumura, Cytokinetics, Mitsubishi‐Tanabe; funding for clinical trials from Cytokinetics; and has been on the advisory boards for Mitsubishi‐Tanabe, Biohaven, and Daihippon‐Sumitomo. Z.S. has served as a consultant for Biohaven; received research support from Biohaven, Mallinckrodt, and Sanofi; and receives a stipend from Wiley as Editor and Chief for Muscle & Nerve . S.P. has received research grants from Target ALS, the Salah Foundation, the Spastic Paraplegia Foundation, the ALS Association, ALS Finding a Cure, the American Academy of Neurology, Amylyx Therapeutics, and Revalesio Corporation; advisory board fees from Roche; and honoraria from Oakstone Publishing and McGraw‐Hill Education. M.K.F. has received support from the intramural program of NINDS, NIH. E.P.P. has received clinical trial and research funding from the NIH/CDC and the ALS Association; consulting fees from Avanir Pharmaceuticals, Biohaven Pharmaceuticals, Cytokinetics, ITF Pharma, MT Pharma America, and Otsuka America. J.A.M.F. has received support for clinical trials from Mallinckrodt Pharmaceuticals. D.H. has received speaker/consultant fees from Biohaven Pharmaceuticals, and research grant support from Cytokinetics, Mallinckrodt Pharmaceuticals, Orion Pharma, Amylyx Therapeutics, Revance Therapeutics, the MDA, the ALS Association, and the Neurologix Foundation. C.N.F. has received research funding from the Department of Veterans Affairs Office of Research and Development (IK2CX001595–02), Mallinckrodt ARD, and Amylyx Pharmaceuticals. B.O. has received grants from Target ALS, NINDS, Biogen, Genentech, Cytokinetics, Orion, Eisai, and consulting/advisory board fees from Mitsubishi Tanabe, Medicinova, and Biohaven. T.H.‐P. has received consultant and advisory board fees from Mitsubishi Tanabe Pharma, Cytokinetics, ITF, and Biohaven. N.M. has received consultant/advisory board fees from Biogen, Clene Nanomedicine, Apellis, Brainstorm, Orion Pharma; DSMC from Orphazyme; and grant support from the ALS Association, the Department of Defense ALSRP, the NINDS, and the Maryland Stem Cell Research Fund. N.J. has received grants from the NIH, NIDDR; clinical trials funding from Novartis, Acceleron, Mitsubishi‐Tanabe; and speaker/consultant fees from Biogen. D.W. has been a consultant for MT Pharma and Sarepta Therapeutics. The remaining authors declare no conflicts of interest. Funding Information: information Spastic Paraplegia Foundation (research grant); David Marren and familyThe authors are grateful to the participants and their families for being part of this clinical study. We thank the study funders for their contributions to the development of the PLSFRS scale. Georgia Christodoulou, MA, University of Southern California, assisted with the preparation of the manuscript and editing. Publisher Copyright: © 2019 Wiley Periodicals, Inc.

PY - 2020/2/1

Y1 - 2020/2/1

N2 - Introduction: Our research aim was to develop a novel clinimetric scale sensitive enough to detect disease progression in primary lateral sclerosis (PLS). Methods: A prototype of the PLS Functional Rating Scale (PLSFRS) was generated. Seventy-seven participants with PLS were enrolled and evaluated at 21 sites that comprised the PLSFRS study group. Participants were assessed using the PLSFRS, Neuro-Quality of Life (QoL), Schwab-England Activities of Daily Living (ADL), and the Clinical Global Impression of Change scales. Participants completed telephone assessments at 12, 24, and 48 weeks after enrollment. Results: The PLSFRS demonstrated internal consistency as well as intrarater, interrater, telephone test-retest reliability, and construct validity. Significant changes in disease progression were detected at 6 and 12 months; changes measured by the PLSFRS vs the ALSFRS-R were significantly higher. Discussion: The PLSFRS is a valid tool to assess the natural history of PLS in a shorter study period.

AB - Introduction: Our research aim was to develop a novel clinimetric scale sensitive enough to detect disease progression in primary lateral sclerosis (PLS). Methods: A prototype of the PLS Functional Rating Scale (PLSFRS) was generated. Seventy-seven participants with PLS were enrolled and evaluated at 21 sites that comprised the PLSFRS study group. Participants were assessed using the PLSFRS, Neuro-Quality of Life (QoL), Schwab-England Activities of Daily Living (ADL), and the Clinical Global Impression of Change scales. Participants completed telephone assessments at 12, 24, and 48 weeks after enrollment. Results: The PLSFRS demonstrated internal consistency as well as intrarater, interrater, telephone test-retest reliability, and construct validity. Significant changes in disease progression were detected at 6 and 12 months; changes measured by the PLSFRS vs the ALSFRS-R were significantly higher. Discussion: The PLSFRS is a valid tool to assess the natural history of PLS in a shorter study period.

KW - MND

KW - PLS

KW - PLSFRS

KW - PUMND

KW - clinical trials

KW - clinimetric scale

UR - http://www.scopus.com/inward/record.url?scp=85077028533&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85077028533&partnerID=8YFLogxK

U2 - 10.1002/mus.26765

DO - 10.1002/mus.26765

M3 - Article

C2 - 31758557

AN - SCOPUS:85077028533

SN - 0148-639X

VL - 61

SP - 163

EP - 172

JO - Muscle and Nerve

JF - Muscle and Nerve

IS - 2

ER -

Primary lateral sclerosis (PLS) functional rating scale: PLS-specific clinimetric scale

Abstract

Bibliographical note

Keywords

Access

OpenUrl availability

Other files and links

Fingerprint

Cite this