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Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature

Respiratory muscle strength is a proven predictor of long-term outcome of neuromuscular disease (NMD), including amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and spinal muscular atrophy. Maximal inspiratory pressure (MIP), a sensitive measure of respiratory muscle strength, one of sev...

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Autores principales: Schoser, Benedikt, Fong, Edward, Geberhiwot, Tarekegn, Hughes, Derralynn, Kissel, John T., Madathil, Shyam C., Orlikowski, David, Polkey, Michael I., Roberts, Mark, Tiddens, Harm A. W. M., Young, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5353799/
https://www.ncbi.nlm.nih.gov/pubmed/28302142
http://dx.doi.org/10.1186/s13023-017-0598-0
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author Schoser, Benedikt
Fong, Edward
Geberhiwot, Tarekegn
Hughes, Derralynn
Kissel, John T.
Madathil, Shyam C.
Orlikowski, David
Polkey, Michael I.
Roberts, Mark
Tiddens, Harm A. W. M.
Young, Peter
author_facet Schoser, Benedikt
Fong, Edward
Geberhiwot, Tarekegn
Hughes, Derralynn
Kissel, John T.
Madathil, Shyam C.
Orlikowski, David
Polkey, Michael I.
Roberts, Mark
Tiddens, Harm A. W. M.
Young, Peter
author_sort Schoser, Benedikt
collection PubMed
description Respiratory muscle strength is a proven predictor of long-term outcome of neuromuscular disease (NMD), including amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and spinal muscular atrophy. Maximal inspiratory pressure (MIP), a sensitive measure of respiratory muscle strength, one of several useful tests of respiratory muscle strength, is gaining interest as a therapeutic clinical trial endpoint for NMD. In this comprehensive review we investigate the use of MIP as a measure of respiratory muscle strength in clinical trials of therapeutics targeting respiratory muscle, examine the correlation of MIP with survival, quality of life, and other measures of pulmonary function, and outline the role of MIP as a clinically significantly meaningful outcome measure. Our analysis supports the utility of MIP for the early evaluation of respiratory muscle strength, especially of the diaphragm, in patients with NMD and as a surrogate endpoint in clinical trials of therapies for NMD.
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spelling pubmed-53537992017-03-22 Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature Schoser, Benedikt Fong, Edward Geberhiwot, Tarekegn Hughes, Derralynn Kissel, John T. Madathil, Shyam C. Orlikowski, David Polkey, Michael I. Roberts, Mark Tiddens, Harm A. W. M. Young, Peter Orphanet J Rare Dis Review Respiratory muscle strength is a proven predictor of long-term outcome of neuromuscular disease (NMD), including amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and spinal muscular atrophy. Maximal inspiratory pressure (MIP), a sensitive measure of respiratory muscle strength, one of several useful tests of respiratory muscle strength, is gaining interest as a therapeutic clinical trial endpoint for NMD. In this comprehensive review we investigate the use of MIP as a measure of respiratory muscle strength in clinical trials of therapeutics targeting respiratory muscle, examine the correlation of MIP with survival, quality of life, and other measures of pulmonary function, and outline the role of MIP as a clinically significantly meaningful outcome measure. Our analysis supports the utility of MIP for the early evaluation of respiratory muscle strength, especially of the diaphragm, in patients with NMD and as a surrogate endpoint in clinical trials of therapies for NMD. BioMed Central 2017-03-16 /pmc/articles/PMC5353799/ /pubmed/28302142 http://dx.doi.org/10.1186/s13023-017-0598-0 Text en © The Author(s). 2017 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Review
Schoser, Benedikt
Fong, Edward
Geberhiwot, Tarekegn
Hughes, Derralynn
Kissel, John T.
Madathil, Shyam C.
Orlikowski, David
Polkey, Michael I.
Roberts, Mark
Tiddens, Harm A. W. M.
Young, Peter
Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
title Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
title_full Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
title_fullStr Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
title_full_unstemmed Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
title_short Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
title_sort maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5353799/
https://www.ncbi.nlm.nih.gov/pubmed/28302142
http://dx.doi.org/10.1186/s13023-017-0598-0
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