Schoser, B;
Fong, E;
Geberhiwot, T;
Hughes, D;
Kissel, JT;
Madathil, SC;
Orlikowski, D;
... Young, P; + view all
(2017)
Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature.
[Review].
Orphanet Journal of Rare Diseases
, 12
, Article 52. 10.1186/s13023-017-0598-0.
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Abstract
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.
| Type: | Article |
|---|---|
| Title: | Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1186/s13023-017-0598-0 |
| Publisher version: | http://doi.org/10.1186/s13023-017-0598-0 |
| Language: | English |
| Additional information: | © The Author(s). 2017. This 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. |
| Keywords: | Science & Technology, Life Sciences & Biomedicine, Genetics & Heredity, Medicine, Research & Experimental, Research & Experimental Medicine, Maximum inspiratory pressure, Neuromuscular disease, Respiratory failure, Endpoint, Survival, Pulmonary function testing, Spirometry, AMYOTROPHIC-LATERAL-SCLEROSIS, OBSTRUCTIVE PULMONARY-DISEASE, RESPIRATORY MUSCLE STRENGTH, DUCHENNE MUSCULAR-DYSTROPHY, CHRONIC HEART-FAILURE, SNIFF NASAL PRESSURE, GUILLAIN-BARRE-SYNDROME, NONINVASIVE VENTILATION, VITAL CAPACITY, POMPE DISEASE |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/1553577 |
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