Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
File(s)
Author(s)
Type
Journal Article
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.
Date Issued
2017-03-16
Date Acceptance
2017-02-23
Citation
ORPHANET JOURNAL OF RARE DISEASES, 2017, 12
ISSN
1750-1172
Publisher
BIOMED CENTRAL LTD
Journal / Book Title
ORPHANET JOURNAL OF RARE DISEASES
Volume
12
Copyright Statement
© 2017 The Author(s). Open Access. 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.
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.
Identifier
http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000397667100001&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
Subjects
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
1199 Other Medical And Health Sciences
Publication Status
Published
Article Number
ARTN 52