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 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.
|Keywords||Endpoint, Maximum inspiratory pressure, Neuromuscular disease, Pulmonary function testing, Respiratory failure, Spirometry, Survival|
|Persistent URL||dx.doi.org/10.1186/s13023-017-0598-0, hdl.handle.net/1765/98701|
|Journal||Orphanet journal of rare diseases|
Schoser, B, Fong, E. (Edward), Geberhiwot, T. (Tarekegn), Hughes, D. (Derralynn), Kissel, J.T. (John T.), Madathil, S.C. (Shyam C.), … Young, P. (Peter). (2017). Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: A comprehensive review of the literature. Orphanet journal of rare diseases (Vol. 12). doi:10.1186/s13023-017-0598-0