Parent Project Muscular Dystrophy (PPMD), the largest US nonprofit group focused on Duchenne muscular dystrophy (DMD), held a Pulmonary Outcomes Workshop from April 14 to 15, 2016. Physicians and industry experts discussed pulmonary outcome measures used in current DMD clinical trials, how those measures might be improved, and their use in furthering drug development.
As DMD progresses, respiratory muscles progressively weaken leading to changes in pulmonary function, making the process of breathing and coughing increasingly difficult for patients. In light of these organ-specific effects, clinical trials that aim to improve pulmonary function in DMD apply several measures to demonstrate changes in DMD pulmonary function. Such measures include changes in inspiratory strength, as measured by maximal inspiratory pressure (MIP), changes in expiratory strength, as measured by maximal expiratory pressure (MEP) and peak expiratory pressure (PEP) and changes in the volume of air that the lungs can hold, as measured by forced vital capacity (FVC), forced vital capacity when compared with normal (FVC%) and peak expiratory flow (PEF).
PPMD believes it is time to reassess these measures, and the goals of the Pulmonary Outcomes Workshop include answering questions regarding the current measures’ clinical meaningfulness and possible limitations. Experts will discuss if there is a need to implement new measures in light of new knowledge, and how such knowledge may improve and facilitate drug development. The forum also serves as an educational platform and an opportunity to discuss protocols and data analyses used in clinical trials.
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