The Importance of Every Single Breath—PPMD to Host Pulmonary Outcomes Meeting

We are well aware that changes in pulmonary function occur as Duchenne progresses. In Duchenne, respiratory muscles (including the diaphragm) weaken and it becomes more difficult to take full breaths in, fully expand the lungs, hold full breaths, breath full breaths out, and to cough effectively. And we know that these changes occur first during sleep.

Measures of Pulmonary Function

There are many tests used to demonstrate changes in respiratory function: 

  • Some show changes in inspiratory strength: 
    Strength of the muscles while breathing in/ MIP-maximal inspiratory pressure. 
  • Some show changes in expiratory strength:
    Strength of muscles while breathing out/ MEP- maximal expiratory pressure, PEP - peak expiratory pressure. 
  • Some show changes in the amount of air that the lungs can hold:
    FVC or forced vital capacity; FVC% - forced vital capacity when compared with normal; PEF - peak expiratory flow. 


Clinical trials that hope to improve pulmonary function include many of these measures of pulmonary function as “pulmonary outcome measures.” But using pulmonary outcome measures creates a whole series of questions we need to clarify.

Addressing the Issues

As such, PPMD and an expert steering committee are facing these questions head on. Thanks in part to support from Santhera Pharmaceuticals, Catabasis Pharmaceuticals, and Pfizer, PPMD will gather experts in Duchenne pulmonary care and evaluation, to discuss these questions in April. 

PPMD 2016 Pulmonary Outcomes Meeting Leadership


  • Jonathan Finder, MD, Children’s Hospital of Pittsburg
  • Craig McDonald, MD, UC Davis Medical Center


Steering committee:

  • Thomas Meier, PhD, Santhera Pharmaceuticals
  • Jodi Wolff, PhD, Santhera Pharmaceuticals
  • Daniel Sheehan, MD, PhD, Buffalo General Medical Center
  • Anne Connolly, MD, St. Louis Children’s Hospital
  • Tina Duong, MPT, Lucille Packard Children’s Hospital Stanford
  • David Birnkrant, MD, Metro Health
  • Kathi Kinnett, MSN, CNP, PPMD


Asking the Right Questions


Some of the questions we will be exploring at our meeting in April include:

  • What are the best measurements of pulmonary function? 
  • What measurements might be the most sensitive to changes in pulmonary function? 
  • What measurements might best show that a drug is improving pulmonary function (or not)? 
  • Do changes in pulmonary function correlate with changes in skeletal muscle, as shown by ambulation, muscle strength, or muscle function?  
  • Do improvements in these measures correlate with decreased infections and increased length of life? 


These questions have been important in Duchenne care, but are becoming increasingly more important as industry seeks to show drug efficacy by demonstrating improvements in pulmonary function. 

Our Goals


The goals of this “Pulmonary Outcomes Meeting” include:

  • Assess currently utilized pulmonary outcome measures, their clinical meaningfulness, possible limitations, and the need to implement novel pulmonary measures
  • Identify how this knowledge can further drug development
  • Utilize this forum to educate ourselves, the greater Duchenne community, and the FDA on the need for reliable, responsive, and meaningful pulmonary outcome measures

We will look at the pulmonary function measures that pulmonary care providers and industry are using now.  Several natural history data sets have collected pulmonary function measures, and we will discuss how those measures have shown changes throughout the natural history of Duchenne.  We will then look at the how measures of pulmonary function have correlated with other areas of patient evaluation (skeletal muscle strength and function, incidence of upper respiratory infections/pneumonia/antibiotic use).  We will discuss how pulmonary data is analyzed, and discuss whether data sets can be compared and whether standardized protocols might enhance the reliability of pulmonary outcome measures. 


We have invited members of the FDA Divisions of Neurology and Pulmonology to give their views on what they may feel to be appropriate and meaningful pulmonary outcome measures. Pulmonary outcomes collected from a variety of trials will be discussed, as well as novel assessments that might be implemented in future trials (such as home based pulmonary monitoring, diaphragm ultrasound, etc.) and future directions that pulmonary monitoring might lead.  A report of this meeting will be published.


It is our hope that this meeting will help to clarify much of the confusion around pulmonary outcome measures.  We must begin by agreeing on which pulmonary outcome measures to collect, how to analyze these measures, and the meaningfulness of these measures. If we can agree on these issues, we can begin to demonstrate the efficacy of drugs on pulmonary function and, hopefully, speed up the drug approval process. It’s a lofty goal but, with this panel of experts, we think we are up to the task.

Related links

Pulmonary Care for Duchenne

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