Current Standards and Future Directions of Duchenne Muscular Dystrophy Respiratory Care: The PPMD Italy Meeting Report.

Abstract

Objective: This report summarizes key discussions from the meeting "Current Standards and Future Directions of Respiratory Assessment and Management of Duchenne Muscular Dystrophy (DMD)," organized by Parent Project Muscular Dystrophy (PPMD) Italy and the United States to address current challenges and opportunities in DMD respiratory care.

Methods: The meeting brought together researchers, clinicians, and patient advocates who shared experiences, discussed advancements in DMD respiratory management, and identified areas of debate that require further research.

Results: The speakers emphasized routine assessment of pulmonary function and of breathing during sleep to achieve timely diagnosis of respiratory complications. Therapeutic discussions focused on airway clearance and assisted ventilation, highlighting noninvasive ventilation (NIV) as the preferred modality, even for advanced respiratory failure. The respiratory implications of new pharmacological therapies were discussed. The speakers endorsed the importance of cardiorespiratory outcomes in assessments of drug efficacy. To assess a drug's clinical impact and to define current respiratory phenotypes, the trajectory of the absolute value of forced vital capacity (FVC) was proposed as a potentially better parameter than FVC percent predicted, which is favored in current drug studies. In regard to management of acute respiratory failure and perioperative situations, standards of care and areas needing future research were identified.

Conclusion: In this meeting, many points of consensus emerged, as well as areas requiring further research. The necessity to involve patients and their families in all aspects of respiratory care was emphasized, as well as the need for patient centered outcomes in medical decision making and research.