Measurement of Respiratory Function Decline in Patients with Duchenne Muscular Dystrophy: A Conjoint Analysis

Abstract

Summary points• In Duchenne muscular dystrophy (DMD), little attention has been paid to severity of respiratory function decline (RFD) based on disease progression.• We performed a conjoint analysis among 123 Italian clinicians to generate a scale for RFD in DMD patients.• Before the interview, 11 attributes were selected by discussion among experts. 32 'patient profiles' were generated. Each physician assessed the severity of RFD for each profile. Each level/attribute was assigned an estimated usefulness to understand its impact on RFD.• The identified attributes were forced vital capacity, forced vital capacity decline, dysphagia, type of ventilation and peak cough flow.• These results allowed the development of a scale for RFD severity.• This scale can stratify DMD patients according to the severity of their RFD.• Further validation in a larger, international sample of physicians, including expert pneumologists, by another robust approach such as a multidisciplinary Delphi panel and/or web-based collection of consensus appears necessary.• This score must be correlated with clinical findings and compared with standard testing based on guidelines, in order to correlate the perceived decline and its quantitative evaluation. • This will help define clinical approach in line with the severity of pulmonary function evaluated by our score and allow early management of DMD patients. Aim:In Duchenne muscular dystrophy (DMD), little attention has been paid to severity of respiratory function decline (RFD) based on disease progression. We performed a conjoint analysis among 123 Italian clinicians to generate a scale for RFD in DMD patients. Methods: Before the interview, 11 attributes were selected by discussion among experts. 32 'patient profiles' were generated. Each physician assessed the severity of RFD for each profile. Each level/attribute was assigned an estimated usefulness to understand its impact on RFD. Results: The identified attributes were forced vital capacity, forced vital capacity decline, dysphagia, type of ventilation and peak cough flow. These results allowed the development of a scale for RFD severity. Conclusion: This scale can stratify DMD patients according to the severity of their RFD.

Preliminary Effectiveness and Safety of High Frequency Oscillation in Addition to Mechanical Insufflation and Exsufflation for Intratracheal Mucus Removal in Patients With Neuromuscular Disease: Protocol for a Prospective Study

“…As the disease progresses, vital capacity declines and the inability to expand and empty the chest fully results in reduced chest wall compliance [1-3]. As peak cough flow correlates with disease progression and lung capacity in patients with NMDs [4,5], insufficient coughing is problematic in patients with severe and advanced NMDs. Cough efficacy is related to clearance of secretions in the lung.…”