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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Measurement of respiratory function decline in patients with Duchenne muscular dystrophy: a conjoint analysis
Elena Ripamonti1, Grazia D'Angelo2
1Elma Research s.r.l., Via Felice Casati 32, Milano, Italy.
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.
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