1. Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystroph.
- Author
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Buyse, Gunnar, Rummey, Christian, Meier, Thomas, Leinonen, Mika, Voit, Thomas, Mayer, Oscar, and McDonald, Craig
- Subjects
Duchenne muscular dystrophy ,Pulmonary function ,idebenone ,peak expiratory flow ,respiration ,Adolescent ,Antioxidants ,Child ,Humans ,Male ,Muscular Dystrophy ,Duchenne ,Peak Expiratory Flow Rate ,Respiratory Function Tests ,Self Care ,Ubiquinone - Abstract
BACKGROUND: Loss of pulmonary function is a main cause of early morbidity and mortality in patients with Duchenne muscular dystrophy (DMD). Standard of care guidelines recommend regular assessment of pulmonary function by hospital-based spirometry to detect onset and monitor progression of pulmonary function decline. OBJECTIVE: To assess the feasibility of home-based monitoring of pulmonary function by a hand-held device (HHD) in adolescent and adult patients with DMD over a period of 12 months. METHODS: In the phase III randomized placebo-controlled DELOS trial in 10-18 year old DMD patients, peak expiratory flow (PEF) measurements were collected weekly at home by the patient (assisted by parent/caregiver) using a peak flow meter HHD. Adherence to the use of the HHD was assessed and 12-month changes in PEF as percent of predicted (PEF% p) for the idebenone (N = 31) and the placebo treatment groups (N = 33) from HHD-derived data were compared to results from hospital-based spirometry. RESULTS: A total of 2689 individual HHD assessments were analysed. Overall adherence to the use of the HHD over the course of the 12-month study duration was good (75.9%, SD 21.5%) and PEF% p data obtained at the same day by HHD and standard spirometry correlated well (Spearmans rho 0.80; p
- Published
- 2018