Duchenne muscular dystrophy: Survival by cardio-respiratory interventions

Abstract

We describe survival in Duchenne dystrophy by invasive and noninvasive ventilation vs. untreated. Patients were untreated prior to 1984 (Group 1), underwent tracheotomy from 1984 until 1991 (Group 2), and were managed by noninvasive mechanical ventilation and cardioprotective medications subsequently (Group 3). Symptoms, vital capacity, and blood gases were monitored for all and spirometry, cough peak flows, carbon dioxide tension, and oximetry for Group 3. Sleep nasal ventilation was initiated for symptomatic hypoventilation. An oximeter and mechanical cough assistance were prescribed for maximum assisted cough peak flow <300 L/m. Patients used continuous noninvasive ventilation and mechanically assisted coughing as needed to maintain pulse oxyhemoglobin saturation ⩾95%. Survival was compared by Kaplan–Meier analysis. The 56 of Group 1 died at 18.6 ± 2.9, the 21 Group 2 at 28.1 ± 8.3 years of age with three still alive, and the 88 using noninvasive ventilation had 50% survival to 39.6 years, p < 0.001, respectively. We conclude that noninvasive mechanical ventilation and assisted coughing provided by specifically trained physicians and therapists, and cardioprotective medication can result in more favorable outcomes and better survival by comparison with invasive treatment.