Duchenne Muscular Dystrophy: Continuous Noninvasive Ventilatory Support Prolongs Survival

OBJECTIVE: To describe survival outcomes with noninvasive ventilation (NIV) for full ventilatory support, and a mechanically assisted cough and oximetry protocol in a series of patients with Duchenne muscular dystrophy. METHODS: We monitored end-tidal carbon dioxide (P-ETCO2), S-pO2, vital capacity, maximum insufflation capacity, and cough peak flow. Nocturnal NIV was initiated for symptomatic hypoventilation. An oximeter and mechanically assisted cough device were prescribed when the patient's maximum assisted cough peak flow fell below 300 L/min. Patients used up to continuous NIV and mechanically assisted cough to return S-pO2 to >= 95% during intercurrent respiratory infections or as otherwise needed. We recorded respiratory and cardiac hospitalizations and mortality, and quantified survival by duration of continuous NW dependence (ie, unable to maintain oxygenation without the ventilator). RESULTS: With advancing Duchenne muscular dystrophy, 101 nocturnal-only NW users extended their NIV use throughout the daytime hours and required it continuously for 7.4 +/- 6.1 years to 30.1 +/- 6.1 years of age, with 56 patients still alive. Twenty-six of the 101 became continuously dependent without requiring hospitalization. Eight tracheostomized users were decannulated to NW. Thirty-one consecutive unweanable intubated patients were extubated to NIV plus mechanically assisted cough. Of the 67 deaths (including 8 patients who died from heart failure before requiring ventilator use), 34 (52%) were probably cardiac, 14 (21%) were probably respiratory, and 19 (27%) were of unknown or other etiology. CONCLUSIONS: Continuous NIV along with mechanically assisted cough and oximetry as needed can prolong life and obviate tracheotomy in patients with Duchenne muscular dystrophy. Unweanable patients can be decannulated and extubated to NIV plus mechanically assisted cough.