Duchenne Muscular Dystrophy

Abstract

OBJECTIVE: To quantitate prolongation of survival for patients with Duchenne muscular dystrophy with the use of noninvasive intermittent positive-pressure ventilation (IPPV) with and without access to a protocol involving mechanically assisted coughing. DESIGN: In this retrospective review of all patients with Duchenne muscular dystrophy visiting a neuromuscular disease clinic, patients were trained to use mouth piece and nasal IPPV and mechanically assisted coughing to maintain oxyhemoglobin saturation >94% (protocol). Survival was considered prolonged when noninvasive IPPV was required full time. RESULTS: Ninety-one of 125 patients used noninvasive IPPV part time for 1.9 +/- 1.3 yr, and 51 went on to require it full time for 6.3 +/- 4.6 yr. Of the 31 noninvasive IPPV users who died without access to the protocol, 20 died from respiratory causes and seven died from cardiac causes. None of the 34 full-time noninvasive IPPV users with access to the protocol underwent tracheotomy or died from respiratory complications during a period of 5.4 +/- 4.0 yr, whereas three died from heart failure. Five patients with no breathing tolerance were extubated or decannulated to continuous noninvasive IPPV. CONCLUSIONS: Noninvasive respiratory aids can prolong survival and permit extubation or decannulation of patients with Duchenne muscular dystrophy with no breathing tolerance.