Spinal muscular atrophy type 1 - A noninvasive respiratory management approach

Study objective: To determine whether spinal muscular atrophy (SMA) type 1 can be managed without tracheostomy and to compare extubation outcomes usin g a respiratory muscle aid protocol vs conventional management. Design: A retrospective cohort study. Methods: Eleven SMA type I children were studied during episodes of respira tory failure. Nine children required multiple intubations. Along with stand ard treatments, these children received manually and mechanically assisted coughing to reverse airway mucus-associated decreases in oxyhemoglobin satu ration. Extubation was not attempted until, most importantly, there was no oxygen requirement to maintain oxyhemoglobin saturation greater than 94%. A fter extubation, all patients received nasal ventilation with positive end- expiratory pressure. Successful extubation was defined by no need to reintu bate during the current hospitalization, Results: Two children have survived for 37 and 66 months and have never bee n intubated despite requiring 24-h nasal ventilation since 5 and 7 months o f age, respectively. One other child underwent tracheostomy for persistent left lung collapse and inadequate home care, another for need for frequent readmission and intubation, and one child was lost to follow-up 3 months af ter successful extubation. The other sh children have been managed at home for 15 to 59 (mean 30. 1) months using nocturnal nasal ventilation after an episode: of respiratory failure. The nine children were successfully extub ated by our protocol 23 of 28 times. The same children managed conventional ly were successfully extubated 2 of 20 times when not using this protocol ( p < 0.001 by the two-tailed Fisher's Exact t Test). Conclusion: Although intercurrent chest colds may necessitate periods of ho spitalization and intubation, tracheostomy can be avoided throughout early childhood for some children with SMA type 1.