Chest
Volume 117, Issue 4, April 2000, Pages 1100-1105
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Clinical Investigations in Critical Care
Spinal Muscular Atrophy Type 1: A Noninvasive Respiratory Management Approach

https://doi.org/10.1378/chest.117.4.1100Get rights and content

Study objective

To determine whether spinal muscular atrophy (SMA) type 1 can be managed without tracheostomy and to compare extubation outcomes using a respiratory muscle aid protocol vs conventional management.

Methods

Eleven SMA type 1 children were studied during episodes of respiratory failure. Nine children required multiple intubations. Along with standard treatments, these children received manually and mechanically assisted coughing to reverse airway mucus-associated decreases in oxyhemoglobin saturation. Extubation was not attempted until, most importantly, there was no oxygen requirement to maintain oxyhemoglobin saturation greater than 94%. After extubation, all patients received nasal ventilation with positive end-expiratory pressure. Successful extubation was defined by no need to reintubate during the current hospitalization.

Results

Two children have survived for 37 and 66 months and have never been intubated despite requiring 24-h nasal ventilation since 5 and 7 months of 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 after successful extubation. The other six children have been managed at home for 15 to 59 (mean 30.4) months using nocturnal nasal ventilation after an episode of respiratory failure. The nine children were successfully extubated by our protocol 23 of 28 times. The same children managed conventionally 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 hospitalization and intubation, tracheostomy can be avoided throughout early childhood for some children with SMA type 1.

Section snippets

Materials and Methods

Eleven consecutively referred SMA type 1 children in respiratory failure were managed as per a protocol (Table 1) that was approved by our Institutional Review Board. All 11 patients had severe skeletal and bulbar muscle weakness to the extent that none had functional extremity movements or ability to take any nutrition by mouth. Three have not developed the ability to verbalize. All of the parents of the 11 had refused tracheostomies on multiple occasions.

Nine of the 11 patients have required

Results

In all, 11 consecutively referred patients were treated by the respiratory aid protocol. The demographic data and the results of management are summarized in Table 2 . They had 28 distinct episodes of respiratory compromise necessitating hospitalizations: 2 postoperative, 2 associated with insidiously progressive inspiratory muscle dysfunction, and 24 sudden episodes mostly due to chest colds. These resulted in a total of 48 intubations. Nonprotocol therapy and extubation were attempted 20

Discussion

This study suggests that it may be possible for infants with SMA type 1 to avoid tracheostomy long enough to be able to cooperate with the use of respiratory muscle aids and possibly safely avoid tracheostomy indefinitely.6, 9 This is important because the parents of children with neuromuscular disease often refuse tracheostomy but want their children to survive.

Hypercapnia can cause oxyhemoglobin desaturation. We have noted that patients with neuromuscular disease tend to become symptomatic

ACKNOWLEDGMENT

We thank the University Hospital respiratory therapists, nurses, patients, and their parents, without whose support this study would not have been possible.

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This work was performed at the University Hospital of the University of Medicine and Dentistry of New Jersey-New Jersey Medical School in Newark, NJ.

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