Original Article

Spinal muscular atrophy type 1: Management and outcomes

  1. John R. Bach MD1,*,
  2. J. Scott Baird MD2,
  3. Daniel Plosky MD1,
  4. Jose Navado MD2,
  5. Brian Weaver BS1

Article first published online: 4 JUN 2002

DOI: 10.1002/ppul.10110

Issue

Pediatric Pulmonology

Pediatric Pulmonology

Volume 34, Issue 1, pages 16–22, July 2002

Additional Information

How to Cite

Bach, J. R., Baird, J. S., Plosky, D., Navado, J. and Weaver, B. (2002), Spinal muscular atrophy type 1: Management and outcomes. Pediatric Pulmonology, 34: 16–22. doi: 10.1002/ppul.10110

Author Information

  1. 1

    Department of Physical Medicine and Rehabilitation, UMDNJ-New Jersey Medical School, Newark, New Jersey

  2. 2

    Department of Pediatrics, UMDNJ-New Jersey Medical School, Newark, New Jersey

*Department of Physical Medicine and Rehabilitation, University Hospital B-403, UMDNJ-New Jersey Medical School, 150 Bergen St., Newark, NJ 07103.

Publication History

  1. Issue published online: 4 JUN 2002
  2. Article first published online: 4 JUN 2002
  3. Manuscript Accepted: 13 MAR 2002
  4. Manuscript Received: 24 OCT 2001

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Keywords:

  • spinal muscular atrophy;
  • mechanical ventilation;
  • respiratory insufficiency;
  • outcomes

Abstract

Our objectives were to describe survival, hospitalization, speech, and outcomes related to respirator needs for spinal muscular atrophy type 1 (SMA1) patients, using noninvasive or tracheostomy ventilation. From 65 SMA patients referred to our clinic since 1996, we chose 56 SMA1 patients who developed respiratory failure before age 2 years. Patients either had tracheostomy tubes (group A), or used noninvasive ventilation and assisted coughing; a previously reported extubation protocol (group B) was used as needed.

Sixteen patients underwent tracheostomy at 10.8 ± 5.0 months of age, 33 were in group B, and 7 others died without life-support interventions. Compared to group B, group A patients had fewer hospitalizations until age 3 years, but more after age 5, and 15 of 16 lost all spontaneous breathing tolerance posttracheostomy and could not speak. One group A patient died at 16 months of age, and the others were 73.8 ± 57 months of age (the oldest was 19 years old). Two group B patients died at 6 and 13 months, respectively, whereas the other 31 were 41.8 ± 26.0 months (and up to 8.3 years) old. Three of 31 in group B required high-span positive inspiratory pressure plus positive end-expiratory pressure (PIP + PEEP) continuously with minimal tolerance for breathing on their own, and 4 could not communicate verbally.

In conclusion, SMA type 1 children can survive beyond 2 years of age when offered tracheostomy or noninvasive respiratory support. The latter is associated with fewer hospitalizations after age 5 years, freedom from daytime ventilator use, and the ability to speak. Pediatr Pulmonol. 2002; 34:16–22. © 2002 Wiley-Liss, Inc.

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