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Spinal muscular atrophy type 1: Management and outcomes
Author(s) -
Bach John R.,
Baird J. Scott,
Plosky Daniel,
Navado Jose,
Weaver Brian
Publication year - 2002
Publication title -
pediatric pulmonology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.866
H-Index - 106
eISSN - 1099-0496
pISSN - 8755-6863
DOI - 10.1002/ppul.10110
Subject(s) - medicine , spinal muscular atrophy , group b , ventilation (architecture) , respiratory failure , assisted ventilation , mechanical ventilation , atrophy , pediatrics , tracheobronchomalacia , surgery , anesthesia , disease , airway , mechanical engineering , engineering
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.