title: Spinal muscular atrophy type 1: are proactive respiratory interventions associated with longer survival?
abstract: BACKGROUND
  Spinal muscular atrophy type 1, an autosomal recessive motor neuron disease, is a leading genetic cause of death in infancy and early childhood.
  
  
  OBJECTIVE
  To determine whether the early initiation of noninvasive respiratory interventions is associated with longer survival.
  
  
  METHODS
  Single-institution retrospective cohort study identified children with spinal muscular atrophy type 1 from January 1, 2002 to May 1, 2009 who were followed for 2.3 mean yrs.
  
  
  METHODS
  Tertiary care children's hospital and outpatient clinics in a vertically integrated healthcare system.
  
  
  METHODS
  Forty-nine children with spinal muscular atrophy type 1 were grouped according to the level of respiratory support their caregivers chose within the first 3 months after diagnosis: proactive respiratory care (n = 26) and supportive care (n = 23).
  
  
  METHODS
  Proactive respiratory care included bilevel noninvasive ventilation during sleep and twice a day cough assist while supportive respiratory care included suctioning, with or without supplemental oxygen.
  
  
  RESULTS
  Kaplan-Meier survival curves were assessed based on intention to treat. Children treated with early proactive respiratory support had statistically longer survival compared to supportive care (log rank 0.047); however, the adjusted hazard ratio for survival was not statistically different (2.44 [95% confidence interval 0.84-7.1]). Children in the proactive group were more likely to be hospitalized for respiratory insufficiency (83% vs. 46%) and had shortened time after diagnosis until first hospital admission for respiratory insufficiency (median 118 vs. 979 days).
  
  
  CONCLUSIONS
  Longer survival time with spinal muscular atrophy type 1 is associated with early, noninvasive respiratory care interventions after diagnosis.
authors.affiliation: Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA. lemoine@hsc.utah.edu
authors.forename: Tara J
authors.initials: TJ
authors.lastname: Lemoine
authors.forename: Kathryn J
authors.initials: KJ
authors.lastname: Swoboda
authors.forename: Susan L
authors.initials: SL
authors.lastname: Bratton
authors.forename: Richard
authors.initials: R
authors.lastname: Holubkov
authors.forename: Michael
authors.initials: M
authors.lastname: Mundorff
authors.forename: Rajendu
authors.initials: R
authors.lastname: Srivastava
mesh_terms: D015331:Cohort Studies; D005260:Female; D017048:Health Care Costs; D006801:Humans; D007223:Infant; D057194:Intention to Treat Analysis; D008297:Male; D010166:Palliative Care / Q000191:economics / Q000379:methods*; D012131:Respiratory Insufficiency / Q000191:economics / Q000209:etiology / Q000401:mortality / Q000628:therapy*; D012138:Respiratory Therapy / Q000191:economics / Q000379:methods*; D012189:Retrospective Studies; D014897:Spinal Muscular Atrophies of Childhood / Q000150:complications* / Q000191:economics / Q000401:mortality; D016019:Survival Analysis; D016896:Treatment Outcome; D014589:Utah