title: Spinal muscular atrophy type 1: avoidance of hospitalization by respiratory muscle support.
abstract: OBJECTIVE
  The aim of this study was to report the outcomes of an oximetry protocol using up to continuous full ventilator-setting noninvasive ventilation (NIV) and mechanically assisted coughing (MAC) to avoid episodes of acute respiratory failure and hospitalizations for children with spinal muscular atrophy type 1 under 3 yrs of age.
  
  
  METHODS
  This study was a retrospective chart review of 16 patients with spinal muscular atrophy type 1 under 3 yrs of age consecutively referred for respiratory decompensations resulting in continuous NIV dependence and oxyhemoglobin desaturation. An avoided hospitalization was defined by the need for continuous NIV using high span bilevel positive airway pressure and reversal of desaturations by MAC in the home setting. The protocol included training and equipping parents to use NIV, MAC, and basic life support.
  
  
  RESULTS
  There were 49 acute episodes (1.20/patient per year), of which 43 met the criteria for an avoided hospitalization. Therefore, only six episodes (0.15/patient per year) required hospitalization, and four required endotracheal intubation (0.1/patient per year). Three of the four were extubated after 4, 9, and 15 days, respectively, to full NIV support and aggressive MAC. The fourth patient, for whom NIV could not be provided, underwent an emergency intubation at home and died at the age of 40 mos.
  
  
  CONCLUSIONS
  A protocol including high span bilevel positive airway pressure along with MAC to expel airway secretions and normalize oxyhemoglobin saturation can be used by trained caregivers to avoid episodes of acute respiratory failure and hospitalization for children with spinal muscular atrophy type 1 under 3 yrs of age.
authors.affiliation: U.O. Anestesia e Rianimazione IRCCS G Gaslini, Genoa, Italy.
authors.forename: Giancarlo
authors.initials: G
authors.lastname: Ottonello
authors.forename: Chiara
authors.initials: C
authors.lastname: Mastella
authors.forename: Alessia
authors.initials: A
authors.lastname: Franceschi
authors.forename: Daniela
authors.initials: D
authors.lastname: Bosticco
authors.forename: Andrea
authors.initials: A
authors.lastname: Wolfler
authors.forename: Marina
authors.initials: M
authors.lastname: Pedemonte
authors.forename: Irene
authors.initials: I
authors.lastname: Esposito
authors.forename: Cesare
authors.initials: C
authors.lastname: Gregoretti
mesh_terms: D000208:Acute Disease; D002675:Child, Preschool; D003371:Cough; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D007442:Intubation, Intratracheal / Q000706:statistics & numerical data; D063087:Noninvasive Ventilation; D010092:Oximetry; D010108:Oxyhemoglobins / Q000032:analysis; D010290:Parents; D012131:Respiratory Insufficiency / Q000517:prevention & control; D012189:Retrospective Studies; D012648:Self Care; D014897:Spinal Muscular Atrophies of Childhood / Q000628:therapy*
chemical_list: D010108:Oxyhemoglobins