title: Identifying who will benefit from non-invasive ventilation in amyotrophic lateral sclerosis/motor neurone disease in a clinical cohort.
abstract: BACKGROUND
  Respiratory failure is associated with significant morbidity and is the predominant cause of death in motor neurone disease/amyotrophic lateral sclerosis (MND/ALS). This study aimed to determine the effect of non-invasive ventilatory (NIV) support on survival and pulmonary function decline across MND/ALS phenotypes.
  
  
  METHODS
  Cohort recruited via a specialist, multidisciplinary clinic. Patients were categorised into four clinical phenotypes (ALS, flail arm, flail leg and primary lateral sclerosis) according to site of presenting symptom and the pattern of upper versus lower motor neurone involvement. NIV was initiated according to current consensus practice guidelines.
  
  
  RESULTS
  Between 1991 and 2011, 1198 patients diagnosed with ALS/MND were registered. 929 patients (77.5%) fulfilled the selection criteria and their data were analysed. Median tracheostomy free survival from symptom onset was 28 months in NIV-treated patients compared to 15 months in untreated (Univariate Cox regression HR=0.61 (0.51 to 0.73), p<0.001). The positive survival effect of NIV persisted when the model was adjusted for age, gender, riluzole and percutaneous endoscopic gastrostomy use (HR=0.72 (0.60 to 0.88, p=0.001). In contrast with the only randomised controlled trial, NIV statistically significantly increased survival by 19 months in those with ALS-bulbar onset (Univariate HR=0.50 (0.36 to 0.70), multivariate HR=0.59 (0.41 to 0.83)). These data confirm that NIV improves survival in MND/ALS. The overall magnitude of benefit is 13 months and was largest in those with ALS-bulbar disease. Future research should explore the optimal timing of NIV initiation within phenotypes in order to optimise respiratory function, quality of life and survival.
authors: Institute for Breathing and Sleep, Bowen Centre, Austin Hospital, Heidelberg, Victoria, Australia.
authors: David J
authors: DJ
authors: Berlowitz
authors: Institute for Breathing and Sleep, Bowen Centre, Austin Hospital, Heidelberg, Victoria, Australia.
authors: Mark E
authors: ME
authors: Howard
authors: Institute for Breathing and Sleep, Bowen Centre, Austin Hospital, Heidelberg, Victoria, Australia.
authors: Julio F
authors: JF
authors: Fiore
authors: Statistical Consulting Centre, The University of Melbourne, Melbourne, Victoria, Australia.
authors: Stephen
authors: S
authors: Vander Hoorn
authors: Institute for Breathing and Sleep, Bowen Centre, Austin Hospital, Heidelberg, Victoria, Australia.
authors: Fergal J
authors: FJ
authors: O'Donoghue
authors: Institute for Breathing and Sleep, Bowen Centre, Austin Hospital, Heidelberg, Victoria, Australia.
authors: Justine
authors: J
authors: Westlake
authors: Neurology Unit, Calvary Health Care, Bethlehem Hospital, Caulfield, Victoria, Australia.
authors: Anna
authors: A
authors: Smith
authors: Institute for Breathing and Sleep, Bowen Centre, Austin Hospital, Heidelberg, Victoria, Australia.
authors: Fiona
authors: F
authors: Beer
authors: Neurology Unit, Calvary Health Care, Bethlehem Hospital, Caulfield, Victoria, Australia.
authors: Susan
authors: S
authors: Mathers
authors: Neurology Unit, Calvary Health Care, Bethlehem Hospital, Caulfield, Victoria, Australia.
authors: Paul
authors: P
authors: Talman
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000175:diagnosis / Q000628:therapy*; D015331:Cohort Studies; D016208:Databases, Factual; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D063087:Noninvasive Ventilation; D011788:Quality of Life; D012189:Retrospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome
keywords: MOTOR NEURON DISEASE; SLEEP