title: Effects of non-invasive ventilation on survival and quality of life in patients with amyotrophic lateral sclerosis: a randomised controlled trial.
abstract: BACKGROUND
  Few patients with amyotrophic lateral sclerosis currently receive non-invasive ventilation (NIV), reflecting clinical uncertainty about the role of this intervention. We aimed to assess the effect of NIV on quality of life and survival in amyotrophic lateral sclerosis in a randomised controlled trial.
  
  
  METHODS
  92 of 102 eligible patients participated. They were assessed every 2 months and randomly assigned to NIV (n=22) or standard care (n=19) when they developed either orthopnoea with maximum inspiratory pressure less than 60% of that predicted or symptomatic hypercapnia. Primary validated quality-of-life outcome measures were the short form 36 mental component summary (MCS) and the sleep apnoea quality-of-life index symptoms domain (sym). Both time maintained above 75% of baseline (T(i)MCS and T(i)sym) and mean improvement (microMCS and microsym) were measured.
  
  
  RESULTS
  NIV improved T(i)MCS, T(i)sym, microMCS, microsym, and survival in all patients and in the subgroup with better bulbar function (n=20). This subgroup showed improvement in several measures of quality of life and a median survival benefit of 205 days (p=0.006) with maintained quality of life for most of this period. NIV improved some quality-of-life indices in those with poor bulbar function, including microsym (p=0.018), but conferred no survival benefit.
  
  
  CONCLUSIONS
  In patients with amyotrophic lateral sclerosis without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy. In patients with severe bulbar impairment, NIV improves sleep-related symptoms, but is unlikely to confer a large survival advantage.
authors.affiliation: Department of Respiratory Medicine, University of Newcastle upon Tyne and the Newcastle Hospitals Trust, Newcastle upon Tyne, UK. Stephen.bourke@northumbria-healthcare.nhs.uk
authors.forename: Stephen C
authors.initials: SC
authors.lastname: Bourke
authors.forename: Mark
authors.initials: M
authors.lastname: Tomlinson
authors.forename: Tim L
authors.initials: TL
authors.lastname: Williams
authors.forename: Robert E
authors.initials: RE
authors.lastname: Bullock
authors.forename: Pamela J
authors.initials: PJ
authors.lastname: Shaw
authors.forename: G John
authors.initials: GJ
authors.lastname: Gibson
mesh_terms: D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011175:Positive-Pressure Respiration; D011788:Quality of Life; D012131:Respiratory Insufficiency / Q000209:etiology / Q000628:therapy; D012891:Sleep Apnea Syndromes / Q000209:etiology / Q000628:therapy; D013534:Survival; D016896:Treatment Outcome