title: Trial of early noninvasive ventilation for ALS: A pilot placebo-controlled study.
abstract: OBJECTIVE
  To evaluate the use and tolerability of noninvasive positive pressure ventilation (NIV) in patients with amyotrophic lateral sclerosis (ALS) early in their disease by comparing active NIV and sham NIV in patients not yet eligible for NIV use as recommended by practice guidelines.
  
  
  METHODS
  This was a single-center, prospective, double-blind, randomized, placebo (sham)-controlled pilot trial. Patients with ALS were randomized to receive either sham NIV or active NIV and underwent active surveillance approximately every 3 months until they reached a forced vital capacity (FVC) <50% or required NIV for clinical symptom management.
  
  
  RESULTS
  In total, 54 participants were randomized. The mean NIV use was 2.0 hours (95% confidence interval [CI] 1.1-3.0) per day in the sham NIV treatment group and 3.3 hours (CI 2.0-4.6) per day in the active NIV group, which did not differ by treatment group (p = 0.347). The majority of sham NIV participants (88%) and active NIV participants (73%) reported only mild or no problem with NIV use. Difference of change in FVC through the treatment period by group (0.44 per month) favored active NIV (p = 0.049). Survival and changes in maximal inspiratory or expiratory pressure did not differ between treatment groups.
  
  
  CONCLUSIONS
  The efficacy of early NIV in ALS should be tested in randomized, placebo-controlled trials. The trial is registered on clinicaltrials.gov (NCT00580593).
  
  
  METHODS
  This study provides Class II evidence that for patients with ALS, adherence with NIV and sham NIV are similar.
authors: From the Departments of Neurosurgery (T.L.J.) and Neurology (T.L.J., D.L.B., E.M.M., T.F.), University of Michigan; Biostatistics (J.B.), University of Michigan School of Public Health, Ann Arbor; and Idera Pharmaceuticals (K.L.G.), Cambridge, MA. teresmit@umich.edu.
authors: Teresa L
authors: TL
authors: Jacobs
authors: From the Departments of Neurosurgery (T.L.J.) and Neurology (T.L.J., D.L.B., E.M.M., T.F.), University of Michigan; Biostatistics (J.B.), University of Michigan School of Public Health, Ann Arbor; and Idera Pharmaceuticals (K.L.G.), Cambridge, MA.
authors: Devin L
authors: DL
authors: Brown
authors: From the Departments of Neurosurgery (T.L.J.) and Neurology (T.L.J., D.L.B., E.M.M., T.F.), University of Michigan; Biostatistics (J.B.), University of Michigan School of Public Health, Ann Arbor; and Idera Pharmaceuticals (K.L.G.), Cambridge, MA.
authors: Jonggyu
authors: J
authors: Baek
authors: From the Departments of Neurosurgery (T.L.J.) and Neurology (T.L.J., D.L.B., E.M.M., T.F.), University of Michigan; Biostatistics (J.B.), University of Michigan School of Public Health, Ann Arbor; and Idera Pharmaceuticals (K.L.G.), Cambridge, MA.
authors: Erin M
authors: EM
authors: Migda
authors: From the Departments of Neurosurgery (T.L.J.) and Neurology (T.L.J., D.L.B., E.M.M., T.F.), University of Michigan; Biostatistics (J.B.), University of Michigan School of Public Health, Ann Arbor; and Idera Pharmaceuticals (K.L.G.), Cambridge, MA.
authors: Timothy
authors: T
authors: Funckes
authors: From the Departments of Neurosurgery (T.L.J.) and Neurology (T.L.J., D.L.B., E.M.M., T.F.), University of Michigan; Biostatistics (J.B.), University of Michigan School of Public Health, Ann Arbor; and Idera Pharmaceuticals (K.L.G.), Cambridge, MA.
authors: Kirsten L
authors: KL
authors: Gruis
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D063087:Noninvasive Ventilation / Q000379:methods*; D010865:Pilot Projects; D011446:Prospective Studies; D016896:Treatment Outcome