title: Evaluation of sham non-invasive ventilation for randomized, controlled trials in ALS.
abstract: Non-invasive positive pressure ventilation (NIV) treatment of advanced respiratory insufficiency prolongs survival in ALS. To investigate the critical question of whether earlier initiation of NIV might provide additional benefit, a randomized trial with an appropriate placebo is needed. This study evaluated sub-therapeutic (sham) continuous positive airway pressure as a potential placebo. In a single-blind design, 40 ALS patients with forced vital capacity>50% were randomized to receive 30 seconds (s) of either active NIV, with 8 cm H2O inspiratory and 4 cm H2O expiratory pressure, or sham NIV with<1 cm of H2O continuous positive airway pressure at the mask. A questionnaire was then used to assess whether subjects thought that they had received a "real" or "pretend" treatment trial. The subjects' median age was 60.5 years, and 38% were female. Twelve of 20 subjects (60%) who received active NIV and 7 (35%) of the 20 subjects who received sham thought that they had tried the active treatment (p = 0.11). Only 8 (20%) of all subjects were confident about their determination that they had received "real" or "pretend" NIV. Thus, sub-therapeutic (sham) continuous positive airway pressure is a promising placebo control for NIV trials in ALS.
authors.affiliation: University of Michigan Health System, Department of Neurology, 1324 Taubman Center, 032221-500 E. Medical Center Drive, Ann Arbor, MI 48109, USA. kgruis@umich.edu
authors.forename: Kirsten L
authors.initials: KL
authors.lastname: Gruis
authors.forename: Devin L
authors.initials: DL
authors.lastname: Brown
authors.forename: Kevin J
authors.initials: KJ
authors.lastname: Weatherwax
authors.forename: Eva L
authors.initials: EL
authors.lastname: Feldman
authors.forename: Ronald D
authors.initials: RD
authors.lastname: Chervin
mesh_terms: D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000503:physiopathology / Q000628:therapy*; D045422:Continuous Positive Airway Pressure; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010919:Placebos; D011659:Pulmonary Gas Exchange; D012107:Research Design; D012121:Respiration, Artificial; D014797:Vital Capacity
chemical_list: D010919:Placebos