Gruis Kirsten L, Brown Devin L, Weatherwax Kevin J, Feldman Eva L, Chervin Ronald D
University of Michigan Health System, Department of Neurology, 1324 Taubman Center, 032221-500 E. Medical Center Drive, Ann Arbor, MI 48109, USA.
Amyotroph Lateral Scler. 2006 Jun;7(2):96-9. doi: 10.1080/14660820600640604.
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.
无创正压通气（NIV）治疗晚期呼吸功能不全可延长肌萎缩侧索硬化症（ALS）患者的生存期。为了探究更早开始使用NIV是否可能带来额外益处这一关键问题，需要进行一项采用合适安慰剂的随机试验。本研究评估了亚治疗性（假）持续气道正压作为一种潜在安慰剂的效果。在单盲设计中，40例用力肺活量>50%的ALS患者被随机分为两组，分别接受30秒的活性NIV（吸气压力8 cm H₂O，呼气压力4 cm H₂O）或面罩处持续气道正压<1 cm H₂O的假NIV治疗。然后使用一份问卷来评估受试者是否认为自己接受的是“真实”或“假装”的治疗试验。受试者的中位年龄为60.5岁，38%为女性。接受活性NIV的20名受试者中有12名（60%）以及接受假NIV的20名受试者中有7名（35%）认为自己接受的是活性治疗（p = 0.11）。所有受试者中只有8名（20%）对自己判断所接受的是“真实”或“假装”的NIV有信心。因此，亚治疗性（假）持续气道正压是ALS中NIV试验一种有前景的安慰剂对照。
