The role of sham continuous positive airway pressure as a placebo in controlled trials: Best Apnea Interventions for Research Trial

Abstract

Study objectives: The main objective of this study was to evaluate the role of sham continuous positive airway pressure (CPAP) compared to conservative medical therapy (CMT) as a control arm in the Best Apnea Interventions for Research (BestAIR) study by assessing differences in subjectively and objectively measured outcomes, adverse events, adherence, and retention rates.

Methods: BestAIR is a clinical trial aimed to identify important design features for future randomized controlled trials of CPAP. Participants with obstructive sleep apnea were randomized to one of four groups; two control arms (CMT, sham-CPAP) and two active CPAP arms (with and without behavioral interventions). Blood pressure and health-related quality of life outcomes were assessed at baseline, 6 and 12 months. Study outcomes, retention, and adverse event rates were compared between the two control arms. Sham-CPAP adherence and self-efficacy were also compared to active-CPAP adherence (without behavioral intervention).

Results: Our sample included 86 individuals in the control arms and 42 participants in the active-CPAP arm. There were no differences in longitudinal profiles in blood pressure, health-related quality of life outcomes, dropout rates, or adverse events in sham-CPAP group compared to CMT-only group (all ps > 0.05); standardized differences were generally small and with inconsistent directionality across measurements. When compared to active-CPAP, sham-CPAP was associated with 93 fewer minutes/night of usage over 12 months (p = 0.007) and lower outcome expectations (p < 0.05).

Conclusion: We observed no evidence of differences in objectively or subjectively measured outcomes with the use of sham-CPAP compared to CMT group. The lower adherence on sham-CPAP and poorer self-efficacy compared to active-CPAP may suggest differences in perceived benefit.

Registration: NCT01261390 Best Apnea Interventions for Research (BestAIR) www.clinicaltrials.gov.

Keywords: CPAP; adherence; clinical trial; placebo; sham; sleep apnea.