Physician's preference-based instrumental variable analysis: is it valid and useful in a moderate-sized study?

Background: Instrumental variable methods can potentially circumvent the unmeasured confounding inherent in observational data analyses.

Methods: We investigated the validity and usefulness of physician's preference instrumental variable analysis in the setting of a moderate-sized clinical study. Using routine care data from 476 elective cardiac surgery patients, we assessed the effect of preoperative corticosteroids on mechanical ventilation time and duration of intensive care and hospital stay, occurrence of infections, atrial fibrillation, heart failure, and delirium.

Results: Although results of the physician's preference-based instrumental variable analysis corresponded in direction to results of a recent large randomized trial of the same therapy, the instrumental variable estimates showed much larger effects with very wide confidence intervals.

Conclusion: The lesser statistical precision limits the usefulness of instrumental variable analysis in a study that might be of sufficient size for conventional analyses, even if a strong and plausible instrument is available.