Bayesian adaptive trials offer advantages in comparative effectiveness trials: an example in status epilepticus

J Clin Epidemiol. 2013 Aug;66(8 Suppl):S130-7. doi: 10.1016/j.jclinepi.2013.02.015.

Abstract

Objective: We present a novel Bayesian adaptive comparative effectiveness trial comparing three treatments for status epilepticus that uses adaptive randomization with potential early stopping.

Study design and setting: The trial will enroll 720 unique patients in emergency departments and uses a Bayesian adaptive design.

Results: The trial design is compared to a trial without adaptive randomization and produces an efficient trial in which a higher proportion of patients are likely to be randomized to the most effective treatment arm while generally using fewer total patients and offers higher power than an analogous trial with fixed randomization when identifying a superior treatment.

Conclusion: When one treatment is superior to the other two, the trial design provides better patient care, higher power, and a lower expected sample size.

Keywords: Adaptive sample size; Bayesian adaptive trials; Comparative effectiveness research; Emergency medicine; Response adaptive randomization; Status epilepticus.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Anticonvulsants / therapeutic use*
  • Bayes Theorem
  • Child
  • Child, Preschool
  • Comparative Effectiveness Research / methods*
  • Comparative Effectiveness Research / statistics & numerical data
  • Early Termination of Clinical Trials
  • Emergency Service, Hospital
  • Humans
  • Intention to Treat Analysis
  • Levetiracetam
  • Middle Aged
  • Models, Statistical*
  • Phenytoin / analogs & derivatives
  • Phenytoin / therapeutic use
  • Piracetam / analogs & derivatives
  • Piracetam / therapeutic use
  • Randomized Controlled Trials as Topic*
  • Research Design / statistics & numerical data*
  • Status Epilepticus / therapy*
  • Treatment Outcome
  • Valproic Acid / therapeutic use
  • Young Adult

Substances

  • Anticonvulsants
  • Levetiracetam
  • Valproic Acid
  • Phenytoin
  • fosphenytoin
  • Piracetam