The influence of enrollment criteria on recruitment and outcome distribution in traumatic brain injury studies: results from the impact study

J Neurotrauma. 2009 Jul;26(7):1069-75. doi: 10.1089/neu.2008.0569.

Abstract

Substantial heterogeneity exists among patients who suffer from traumatic brain injury (TBI). Strict enrollment criteria may diminish heterogeneity in randomized controlled trials (RCTs), but will also decrease recruitment and may affect the outcome distribution. The aim of this study was to investigate the influences of commonly used enrollment criteria for RCTs in TBI on potential recruitment and on outcome distribution. We used individual patient data from the International Mission on Prognosis and Analysis of Clinical Trials in TBI (IMPACT) database, including six therapeutic phase III RCTs (n = 5816) and three surveys (n = 2217) in TBI. The primary outcome was the Glasgow Outcome Scale (GOS) at 6 months after injury, which we dichotomized as favorable/unfavorable. We investigated the influences of commonly used enrollment criteria on recruitment and outcome distribution: time window between injury and admission to study hospital <or= 8 h; age at injury <or= 65 years; >or= 1 reactive pupil; motor score > 1; Glasgow Coma Scale <or= 8. Application of all enrollment criteria resulted in a large reduction of recruitment in both the surveys (up to 65%) and the RCTs (up to 41%). Among the remaining patients, fewer had an unfavorable outcome in both the surveys (original, 60%; remaining, 44%) and the RCTs (original, 43%; remaining, 38%). Applying these enrollment criteria to patients from the surveys resulted in an outcome distribution that approximated the outcome observed in the RCTs. The use of strict enrollment criteria leads to substantial reductions in the recruitment of RCTs in TBI. The outcome in TBI studies depends strongly on the enrollment criteria.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Brain Injuries / therapy*
  • Clinical Trials as Topic*
  • Glasgow Coma Scale
  • Glasgow Outcome Scale
  • Humans
  • Patient Selection*
  • Treatment Outcome