J Head Trauma Rehabil. 2010 Mar 8.

Measuring Outcome in Traumatic Brain Injury Treatment Trials: Recommendations From the Traumatic Brain Injury Clinical Trials Network.

Bagiella E, Novack TA, Ansel B, Diaz-Arrastia R, Dikmen S, Hart T, Temkin N.

Department of Biostatistics, Mailman School of Public Health, Columbia University, New York (Dr Bagiella); Physical Medicine and Rehabilitation, University of Alabama at Birmingham (Dr Novack); National Center for Medical Rehabilitation Research, National Institute of Child Health and Human Development, Bethesda, Maryland (Dr Ansel); Department of Neurology, University of Texas, Southwestern Medical Center, Dallas (Dr Diaz-Arrastia); Departments of Rehabilitation Medicine, Division of Clinical Psychology and Neuropsychology, School of Medicine (Dr Dikmen) and Neurological Surgery and Biostatistics (Dr Temkin), University of Washington, Seattle; and Moss Rehabilitation Research Institute, Philadelphia, Pennsylvania (Dr Hart).

BACKGROUND: Traumatic brain injury (TBI) involves several aspects of a patient's condition, including physical, mental, emotional, cognitive, social, and functional changes. Therefore, a clinical trial with individuals with TBI should consider outcome measures that reflect their global status. METHODS: We present the work of the National Institute of Child Health and Development-sponsored Traumatic Brain Injury Clinical Trials Network Outcome Measures subcommittee and its choice of outcome measures for a phase III clinical trial of patients with complicated mild to severe TBI. RESULTS: On the basis of theoretical and practical considerations, the subcommittee recommended the adoption of a core of 9 measures that cover 2 different areas of recovery: functional and cognitive. These measures are the Extended Glasgow Outcome Scale; the Controlled Oral Word Association Test; the Trail Making Test, Parts A and B; the California Verbal Learning Test-II; the Wechsler Adult Intelligence Scale-III Digit Span subtest; the Wechsler Adult Intelligence Scale-III Processing Speed Index; and the Stroop Color-Word Matching Test, Parts 1 and 2. CONCLUSIONS: The statistical methods proposed to analyze these measures using a global test procedure, along with research and methodological and regulatory issues involved with the use of multiple outcomes in a clinical trial, are discussed.