Measurement of neurodevelopmental changes in children treated with radiation for brain tumors: what is a true 'baseline?'

OBJECTIVE: To review the various ways in which baseline neuropsychological functioning is measured in the extant literature on pediatric brain tumors, describe the pros and cons of each approach, and increase the awareness of researchers as to the implications of each.

METHOD: We reviewed the literature from 1993 to 2013, and classified studies by baseline approach and explicitness of selection of approach.

RESULTS: There are multiple approaches to operationalizing baseline levels of ability and to assess change from baseline. Each approach has strengths and weaknesses, and selection may depend on the question under investigation. Approaches to baseline estimation varied widely with a trend over time toward reliance on statistical modeling. Researchers were often insufficiently explicit about the reasons for adopting a particular approach. The common use of standardized scores requires caution as they obscure critical inferential limitations about change and magnitude of change. Some viable approaches were infrequently used, such as actuarial prediction formulas. Multiple simultaneous methods akin to theory testing and formal methods of construct validation could enhance scientific yield since all approaches are fallible.

CONCLUSIONS: Estimating baseline neuropsychological functioning is very challenging, particularly when it concerns children in the preschool years. Nevertheless, it is a crucial methodological decision with important implications for the interpretation of research findings that needs to be dealt with explicitly.