Outcomes in pediatric neurology: a review of conceptual issues and recommendationsThe 2010 Ronnie Mac Keith Lecture

Abstract

This paper discusses how to evaluate whether, and in what ways, treatments affect the lives of children with neurological conditions and their families. We argue that professionals should incorporate perspectives from patients and families to help them make decisions about what 'outcomes' are important, and we discuss how those outcomes might be assessed. A case vignette illustrates the differences and complementarity between the perspectives of clinicians and those of children and their parents. We recommend methods for expanding the range of relevant health outcomes in child neurology to include those that reflect the ways patients and families view their conditions and our interventions. We explore the added value of a 'non-categorical' approach to the choice of outcomes. The International Classification of Functioning, Disability and Health is a useful biopsychosocial framework to 'rule in' relevant aspects of child and family issues to create a dynamic system of possible influences on outcomes. We examine the meaning of 'health', 'health-related quality of life', and 'quality of life' as related but conceptually distinct outcomes. Specific issues are discussed about the construction, validation, and appraisal of outcome measures, as well as practical recommendations on how to select outcome measures in the clinical setting and research.