Evaluation of Clinical Trial Outcome Instruments in Children and Youth
Children and youth with spinal cord injury (SCI) are often excluded from clinical trials that may lead to better treatment for themselves and others. One barrier to children’s participation is the void in pediatric psychometric studies of commonly used clinical trials outcome measures. This body of work is intended to develop, validate and provide evidence-informed recommendations for use of outcome instruments with children and youth with SCI. The work is a multi-center and international effort focused on the Walking Index for SCI (WISCI-III), Graded Redefined Assessment of Sensation, Strength and Prehension (GRASSP), Capabilities of Upper Extremity Test (CUE-T), Spinal Cord Independence Measure (SCIM-III), the National Institutes of Neurological Diseases and Stroke (NINDS) Common Data Elements (CDE) and the International SCI Basic Data Sets. Dr. Mulcahey is also collaborating on a multi-center research project that will develop and validate a SCI module for the PedsQL.
Development, Validation and Knowledge Translation of Computerized Adaptive Tests (CATs) and Short Forms (SFs)
IRT-based outcome instruments such as CATS and SFs, provide a mechanism for meaningful, precise and low burden assessment of patient-reported and performance-based outcomes. This body of work is focused on development, validation, replenishment and dissemination of The Pediatric Spinal Cord Injury Activity Measure (PEDI-SCI AM), the Pediatric Measure of Participation (PMoP), the Cerebral Palsy Profile (CP-PRO), the Brachial Plexus Birth Palsy (BPBP) short forms, and the Spinal Cord Injury Functional Index (SCI-FI). Linking estimates have been established and validate for the PEDI-SCI AM (pediatric measure) and the SCI-FI (adult measure) to enable assessment across the lifespan using a common metric. Implementation science methodologies are being utilized to engage stakeholders and end-users to develop meaningful score reports that support shared decision-making among providers, recipient of services, and their caregivers.
Development, Validation and Clinical Relevance of Imaging Biomarkers for SCI
The neurological consequence (level of injury, complete versus incomplete, etc) of SCI is determined by clinical examinations that involve strength testing of 20 muscles, sensory testing of 56 dermatomes, and a rectal examination. These examinations require full participation and cognitive awareness of the person being examined. We have previously shown that children younger than 6 years of age, and some as old as 10 years of age do not have the developmental capabilities and\or ability to engage in the examinations. Likewise, many adults with acute injuries cannot engage due to multiple trauma, brain injury, coma, medical-induced sedation, and at times substance use\abuse. The barriers to the clinical examinations and the need for a more objective measure of injury to the spinal cord catalyzed a body of work that is currently focused on developing and validating spinal cord imaging biomarkers, and creating imaging prediction models for SCI.
Dr. Mulcahey is conducting outcomes research in a variety of areas, utilizing the robust and psychometrically sound instruments that she and others have developed and validated. The intent of this research is to establish evidence-informed interventions for persons living and affected by chronic conditions. Examples of interventions include upper extremity recovery (activity based rehabilitation), restorative (nerve\tendon transfers) and compensatory (functional electrical stimulation) treatments and coaching for caregivers of youth with SCI.