The Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy

Health-related quality of life (HRQoL) is defined as an individual s perception of his or her well-being and satisfaction with their current circumstances. As is oftentimes the case, in childhood-onset neuromuscular disease (NMD) and cerebral palsy (CP), no definitive cures currently exist. As a result, the primary goal of treatment for these patient populations is to improve quality of life or, more specifically, HRQoL. The medical community s interest in patient-based outcomes such as HRQoL has increased in the last two decades as it allows a multidimensional consideration of a patient s health. This focus has been the impetus for the development of computerized adaptive testing (CAT) instruments such as Quality of Life in Neurological Disorders (Neuro-QOL) and Patient Reported Outcomes Measurement Information System (PROMIS) to provide a means of assessing quality of life in patient populations with chronic diseases, such as neuromuscular disease and cerebral palsy.

The objective of this study is thus to validate the PROMIS and Neuro-QOL scales pediatric domains of self-assessment for individuals with CP and NMD and the PROMIS proxy domains for proxy-assessment of the same populations. The questionnaires will be administered to a combined total of 650 individuals from two sites in order to also calibrate the items and convert the questionnaires into CAT format.

The study population is patients with CP or NMD, ages 8-17 years, caregivers of patients with CP or NMD, whose children are ages 5-17 years. Caregivers are defined in this study as parents or legal guardians of children.

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The design is a prospective study in which patients and caregivers will complete online versions of the PROMIS and Neuro-QOL questionnaires at one of two different sites.