The overall goals of this proposed work are to determine the comparative burden of acute disease exacerbations on child patient reported outcomes (cPROs), to track cPROs over time, and to validate new cPRO instruments in the Patient Reported Outcomes Measurement Information System (PROMIS) in children with sickle cell disease, asthma, and diabetes. Children with sickle cell disease, asthma, and diabetes experience acute exacerbations of their disease that often result in acute healthcare utilization that impacts their functioning. Repeated exacerbations and disease related complications contribute to the ongoing impact of the disease on the child. Additionally, acute disease exacerbations are more likely to occur in children who are uninsured, have public insurance, or have lower family income. Using a common metric to measure cPROs over time allows for comparison within and across disease cohorts and provides an opportunity to tailor care and interventions that optimize patient functioning for children with chronic disease. Newly released PROMIS measures allow for increased precision in the measurement of cPROs but there is a need for further validation in additional cohorts of children with chronic disease. In addition, it is unknown whether the new measures and PROMIS profiles are responsive to change, and if there is change, whether the change is clinically meaningful. To address these gaps, the following aims are proposed: 1a) To determine the trajectory of cPROs across and within cohorts of children with sickle cell disease, asthma, and diabetes and the association with repeated disease exacerbations, disease status, and socioeconomic deprivation, 1b) To compare the impact of an acute disease exacerbation on cPROs in children with sickle cell disease, asthma, and diabetes, 2) To determine the validity of the new Pain Behavior, Pain Quality, and Physical Health (Physical Activity, Physical Stress Experience, and Strength Impact ) domains in children with sickle cell disease, asthma, and diabetes, and 3) To determine the responsiveness and clinical meaning of change in cPROs of a PROMIS profile and the new PROMIS Pain Behavior, Pain Quality, and Physical Health (Physical Activity, Physical Stress Experience, and Strength Impact) domains in children with sickle cell disease, asthma, and diabetes.
| Flynn, Kathryn E; Kliems, Harald; Saoji, Nikita et al. (2018) Content validity of the PROMIS® pediatric family relationships measure for children with chronic illness. Health Qual Life Outcomes 16:203 |
