M-cPROs Sickle Cell Disease Project
Sickle cell disease, an autosomal recessive disease diagnosed at birth in the United States, affects one in 400 black infants. Patients with sickle cell disease have 2.6 acute care encounters/year due to pain and experience the highest rate of rehospitalization (33.4%) of any other condition, highlighting the significant burden this disease has on individuals and on the healthcare system. Further, the average length of hospital stay for patients with vasoocclusive painful episodes is four days; this contributes to the burden on both patients and health systems.
The overall objective of the Midwest Child Patient Reported Outcomes (M-cPROs) Consortium is to advance the understanding and measurement of cPROs in children with chronic disease using the Patient Reported Outcomes Measurement Information System® (PROMIS®) to allow for widespread use in both clinical care and research to optimize the well-being and functioning of the child. To achieve this objective, the scientific research projects within the M-cPROs Consortium aim to: 1) improve outcomes that matter to children with chronic disease by: understanding the impact of disease factors, including acute disease exacerbations on patient pain behavior, pain quality, physical activity, physical stress experience, strength impact, and family relationships, assessing the relationship between the family relationships measure and other cPROs, and evaluating the impact of environmental stressors (e.g., socioeconomic deprivation) on patient pain behavior, pain quality, physical activity, physical stress experience, strength impact, and family relationships; 2) validate new PROMIS measures in multiple chronic disease populations to support a common metric for cPROs measurement in children with chronic disease; and 3) determine whether PROMIS measures are responsive to change in health and whether that change is clinically relevant in children with chronic disease.
The project consists of a prospective observational cohort study of sickle cell disease patients aged 5-17 years who will be followed for up to three years. We will also study sickle cell disease patients with disease exacerbations who present to the emergency department. The PROMIS measures that will be used include the Pediatric Profile 25 and the new PROMIS measures (pain behavior, pain quality, physical activity, physical stress experience, strength impact, and family relationship.
