Primary Investigator: Bradley S. Marino, MD, MPP, MSCE
Co-investigators: Dennis Drotar, PhD, Richard Ittenbach, PhD, Peter Margolis, MD, PhD, Michael Seid, PhD, Robert Beekman, MD
Congenital heart disease (CHD) is the most common defect in children. Over the last several decades, new surgical techniques and advances in cardiopulmonary bypass (CPB), intensive care, cardiac catheterization, heart transplantation, imaging modalities, and medical therapies have improved survival and prolonged the lives of children and adolescents with CHD. Operative mortality for children with the most complex CHD is now less than 10%. This has changed the focus of clinical research on the pediatric cardiac population from short-term surgical survival to the assessment of short- and long-term morbidity. The hemodynamic effects of the specific heart defect and the medical and surgical therapy received by the child can result in significant morbidity. The child’s neurodevelopmental, psychosocial, and physical functioning are impacted by these morbidities and may adversely affect the child’s quality of life (QOL).
Quality of life refers to the impact of a specific illness or medical therapy on the ability of the child to function in situational contexts (e.g., family, school, peers) and to draw personal satisfaction from a physical, psychological, and social functioning perspective. In the past, quantitative assessment of QOL in the pediatric cardiac population has been limited due to the wide age range of children, varying and developing neurodevelopmental and psychosocial capabilities, the variety of underlying disease processes and treatment modalities, and the spectrum of outcomes. Disease specific QOL instruments are more comprehensive for a specific disease, more sensitive to change in condition over time and a better discriminator of differences in subgroups within a disease category. Existing disease-specific pediatric cardiac instruments were limited by lack of patient and parent reporting, narrow age range, inadequate generalizability data, and poor discrimination between subgroups in the pediatric cardiac population.
To address the limitations, our research team developed the Pediatric Cardiac Quality of Life Inventory (PCQLI) in 2004. The PCQLI is a self-administered, reliable and valid, disease-specific questionnaire that quantitatively assesses health-related QOL in children (age 8-12) and adolescents (age 13-18) with congenital and acquired heart disease. Over the last four years, this tool has undergone extensive reliability, validity, and generalizability testing in a multi-center, multi-national testing trial at 11 centers in the United States and 3 centers in the United Kingdom. To date, over 1,500 patients and their parents (>3,000 respondents) have participated in this study. This study is currently funded by an NICHD K23 award (PI: Brad Marino, MD) and by a local CCHRF grant award.
Data from the PCQLI Testing Trial has shown that higher disease complexity is associated with lower QOL score in the CHD population and that increasing medical care utilization is associated with a lower QOL. However, the analysis also showed that QOL score varied significantly within specific diagnostic sub-groups in the CHD population. Given the variability in QOL score, our team tested for demographic and medical predictors of QOL, and found that they account for only a small portion of the variability in QOL scores in these children. We hypothesize that neurodevelopmental, psychosocial, and physical functioning morbidity factors account for some of this unexplained variability in QOL score. Currently, we are conducting a study looking at how specific psychosocial morbidity factors (post-traumatic stress disorder symptomatology, trait anxiety, parental stress, and family functioning) mediate the association between CHD complexity and QOL score. In addition, we are initiating a study to assess the association between neurodevelopmental outcome (intelligence; academic achievement; neuropsychological functioning) and QOL. The overall, long term goal of our research team is to develop and test comprehensive biological (e.g. cardiovascular anatomic, hemodynamic, physical functioning, and surgical variables), neurodevelopmental, and psychosocial models that will reveal modifiable predictors of lower QOL in children with CHD, and to create new early opportunities for prevention and intervention to improve QOL for children and their families.
