General Clinical Research Center

Over 4,200 children and adolescents in the United States have end stage renal disease (ESRD) requiring dialysis, at an annual Medicare cost of $39,600 per patient year. Despite tremendous gains in survival for these children, their "outcomes" measured in terms of patient mortality, frequency of hospitalization and the development of comorbid conditions, remain poor. No national study has previously addressed how particular treatment regimens and clinical experience with ESRD care affect important patient outcomes such as mortality, hospitalization, growth and educational achievement. Furthermore, reliable measures of functional outcomes, as an alternative measure of treatment effectiveness, have not been systemically studied in children with ESRD. The long-term objectives of this proposal are to define patient, provider and treatment factors associated with optimal clinical outcomes in children with renal failure, and to test the use of a generic health questionnaire assessing functional and emotional health status in adolescents with renal disease. Through separate cross-sectional and longitudinal analyses of data from the U.S. Renal Data System (USRDS) on all U.S. children with renal failure in 1990, the investigators propose to identify whether ESRD particular treatment regimens (hemodialysis, peritoneal dialysis or transplantation) and the facility’s experience with pediatric patients independently affect clinical outcomes. Additionally, they will assess the reliability, validity and responsiveness of a generic health status questionnaire, the Child Health and Illness Profile (CHIP-AE), in measuring the health status of children with renal disease as an alternative outcome measure. This proposal will therefore provide an in-depth analysis of outcomes in pediatric ESRD, and will provide clinicians with valuable information regarding optimal treatment regimens and patient care for children and adolescents with renal disease. Such a study is necessary to allow parents and physicians to make rational treatment choices for this vulnerable group of children. During the institution of this protocol, the applicant will gain new skills and knowledge in the application of sophisticated research techniques such as multivariate risk adjustment, analysis of longitudinal data and psychometric assessment. This work will provide the basis for further studies in outcomes research in pediatric chronic disease, and will allow the applicant to develop into an independent investigator in a nurturing academic environment.