Dr. Furth, who trained in Pediatrics and Pediatric Nephrology at Johns Hopkins, is seeking this mentored award to study clinical outcomes associated with treatment choices for pediatric end stage renal disease (ESRD), complete a PhD in Clinical Investigation and transition to an independent career in clinical research. Under the mentorship of Dr. Neil Powe, she received the NIDDK Renal Disease Epidemiology Training Grant, was appointed as an Assistant Professor in the Division of Pediatrics, co-authored several published papers, developed the proposed research plan and presented several abstracts at important National Pediatrics and Nephrology meetings. Over 4,600 children and adolescents in the U.S. live with ESRD, at an annual Medicare cost of 40,000 dollars per patient year. Despite tremendous gains in survival, their """"""""outcomes"""""""" in terms of mortality, hospitalizations, 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 pediatric patient mortality, hospitalization, growth and educational achievement. Furthermore, reliable measures of functional outcome, as an alternative measure of treatment effectiveness, have not been systematically 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 pediatric ESRD, and to test the use of a generic health questionnaire assessing functional health in adolescents with ESRD. We will achieve these aims through cross- sectional and longitudinal analyses of national data from the U.S. Renal Data System. We propose to identify how treatment with hemodialysis, peritoneal dialysis or transplantation and the facility's experience with children independently affect clinical outcomes. Additionally, we will assess the responsiveness of a generic adolescent health questionnaire in assessing changes in health status over time as an alternative outcome measure. This research proposal will provide an in- depth analysis of health outcomes, and will provide valuable information regarding optimal treatment choices for children with kidney failure. During this project, Dr. Furth will gain new skills and knowledge in the sophisticated research techniques of multivariate risk adjustment, analysis of longitudinal data and psychometric assessment. The combination of observational and experimental research will provide the basis for further prospective studies in pediatric ESRD, and will give Dr. Furth the tools she needs to develop into an independent clinical investigator in a nurturing academic environment.
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