This application addresses Challenge Area (04) Clinical Research and specific Challenge Topic 04-HL-114: Using existing datasets to plan effectiveness trials in pediatric cardiology. Most of the treatments used in children undergoing congenital heart surgery are not evidence-based. Treatment decisions are instead usually based on small observational studies, extrapolation from adult data, or clinician experience. We propose to link two rich data sources: The Society of Thoracic Surgeons Congenital Heart Surgery (STS-CHS) Database, which contains detailed clinical, diagnostic, operative, and outcomes data for children undergoing heart surgery, and the Pediatric Health Information System (PHIS) Database, which contains detailed information on medication utilization in children's hospitals. The linked data will include information on over 45,000 patients from 29 centers, which will be utilized to assess current variations in peri-operative treatment, and evaluate comparative effectiveness and safety of medications used in the peri-operative setting. This application will focus on investigating medications in 3 therapeutic areas: anti-fibrinolytic agents used during surgery, afterload reduction in the post-operative setting, and peri-operative antibiotic prophylaxis. Our overall hypothesis is that the linking and analysis of existing large pediatric data sources can address many of these current knowledge gaps and provide robust data in 3 specific areas: 1) identifying current practice patterns and variation in peri-operative care of children undergoing congenital heart surgery;2) performing observational comparative effectiveness studies which will provide data on efficacy and safety, as well as sample sizes, event rates, and drug effect to be used in planning subsequent trials;4) establishing a network of sites willing to participate in subsequent studies. Our research team has extensive experience in the analysis and linkage of large-scale clinical and administrative datasets and in conducting pediatric trials, and is uniquely suited to conduct the proposed study.

Public Health Relevance

Congenital heart defects are the most common birth defects and cause a large health burden resulting in >100,00 years of life lost each year and an estimated 6 billion dollars annually in acute care costs alone. However, most medications used to treat children with congenital heart disease have not been tested in them. We propose to link two rich data sources: The Society of Thoracic Surgeons Congenital Heart Surgery Database and the Pediatric Health Information System Database to evaluate the comparative effectiveness of medications used in children undergoing heart surgery and to plan future studies to benefit children with heart disease.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
NIH Challenge Grants and Partnerships Program (RC1)
Project #
Application #
Study Section
Special Emphasis Panel (ZRG1-PSE-C (58))
Program Officer
Pearson, Gail D
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
Duke University
Schools of Medicine
United States
Zip Code
Johnson, Jason N; Hornik, Christoph P; Li, Jennifer S et al. (2015) Response to letters regarding article, ""Cumulative radiation exposure and cancer risk estimation in children with heart disease"". Circulation 131:e419-20
Tunks, Robert D; Barker, Piers C A; Benjamin Jr, Daniel K et al. (2014) Sildenafil exposure and hemodynamic effect after Fontan surgery. Pediatr Crit Care Med 15:28-34
Pasquali, Sara K; He, Xia; Jacobs, Marshall L et al. (2014) Excess costs associated with complications and prolonged length of stay after congenital heart surgery. Ann Thorac Surg 98:1660-6
Pasquali, Sara K; Jacobs, Marshall L; He, Xia et al. (2014) Variation in congenital heart surgery costs across hospitals. Pediatrics 133:e553-60
Pasquali, Sara K; Peterson, Eric D; Jacobs, Jeffrey P et al. (2013) Differential case ascertainment in clinical registry versus administrative data and impact on outcomes assessment for pediatric cardiac operations. Ann Thorac Surg 95:197-203
Pasquali, Sara K; He, Xia; Jacobs, Marshall L et al. (2013) Hospital variation in postoperative infection and outcome after congenital heart surgery. Ann Thorac Surg 96:657-63
Pasquali, Sara K; Li, Jennifer S; He, Xia et al. (2012) Comparative analysis of antifibrinolytic medications in pediatric heart surgery. J Thorac Cardiovasc Surg 143:550-7
Pasquali, Sara K; Li, Jennifer S; He, Xia et al. (2012) Perioperative methylprednisolone and outcome in neonates undergoing heart surgery. Pediatrics 129:e385-91
Pasquali, Sara K; Jacobs, Jeffrey P; Shook, Gregory J et al. (2010) Linking clinical registry data with administrative data using indirect identifiers: implementation and validation in the congenital heart surgery population. Am Heart J 160:1099-104