Screening Outcomes and Long-term Data VICTORIA l Vetter, M.D.,MPH

Using Data to Determine Outcomes Screening using ECGs is occurring. Over 430,566 children screened by PHW members in past 10 years. Can we do it better and if so, how do we standardize and improve what is being done currently? What can we learn from these data? Can current screening help determine normal ECG findings? Does screening find cardiac conditions? If Yes, what happens to those children? If No, what happens? Can screening identify safety signals? Can screening prevent sudden cardiac death? If Yes, what type of screening is best?

Screening Results DATA, DATA, DATA Personal History Family History ECG ECHO Physical Exam Other Testing 

Post-Screening Data Outcomes DATA SHARING Post-Screening Data Outcomes ECHO ECG H&P FAMILY PCP Foundations Pharma Industry FDA Academia

Testing Data: Initial Encounter Physical Exam Demographic Data History Murmur Blood Pressure Marfan Stigmata Brachial vs. Femoral pulses Height and Weight Personal History Chest Pain/Shortness of Breath Syncope Exercise Intolerance Activity Family History Sudden death, syncope, seizure Specific SCD associated condition ECG ECHO

Testing Data Outcomes ECG ECHO Normal values Modifying factors Age Sex Race Ethnicity Modifying factors Activity Body habitus/BSA/BMI Blood Pressure Normal values Age Sex Race Ethnicity Modifying factors Activity Body habitus/BSA/BMI Blood Pressure

Testing Data Abnormalities ECG ECHO Diagnosis: WPW Possible Diagnosis: LQTS Abnormal Finding: LVH Normal True negative False negative (CPVT, Coronary artery anomaly) Diagnosis: HCM, Anomalous Coronary Artery Origin Possible Diagnosis: HCM vs. Athletic Heart Normal True negative False negative (HCM, ARVD, LVNC) Long-term Data and Outcomes

Testing Data Abnormalities Long-term Data and Outcomes Follow Up Testing: Type and Results Follow Up Diagnoses Validate “Definitive” Diagnosis and determine Outcomes WPW or LQTS Clarify Diagnosis LVH: HCM or Athletic Heart or False positive? Follow Those with No Diagnosis Do well long term Have Cardiac Event Core Annual Data

Testing Data Abnormalities Long-term Data and Outcomes Core Longitudinal Data: Annual Questions Is the child alive? Is the child well or not? Has the child died? Did the child have any heart test in the past year? Does the child have a new heart condition? Was the child started on any heart medication? Did the child have a heart procedure? Did the child have a hospital stay in the past year? Has the child had seizures or passed out with exercise?

Core Longitudinal Data How To Obtain How to Store and Access Family Assisted Automation Social Media Clearinghouse Data Warehouse/Repository Data Transmission

NEXT STEPS: Long-term Results and Outcomes Determine where can data be stored and how can it be accessed. Need Manual of Operations (MOO) on how to code the variables. Need Policies and Procedures on cleaning, storage, and access to data. Need Process to obtain follow up/outcome data after initial screens. Discuss different needs of academia, the FDA, Industry/PhRMA and Foundation/Grass Roots Groups. Determine other outcomes that should be added. What other questions does the group have?

Thank You!

Screening Process Variation Who, What, Where? Screening Organization Screening Location Data Acquisition Individuals Screened Equipment and Personnel Test Interpretation: Criteria and Personnel

Screening Process Variation What is Done? History Personal History Family History ECG ECHO Physical Exam Genetics

STANDARDIZATION: FITTING THE PIECES TOGETHER