Ebstein Anomaly Cohort: Update Research Team Joseph Dearani Chris Knott-Craig Christian Pizarro Kimberly Holst Jim Meza test

EA Cohort Update Status Update Brief Cohort Review Next Steps Timeline for Patient Enrollment Planning ahead, facilitating site initiation

Since the last Work Weekend: Sick Kids’ REB Approval! Research Protocol Data Collection Forms Foundation of Echo CORE Lab

Cohort Overview: Research Questions Classification System Optimal Approach (timing, operation) Neonate Infant Child Early morbidity and mortality Late morbidity, reoperation, mortality Late functional status

Cohort Inclusion Criteria: Ebstein anomaly dx at CHSS member institution Neonate to 21 years old Neonate ≤ 1 month Pediatric > 1 month Operative and medically managed patients Exclude: AV-VA discordance HCM, PAPVC, AVSD, genetic syndromes Retrospective (Look Back to 2010) to Prospective

Imaging Impacts all aspects of this cohort Crucial to be accurate and uniform Echocardiogram Core Lab Pre-op TTE, intra-op TEE, post-op TTE 2D and 3D when available Fetal Echocardiograms when available MRI Need standard interpretation

1. Classification System Neonates (GOSE vs other) Fetal? Pediatric patients Correlation of anatomy: Echo MRI Surgery Predictors of valve repairability

1. Classification System, cont To establish prognostic models based on classification system RF for mortality prior to surgery (neonates) Predictors of successful medical management RF for mortality related to surgery Models by age

2. Optimal Management Neonates Current practice patterns Timing of operative intervention Patient anatomy Physiology Operative Approach 1V vs 2V Risk factors Transplant Mortality

2. Optimal Management, cont. Pediatric patients Current practice patterns Timing of operative intervention Physiology, symptoms Patient anatomy Operative Approach 1.5V vs 2V Predictors of valve repairability Optimal anti-arrhythmia procedures

3. Early morbidity and mortality Describe early outcomes Identify operative and perioperative RF Early morbidity Early mortality Determine modifiable risk factors to improve early outcome

4. Late morbidity, reop, and mortality Describe and determine predictors Late arrhythmia Right ventricular dysfunction Exercise intolerance Re-hospitalization Reoperation Risk factors Risk profile and outcomes: Reoperation vs. primary operation Transplant Late survival

5. Late functional status Accurately describe late functional status Exercise tests Quality of life Identify predictors of better/worse function Operative Medication Arrhythmia management

Questions regarding Research Protocol and/or Research Questions?

Moving Forward: Needed Steps BEFORE enrollment: Imaging Protocol Refinement of Data Collection Forms Database Construction Training Materials Imaging Study Coordinators Site Initiation Roll-Out Training IRB/REB

Imaging Protocol Establishment of Echo CORE group of Echocardiographers Welcome of additional cardiologists Establish Protocols: Image collection Data Collection Forms What images to review Fetal Echocardiograms when available Will require Sick Kids REB amendment

Data Collection Forms and Database Construction Review and make updates if needed Data Center Review Feasible to include in the database Database Construction Research Coordinator Review Feasible to send from individual institutions Changes will be submitted to Sick Kids REB

Training Materials and Roll-Out Imaging Team CORE team Institutional imaging teams Study Coordinators Study Enrollment and Protocol Training Data Collection IRB/REB Submission

Updated Timeline October 2016: Initial REB Approval Now: 1-3 months Establish CORE Lab and needed protocols Refine DCFs if needed Develop Training Materials REB Modifications: 2-3 months to review Database Build: 1 month Institutional Roll-Out: 1-2 months Regulatory process: 2-3 months Patient Enrollment: 6-9 months ..