1. MCS in Special Populations: The Use of Mechanical Support in Adults with Congenital Heart Disease 9 th Annual Meeting May 15, 2015 Christina VanderPluym,MD Medical Director VAD Program Boston Children’s Hospital

2. Relevant Financial Relationship Disclosure Statement I have no significant or relevant financial disclosures related to the content of this presentation.. Christina VanderPluym M.D.

3. The Unknowns of VAD support for ACHD 1.How many adults with CHD need mechanical circulatory support? Who needs it and why? 2.How many adults with CHD are being supported on MCS as BTT, BTR and DT? 3.What are the outcomes of different variants of CHD with MCS as BTT and DT? Is DT possible for ACHD? 4.What are the obstacles and challenges to MCS in adults with CHD? Are they surmountable?

4. The Unknowns of VAD support for ACHD 1.How many adults with CHD need mechanical circulatory support? Who needs it and why? 2.How many adults with CHD are being supported on MCS as BTT, BTR and DT? 3.What are the outcomes of different variants of CHD with MCS as BTT and DT? Is DT possible for ACHD? 4.What are the obstacles and challenges to MCS in adults with CHD? Are they surmountable?

5. Adult Congenital Heart Disease Wide range of hemodynamic abnormalities: – Biventricular pressure and volume overload lesions (d- TGA, L-TGA, AVSD, TOF, etc.) – Univentricular failure (palliated Fontan circulation) Multiple clinical presentations: 1.Diagnosed and palliated in childhood 2.Diagnosed in adulthood with late sequelae of untreated hemodynamic abnormalities Lesions specific complications and natural morbidities of aging (hypertension, diabetes, hyperlipidemia)

6. Scope of the Problem? Prevalence of ACHD in difficult to characterize – No database of systematically collected population data, CDC currently funding 3 pilot projects for ACHD surveillance Based on Canadian studies, est. ~850 000 adults with CHD in US in 2000, with 5% increase annually, resulting in >1.3 million US adults by 2014 Currently, more adults with CHD than children Unclear what percentage of these patients progress to end stage heart failure necessitating MCS [ Hoffman et al. Am Heart J. 2004, Bhatt et al. Circ 2015]

7. Incidence of heart failure in adults with CHD after cardiac surgery [Norozi et al. Am J Cardiol 2006]

8. The Unknowns of VAD support for ACHD 1.How many adults with CHD need mechanical circulatory support? Who needs it and why? 2.How many adults with CHD are being supported on MCS as BTT, BTR and DT? 3.What are the outcomes of different variants of CHD with MCS as BTT and DT? Is DT possible for ACHD? 4.What are the obstacles and challenges to MCS in adults with CHD? Are they surmountable?

9. How many ACHD are being supported on MCS? OPTN SRTR data from 1987-2012 – 47 160 adult transplant recipients, 1213 (2.6%) had CHD – Proportion of ACHD transplants recipients relative to all adult transplant increased yearly, with concomitant increase in MCS for ACHD yearly – MCS used in 83 patients (6.8%) with CHD as compared to 8625 (18.8%) patients without CHD – No difference in 30d mortality between MCS and non MCS patients with ACHD, but both had higher short term mortality than adults without CHD [Maxwell et al. European J of Cardio-thoracic Surg 2013]

10. Proportion of ACHD transplants relative to all transplants Proportion of ACHD transplants supported With MCS relative to all ACHD 30d mortality rate by year of transplant for all ACHD patients [Maxwell et al. European J of Cardio-thoracic Surg 2013]

11. DATA SUMMARY 2008-2014 ACHD AND MCS

12. Intermacs Data Inclusions:  Primary LVAD/BiVAD/ TAH Implants  Adults (age ≥ 19 years at implant)  Implant dates: April 21, 2008 – December 31, 2014  Follow-up date: December 31, 2014 Study Group  Patients: 13288  Patient years:16730.83  Total deaths with a device in place: 3638  Total heart transplants: 3864  Total device removal due to recovery: 171  Mean Follow-up (Months): 15.11  Total number of contributing hospitals:154

13. Distribution of CHD and no CHD by device type: Continuous and Pulsatile Devices 5.7%

14. Distribution of Device Type in ACHD Continuous Flow – 59 patients (78%) 95% were LVADs 5% were BiVADs Pulsatile Flow – 17 patients (22%) 47% (8) Total artificial heart 35% (6) BiVAD 18% (3) LVAD Similar distribution to adults without CHD Adults without CHD had 49% with LVAD and only 24% with TAH

15. Demographics Age distribution of patients with CHD and without CHD in Intermacs Younger population requiring VAD support as compared to adults without CHD

16. Distribution of Device Strategy for ACHD and non CHD patients

17. Survival of adults with and without CHD on all devices: June 2006- April 2014 Not Congenital, n=13212; deaths=3613

18. Survival of adults with and without CHD on CF device

19. Survival of adults with and without CHD on pulsatile flow device

21. The Unknowns of VAD support for ACHD 1.How many adults with CHD need mechanical circulatory support? Who needs it and why? 2.How many adults with CHD are being supported on MCS as BTT, BTR and DT? 3.What are the outcomes of different variants of CHD with MCS as BTT and DT? Is DT possible for ACHD? 4.What are the obstacles and challenges to MCS in adults with CHD? Are they surmountable?

22. Challenges of MCS in ACHD Difficult to analyze and understand outcomes of VAD support for ACHD given the heterogeneity of palliated anatomic lesions Physiologic burden of ACHD results in ?irreversible end organ compromise – Liver cirrhosis and coagulopathy – Ascites, compromised nutrition and cachexia with consequent poor wound healing Technical challenges of cannula positioning, reconfiguring anatomy for systemic VAD (ie Fontan to Glenn) – Lessons from pediatric congenital VAD surgeons

23. Expanded data capture of CHD Pre-implant (primary diagnosis)

24. Thank you