Outcomes of Hypertrophic Cardiomyopathy HCM Patients Listed for Transplant

1. Outcomes of Hypertrophic Cardiomyopathy (HCM) Patients Listed for Transplant R Gajarski MD*, D Naftel PhD, E Pahl MD, J Alejos MD, B Pearce MD, J Kirklin MD, M Zamberlan CPNP, A Dipchand** MD, PHTS Study Group Divisions of Pediatric Cardiology, University of Michigan*, Ann Arbor, MI and Hospital for Sick Children**, Toronto, ON

2. Relevant Financial Relationship Disclosure Statement Outcomes of Hypertrophic Cardiomyopathy (HCM) Patients Listed for Transplant R Gajarski MD, D Naftel PhD, E Pahl MD, J Alejos MD, B Pearce MD, J Kirklin MD, M Zamberlan CPNP, A Dipchand MD, PHTS Study Group I will not discuss off label use and/or investigational use of the following drugs/devices: No relevant financial relationships exist related to this presentation

3. Background Pediatric patients with HCM exhibit wide spectrum of clinical presentations variable clinical courses Findings at presentation asymptomatic hypertrophy malignant dysrhythmia severe outflow obstruction with heart failure

4. Background Outcomes from the PCMR database patients presenting at < 1 year of age poorest survival idiopathic HCM patients living to 1 year of age low annual mortality thereafter (~1%/yr) comparable to results in adult population-based studies Treatment for HCM medical (�-blockers, Ca++ blockers) surgical myectomy heart transplantation

5. Background Indications for heart transplant (HTx) ill-defined intractable life-threatening arrhythmia (with ICD) clinical symptoms non-responsive to medical therapy �burned out� HCM with end-stage heart failure Outcomes after listing for transplant have not been well characterized

6. Study Objectives 1) To determine early and late outcomes of HCM patients listed for HTx To identify potential risk factors which might adversely impact survival while listed or post-HTx

7. Methods Data obtained from the PHTS prospective, multi-institutional, event-driven data registry patients < 18 years of age listed for HTx between 1/93-12/06 Pre-listing clinical variables demographics diagnosis and age at listing gender, ethnicity history of arrhythmia, failure to thrive

8. Methods Clinical status at listing UNOS status use of inotropes mechanical support ventilator ECMO / VAD Outcomes successful HTx vital status

15. Causes of Death in Listed HCM Patients

20. Causes of Death in Transplanted HCM Patients

22. Conclusions HCM patients comprise the smallest proportion of children with CM listed for HTX HCM patients listed <1yr of age and UNOS status 1 had highest mortality awaiting HTx, but had survival similar to other groups once transplanted While a small cohort size may have contributed to statistical differences in long-term post-HTx survival between HCM and DCM pts, intermediate-term survival was unaffected by age and similar to that for other transplanted myopathies

23. Implications Given the limited donor pool, HTx for infants with HCM requires careful reconsideration Particularly among infants, more rigorous identification of key pre-HTx risk factors, such as underlying etiology, is necessary to ensure optimal post-HTx survival benefit