SSA Hearing on Compassionate Allowances

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SSA Hearing on Compassionate Allowances

• Janet N Scheel MD
• November 9,2010

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Cardiomyopathy

• Restrictive cardiomyopathy
• Hypertrophic cardiomyopathy
• Dilated cardiomyopathy

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(No Transcript)
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Frank Starling Curve
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Causes of DCM in Children

• Genetic
• Infectious
• Metabolic
• Arrhythmias

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Causes of DCM in Children

• Inflammatory
• Nutritional
• Structural heart disease
• Chemotherapy

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CHF symptoms in Adults
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CHF Symptoms in ChildrenRoss Classification

• Class I- no symptoms
• Class II-Mild tachypnea or diaphoresis with
  feedings/exertion. No growth failure
• Class III-Marked tachypnea or diaphoresis with
  feedings/exertionprolonged feeding timegrowth
  failure
• Class IV-Symptomatic at rest

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Treatment options

• Oral medical therapy
• IV inotropes
• Pacing
• ECMO/VAD
• Transplant

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ECMO
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ECMO
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Long term devices specific for children
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Selection for Pediatric Heart Transplant

• End stage congenital heart disease not amenable
  to surgical or medical therapy
• Ross Classification III-IV
• Failure to thrive
• Protein losing enteropathy
• Intractable arrhythmias
• Plastic bronchitis

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Selection for Pediatric Heart Transplant

• Dilated Cardiomyopathy symptomatic on maximal
  medical therapy
• Restrictive Cardiomyopathy

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Exclusion Criteria

• Genetic syndrome with poor long term prognosis
• Neurologic abnormalities with poor long term
  prognosis
• Irreversible end-organ damage
• Socio-economic factors leading to poor long term
  compliance

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Exclusion Criteria

• Genetic syndrome with poor long term prognosis
• Neurologic abnormalities with poor long term
  prognosis
• Irreversible end-organ damage
• Socio-economic factors leading to poor long term
  compliance

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Exclusion Criteria

• Pulmonary Hypertension (gt5-6 woods units)
• Unresponsive to oxygen or pulmonary vasodilators
• Transpulmonary gradient gt 15mmHg
• Pulmonary vein stenosis
• Active infection
• Active malignancy

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AGE DISTRIBUTION OF PEDIATRIC HEART
RECIPIENTSBy Year of Transplant
NOTE This figure includes only the heart
transplants that are reported to the ISHLT
Transplant Registry. As such, this should not be
construed as evidence that the number of hearts
transplanted worldwide has increased and/or
decreased in recent years.
ISHLT
2009

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PEDIATRIC HEART TRANSPLANTATIONKaplan-Meier
Survival (Transplants 1/1982-6/2007)
ISHLT
2009

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PEDIATRIC HEART TRANSPLANTATIONConditional
Kaplan-Meier Survival (Transplants
1/1982-6/2007)
ISHLT
2009

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PEDIATRIC HEART TRANSPLANTATIONConditional
Kaplan-Meier Survival for Recent Era
(Transplants 1/1999-6/2007)
ISHLT
2009

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PEDIATRIC HEART TRANSPLANTS (1/1995-6/2007)Risk
Factors For 1 Year Mortality
N3,756
ISHLT
2009
Reference diagnosis cardiomyopathy

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PEDIATRIC HEART RECIPIENTS Functional Status of
Surviving Recipients(Follow-ups April 1994 -
June 2008)
ISHLT
2009

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PEDIATRIC HEART RECIPIENTS Functional Status of
Surviving Recipients(Follow-ups April 1994 -
June 2008)For the Same Patients
ISHLT
2009

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PEDIATRIC HEART RECIPIENTS Rehospitalization
Post-transplant of Surviving Recipients(Follow-up
s April 1994 - June 2008)
ISHLT
2009

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PEDIATRIC HEART RECIPIENTS Maintenance
Immunosuppression at Time of Follow-up for Same
Patients at Each Time Point(Follow-ups January
2001 - June 2008)
of Patients
ISHLT
Analysis is limited to patients who were alive at
the time of the follow-up
2009

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FREEDOM FROM CORONARY ARTERY VASCULOPATHY For
Pediatric Heart Recipients (Follow-ups April
1994 - June 2008)
ISHLT
2009

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GRAFT SURVIVAL FOLLOWING REPORT OF CORONARY
ARTERY VASCULOPATHY For Pediatric Heart
Recipients (Follow-ups April 1994 - June
2008)Stratified by Age Group
ISHLT
2009

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FREEDOM FROM SEVERE RENAL DYSFUNCTIONFor
Pediatric Heart Recipients (Follow-ups April
1994 - June 2008)
ISHLT
2009

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MALIGNANCY POST-HEART TRANSPLANTATION FOR
PEDIATRICSCumulative Prevalence in Survivors
(Follow-ups April 1994 - June 2008)
Malignancy/Type Malignancy/Type 1-Year Survivors 5-Year Survivors 10-Year Survivors
No Malignancy No Malignancy 3,361 (98.1) 1,343 (95.2) 332 (92.2)
Malignancy (all types combined) Malignancy (all types combined) 64 (1.9) 68 (4.8) 28 (7.8)
Malignancy Type Lymph 59 64 26
Malignancy Type Other 4 5 2
Malignancy Type Skin 1
Malignancy Type Type Not Reported 1
NOTE Multiple types may be reported sum of
types may be greater than total number with
malignancy.
ISHLT
2009

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FREEDOM FROM MALIGNANCYFor Pediatric Heart
Recipients (Follow-ups April 1994 - June 2008)
ISHLT
2009

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PEDIATRIC HEART RECIPIENTS Incidence of
Hypertension between 1 and 3 Years(Transplants
April 1993 - June 2005)
Maintenance Immunosuppression at discharge and 1 year HTN reported between 1 and 3 years HTN reported between 1 and 3 years P-value
Maintenance Immunosuppression at discharge and 1 year For Patients on drug For Patients not on drug P-value
Azathioprine 22.0 26.0 0.1643
Cyclosporine 21.4 26.2 0.1083
MMF 23.4 22.7 0.8049
Prednisone 29.6 10.7 lt.0001
Rapamycin 37.5 22.5 - 
Tacrolimus 28.8 20.3 0.0039
ISHLT
2009

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PEDIATRIC HEART RECIPIENTS Incidence of
Hypertension between 3 and 8 Years(Transplants
April 1993 - June 2000)
Maintenance Immunosuppression at discharge and 1 year HTN reported between 3 and 8 years HTN reported between 3 and 8 years P-value
Maintenance Immunosuppression at discharge and 1 year For Patients on drug For Patients not on drug P-value
Azathioprine 37.5 48.9 0.1623
Cyclosporine 36.5 43.8 0.4302
MMF 41.4 37.6 0.6949
Prednisone 47.1 20.2 lt.0001
Rapamycin . 37.6 - 
Tacrolimus 42.3 34.8 0.4484
ISHLT
2009

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PEDIATRIC HEART TRANSPLANT RECIPIENTS Cause of
Death (Deaths January 1992 - June 2008)
CAUSE OF DEATH 0-30 Days (N 461) 31 Days - 1 Year (N 421) gt1 Year - 3 Years (N 307) gt3 Years - 5 Years (N 226) gt5 Years - 10 Years (N 350) gt10 Years (N 172)
CORONARY ARTERY VASCULOPATHY 5 (1.1) 30 (7.1) 62 (20.2) 69 (30.5) 98 (28.0) 49 (28.5)
ACUTE REJECTION 44 (9.5) 100 (23.8) 71 (23.1) 31 (13.7) 45 (12.9) 10 (5.8)
LYMPHOMA 10 (2.4) 12 (3.9) 6 (2.7) 33 (9.4) 11 (6.4)
MALIGNANCY, OTHER 4 (1.0) 2 (0.7) 1 (0.4) 5 (1.4) 11 (6.4)
CMV 1 (0.2) 11 (2.6) 1 (0.3)
INFECTION, NON-CMV 54 (11.7) 65 (15.4) 20 (6.5) 8 (3.5) 17 (4.9) 13 (7.6)
PRIMARY FAILURE 102 (22.1) 23 (5.5) 10 (3.3) 15 (6.6) 18 (5.1) 5 (2.9)
GRAFT FAILURE 97 (21.0) 45 (10.7) 62 (20.2) 53 (23.5) 74 (21.1) 44 (25.6)
TECHNICAL 27 (5.9) 3 (0.7) 2 (0.7) 2 (0.9) 4 (1.1) 1 (0.6)
OTHER 25 (5.4) 26 (6.2) 29 (9.4) 24 (10.6) 30 (8.6) 10 (5.8)
MULTIPLE ORGAN FAILURE 46 (10.0) 54 (12.8) 11 (3.6) 6 (2.7) 10 (2.9) 8 (4.7)
RENAL FAILURE 1 (0.2) 4 (1.0) 1 (0.3) 1 (0.4) 1 (0.3) 3 (1.7)
PULMONARY 29 (6.3) 30 (7.1) 15 (4.9) 8 (3.5) 8 (2.3) 5 (2.9)
CEREBROVASCULAR 30 (6.5) 16 (3.8) 9 (2.9) 2 (0.9) 7 (2.0) 2 (1.2)
ISHLT
2009

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PEDIATRIC HEART TRANSPLANT RECIPIENTS Cause of
Death (Deaths January 1998 - June 2008)
CAUSE OF DEATH 0-30 Days (N 213) 31 Days - 1 Year (N 241) gt1 Year - 3 Years (N 192) gt3 Years - 5 Years (N 153) gt5 Years - 10 Years (N 286) gt10 Years (N 165)
CAV 2 (0.9) 14 (5.8) 33 (17.2) 43 (28.1) 77 (26.9) 47 (28.5)
ACUTE REJECTION 22 (10.3) 45 (18.7) 36 (18.8) 23 (15.0) 36 (12.6) 10 (6.1)
LYMPHOMA 6 (2.5) 7 (3.6) 4 (2.6) 28 (9.8) 11 (6.7)
MALIGNANCY, OTHER 1 (0.4) 1 (0.5) 4 (1.4) 10 (6.1)
CMV 7 (2.9) 1 (0.5)
INFECTION, NON-CMV 26 (12.2) 31 (12.9) 11 (5.7) 3 (2.0) 13 (4.5) 11 (6.7)
PRIMARY FAILURE 44 (20.7) 9 (3.7) 4 (2.1) 6 (3.9) 10 (3.5) 5 (3.0)
GRAFT FAILURE 31 (14.6) 25 (10.4) 48 (25.0) 44 (28.8) 66 (23.1) 42 (25.5)
TECHNICAL 14 (6.6) 2 (1.0) 4 (1.4) 1 (0.6)
OTHER 19 (8.9) 20 (8.3) 24 (12.5) 17 (11.1) 26 (9.1) 10 (6.1)
MULTIPLE ORGAN FAILURE 27 (12.7) 40 (16.6) 10 (5.2) 5 (3.3) 8 (2.8) 8 (4.8)
RENAL FAILURE 4 (1.7) 1 (0.5) 1 (0.7) 1 (0.3) 3 (1.8)
PULMONARY 11 (5.2) 27 (11.2) 10 (5.2) 6 (3.9) 7 (2.4) 5 (3.0)
CEREBROVASCULAR 17 (8.0) 12 (5.0) 4 (2.1) 1 (0.7) 6 (2.1) 2 (1.2)
ISHLT
2009

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PEDIATRIC HEART TRANSPLANT RECIPIENTS Relative
Incidence of Leading Causes of Death (Deaths
January 1998 - June 2008)
ISHLT
2009