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SSA Hearing on Compassionate Allowances. Janet N Scheel MD November 9,2010. Cardiomyopathy. Restrictive cardiomyopathy Hypertrophic cardiomyopathy Dilated cardiomyopathy*. Frank Starling Curve. Causes of DCM in Children. Genetic Infectious Metabolic Arrhythmias.

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ssa hearing on compassionate allowances

SSA Hearing on Compassionate Allowances

Janet N Scheel MD

November 9,2010

cardiomyopathy
Cardiomyopathy
  • Restrictive cardiomyopathy
  • Hypertrophic cardiomyopathy
  • Dilatedcardiomyopathy*
causes of dcm in children
Causes of DCM in Children
  • Genetic
  • Infectious
  • Metabolic
  • Arrhythmias
causes of dcm in children6
Causes of DCM in Children
  • Inflammatory
  • Nutritional
  • Structural heart disease
  • Chemotherapy
chf symptoms in children ross classification
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/exertion;prolonged feeding time;growth failure
  • Class IV-Symptomatic at rest
treatment options
Treatment options
  • Oral medical therapy
  • IV inotropes
  • Pacing
  • ECMO/VAD
  • Transplant
selection for pediatric heart transplant
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
selection for pediatric heart transplant14
Selection for Pediatric Heart Transplant
  • Dilated Cardiomyopathy –symptomatic on maximal medical therapy
  • Restrictive Cardiomyopathy
exclusion criteria
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
exclusion criteria16
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
exclusion criteria17
Exclusion Criteria
  • Pulmonary Hypertension (>5-6 woods units)
    • Unresponsive to oxygen or pulmonary vasodilators
    • Transpulmonary gradient > 15mmHg
  • Pulmonary vein stenosis
  • Active infection
  • Active malignancy
age distribution of pediatric heart recipients by year of transplant
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

pediatric heart transplantation kaplan meier survival transplants 1 1982 6 2007
PEDIATRIC HEART TRANSPLANTATIONKaplan-Meier Survival(Transplants: 1/1982-6/2007)

ISHLT

2009

pediatric heart transplantation conditional kaplan meier survival transplants 1 1982 6 2007
PEDIATRIC HEART TRANSPLANTATIONConditional Kaplan-Meier Survival (Transplants: 1/1982-6/2007)

ISHLT

2009

slide21
PEDIATRIC HEART TRANSPLANTATIONConditional Kaplan-Meier Survival for Recent Era (Transplants: 1/1999-6/2007)

ISHLT

2009

pediatric heart transplants 1 1995 6 2007 risk factors for 1 year mortality
PEDIATRIC HEART TRANSPLANTS (1/1995-6/2007)Risk Factors For 1 Year Mortality

N=3,756

ISHLT

2009

Reference diagnosis = cardiomyopathy

slide23
PEDIATRIC HEART RECIPIENTSFunctional Status of Surviving Recipients(Follow-ups: April 1994 - June 2008)

ISHLT

2009

slide24
PEDIATRIC HEART RECIPIENTSFunctional Status of Surviving Recipients(Follow-ups: April 1994 - June 2008)For the Same Patients

ISHLT

2009

slide25
PEDIATRIC HEART RECIPIENTS Rehospitalization Post-transplant of Surviving Recipients(Follow-ups: April 1994 - June 2008)

ISHLT

2009

slide26

PEDIATRIC HEART RECIPIENTSMaintenance Immunosuppression at Time of Follow-up for Same Patients at Each Time Point(Follow-ups: January 2001 - June 2008)

% of Patients

ISHLT

2009

Analysis is limited to patients who were alive at the time of the follow-up

slide27
FREEDOM FROM CORONARY ARTERY VASCULOPATHYFor Pediatric Heart Recipients (Follow-ups: April 1994 - June 2008)

ISHLT

2009

slide28

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

ISHLT

2009

slide29
FREEDOM FROM SEVERE RENAL DYSFUNCTION*For Pediatric Heart Recipients(Follow-ups: April 1994 - June 2008)

ISHLT

2009

slide30
MALIGNANCY POST-HEART TRANSPLANTATION FOR PEDIATRICSCumulative Prevalence in Survivors (Follow-ups: April 1994 - June 2008)

NOTE: Multiple types may be reported; sum of types may be greater than total number with malignancy.

ISHLT

2009

freedom from malignancy for pediatric heart recipients follow ups april 1994 june 2008
FREEDOM FROM MALIGNANCYFor Pediatric Heart Recipients(Follow-ups: April 1994 - June 2008)

ISHLT

2009

slide32
PEDIATRIC HEART RECIPIENTSIncidence of Hypertension between 1 and 3 Years(Transplants: April 1993 - June 2005)

ISHLT

2009

slide33
PEDIATRIC HEART RECIPIENTSIncidence of Hypertension between 3 and 8 Years(Transplants: April 1993 - June 2000)

ISHLT

2009

pediatric heart transplant recipients cause of death deaths january 1992 june 2008
PEDIATRIC HEART TRANSPLANT RECIPIENTS:Cause of Death (Deaths: January 1992 - June 2008)

ISHLT

2009

pediatric heart transplant recipients cause of death deaths january 1998 june 2008
PEDIATRIC HEART TRANSPLANT RECIPIENTS:Cause of Death (Deaths: January 1998 - June 2008)

ISHLT

2009

slide36
PEDIATRIC HEART TRANSPLANT RECIPIENTS:Relative Incidence of Leading Causes of Death(Deaths: January 1998 - June 2008)

ISHLT

2009