Hypertrophic Cardiomyopathy, Sudden Cardiac Death
Conditions
Keywords
Pediatric, Risk Prediction
Brief summary
This is a retrospective cohort study of pediatric hypertrophic cardiomyopathy (HCM) patients using chart and registry review methodology. The studies objective is to develop and validate a sudden cardiac death (SCD) risk calculator that is age-appropriate for children with HCM that includes clinical and genetic factors.
Interventions
None listed
Sponsors
Stanford University
Children's Hospital of Philadelphia
New York Presbyterian Hospital
University of Texas Southwestern Medical Center
University of Michigan
Children's Hospital Medical Center, Cincinnati
Provincial Health Services Authority British Columbia
Stollery Children's Hospital
Children's Hospital of Eastern Ontario
Royal Children's Hospital
Children's Healthcare of Atlanta
Primary Children's Hospital
Baylor College of Medicine
The Cleveland Clinic
Monroe Carell Jr. Children's Hospital at Vanderbilt
Children's Hospital Colorado
Indiana University Health
OHSU Doernbecher Children's Hospital
Children's Hospital Los Angeles
The Hospital for Sick Children
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* phenotype-positive patients diagnosed with hypertrophic cardiomyopathy * phenotype-negative, genotype positive patients considered at risk for developing hypertrophic cardiomyopathy
Exclusion criteria
* Neuromuscular, metabolic, syndromic (other than Noonan Syndrome and related RAS-opathies) or endocrine (including infants of diabetic mothers) causes of HCM * (Other treatable causes of left ventricular hypertrophy (systemic hypertension, anatomic defects causing left ventricular outflow tract obstruction e.g. aortic stenosis, subAS, subaortic membrane, coarctation)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With a Composite Sudden Cardiac Death Event | Time to a composite sudden cardiac death event during 5-year follow-up | The composite SCD event includes post diagnosis SCD, aborted SCD (including ventricular fibrillation, sustained ventricular tachycardia), primary ICD insertion with appropriate shock, secondary ICD insertion |
Participant flow
Recruitment details
This was a multicenter retrospective observational cohort study of pediatric patients with clinically diagnosed childhood-onset HCM. The primary cohort included patients from 11 sites participating in the PRIMaCY study (Precision Medicine for Cardiomyopathy), an international registry of patients with pediatric HCM launched in 2017. These included 4 Canadian, 6 US, and 1 Australian site. For model validation, we used data from the Sarcomeric Human Cardiomyopathy Registry.
Participants by arm
| Arm | Count |
|---|---|
| Hypertrophic Cardiomyopathy Patients included were those with a clinical diagnosis of HCM defined as having LV posterior wall or septal thickness z score ≥2, age \<18 years at the time of diagnosis, absence of a SCD event before diagnosis, and at least 1 follow-up assessment after diagnosis. | 572 |
| Total | 572 |
Baseline characteristics
| Characteristic | Hypertrophic Cardiomyopathy | — |
|---|---|---|
| Age, Continuous | 9.8 years | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment Australia | 64 participants | — |
| Region of Enrollment Canada | 273 participants | — |
| Region of Enrollment United States | 235 participants | — |
| Sex: Female, Male Female | 178 Participants | — |
| Sex: Female, Male Male | 394 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Number of Participants With a Composite Sudden Cardiac Death Event
The composite SCD event includes post diagnosis SCD, aborted SCD (including ventricular fibrillation, sustained ventricular tachycardia), primary ICD insertion with appropriate shock, secondary ICD insertion
Time frame: Time to a composite sudden cardiac death event during 5-year follow-up
Population: Patients with a clinical diagnosis of HCM defined as having an LV posterior wall or septal thickness z score ≥2, age \<18 years at the time of diagnosis, absence of a sudden cardiac death event before diagnosis, and at least 1 follow-up assessment after diagnosis.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Hypertrophic Cardiomyopathy | Number of Participants With a Composite Sudden Cardiac Death Event | 53 Participants |