Synapse
⌘+K
Synapse
PulseExploreClubsResearchersJournals
Instagram
HomeClubsExplore
May 17, 2023Egyptian Journal of Medical Human GeneticsOpen Access

Genetic testing and family screening in idiopathic pediatric cardiomyopathy: a prospective observational study from a tertiary care center in North India

View Full Paper
Ask AI
Bookmark
Share

Key result

In 20 children with idiopathic cardiomyopathy, dilated cardiomyopathy was the most common morphology with a 44.4% mortality rate, and whole-exome sequencing identified pathogenic or likely pathogenic variants in 55% of tested patients.

Why the study?

Data on family screening and genetic testing in pediatric cardiomyopathy from India are limited.

Population

20 children (birth to 18 years of age) with cardiomyopathy at a tertiary care hospital in North India

Comparison

Family screening, multi-panel gene testing, and whole-exome sequencing

Design

Prospective observational study

Follow-up

Median 15 months

Authors

DBDheeraj Deo BhattSMSusi MathewsVAVanshika Ahuja

Discussion

Loading...

Member takes

Overview

High mortality in pediatric idiopathic DCM warrants close surveillance; small cohort leaves WES yield open to confirmation.

Study Design

Type

Observational (n=20)

Multicenter

No

Structured PICO

P
Population
20 children (median age 42 months, 50% female) with idiopathic pediatric cardiomyopathy, predominantly dilated cardiomyopathy, followed for a median of 15 months.
E
Exposure
Family screening and genetic testing (multi-panel gene testing in 18 patients, whole-exome sequencing in 9 patients)
O
Outcome
Morphologic spectrum, prevalence of familial cardiomyopathy, and identification of pathogenic/likely pathogenic genetic variants

In pediatric idiopathic cardiomyopathy in North India, dilated cardiomyopathy is the most common phenotype with high short-term mortality, and whole-exome sequencing may offer a higher diagnostic yield for genetic causes compared to multi-panel testing.

Limitations

  • Small sample size
  • Exclusion of viral myocarditis was based on history without myocardial biopsy or MRI
  • Investigations to rule out metabolic causes were not done for all patients
  • Whole-exome sequencing was not done for all patients and selection was arbitrary
  • Absence of a control population
  • Small cohort size
  • Clinical phenotype screening was done only in 61.1% of eligible first-degree relatives
  • Lack of access to medical records of relatives
  • Screening of relatives at only one point during the study
  • Study was not designed to compare whole-exome sequencing and genetic panel testing

Cite This Study

Bhatt et al. (2023) conducted an observational in Idiopathic pediatric cardiomyopathy (n=20). Genetic testing and family screening was evaluated on Identification of pathogenic or likely pathogenic variants via whole-exome sequencing. In 20 children with idiopathic cardiomyopathy, dilated cardiomyopathy was the most common morphology with a 44.4% mortality rate, and whole-exome sequencing identified pathogenic or likely pathogenic variants in 55% of tested patients.

synapsesocial.com/papers/6a984e3f3de45e4adad7e915https://doi.org/10.1186/s43042-023-00414-0
View Full Paper
Ask AI
Bookmark
Share

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Genetic Causes of Cardiomyopathy in Children: First Results From the Pediatric Cardiomyopathy Genes Study2021 · 71 citations
  2. 2Genetic Basis of Childhood Cardiomyopathy2022 · 65 citations
  3. 3Molecular analysis of dilated and left ventricular noncompaction cardiomyopathies in Egyptian children2021 · 8 citations
  4. 4Epidemiologic study of paediatric genetic cardiomyopathy in South Korea2025
  5. 5CARDIOGENETICS IN PEDIATRIC AGE: USES AND LIMITATIONS2026