Utility of genetic testing in pediatric epilepsy: Experience from a low to middle-income country

Fizza Akbar1, Raisa Saleh2, Salman Kirmani1

  • 1Department of Paediatrics and Child Health, Aga Khan University, Karachi, Pakistan.

Insights

Genetic testing for monogenic epilepsies in Pakistan found pathogenic variants in one-third of pediatric patients. This yield is comparable to North America, highlighting the need for South Asian genotype characterization.

Area of Science:

  • Genetics
  • Neurology
  • Pediatrics

Background:

  • Monogenic epilepsies are a key cause of pediatric epilepsy, increasingly diagnosed via genetic testing.
  • The effectiveness of genetic testing in low to middle-income countries (LMICs) remains under-investigated.

Purpose of the Study:

  • To evaluate the utility and yield of genetic testing for pediatric epilepsy in a LMIC setting.
  • To assess the diagnostic yield of next-generation sequencing (NGS) in patients with suspected monogenic epilepsy in Karachi, Pakistan.

Main Methods:

  • A retrospective review of 77 pediatric patients with suspected genetic epilepsy who underwent next-generation sequencing (NGS).
  • Variant classification using American College of Medical Genetics and Genomics (ACMG) guidelines, including reclassification of variants of uncertain significance (VUSs).

Main Results:

  • Initially, 27% of patients had pathogenic (P) or likely pathogenic (LP) results, increasing to 32% after VUS reclassification.
  • Post-reclassification, 25% had negative results, and 43% had VUSs; 66% initially had at least one VUS.
  • The diagnostic yield was comparable to North American cohorts, with a higher proportion of P/LP variants in *SCN1A*, including two novel variants.

Conclusions:

  • Genetic testing is effective for diagnosing pediatric epilepsy in LMICs, with a diagnostic yield comparable to high-income countries.
  • A significant proportion of VUSs initially complicates interpretation, underscoring the need for better characterization of the South Asian genotype.
  • Further research into population-specific genetic variations is crucial for improving diagnostic accuracy in diverse populations.