Malformations of cortical development (MCD) such as Polymicrogyria (PMG) and Periventricular Nodular Heterotopia (PVNH) have genetic and non-genetic causes. They commonly present with a spectrum of clinical features beyond their structural brain abnormalities. Around 70-80% of individuals with PMG and PVNH experience seizures1,2, yet the factors underlying variability in seizure susceptibility and severity are unclear. Common polygenic epilepsy risk background has recently been shown to modify the clinical expression of rare pathogenic variants in monogenic epilepsies3. Here, we assessed evidence for polygenic modification of MCDs by testing for common variant epilepsy risk enrichment in PMG and PVNH.
Patients with polymicrogyria or periventricular nodular heterotopia (PVNH) on brain magnetic resonance imaging (MRI) were studied through the Epilepsy Phenome Genome Project4. All patients had epilepsy. Polygenic risk scores (PRS) were calculated for PMG (n=55), PVNH (n=49), and population controls (n=10,167) of European ancestry. PRS were generated with the PRS-CS-auto method5, using summary statistics from the largest all-epilepsy genome-wide association study (ILAE 2023)6. Statistical analyses used a fixed-effects regression model to account for sex and ancestry (top 10 principal components derived using PC-AiR).
PMG cases showed a nominally higher epilepsy PRS than population controls (mean 0.24 vs 0, OR = 1.30, P = 0.064). In contrast, epilepsy PRS did not differ in PVNH cases when compared to controls (mean -0.007 vs 0, P = 0.96).
These results suggest a modest enrichment of epilepsy-related polygenic risk in PMG that is not observed in PVNH, indicating potential differences in the genetic architecture underlying these MCDs. Common epilepsy-associated risk variants may contribute to the variable seizure expression seen in PMG. A replication analysis including >150 additional cases across three sites is underway, including cases with and without seizures to assess whether polygenic risk relates to cortical malformation itself or to seizure susceptibility.