Phenotypes and genotypes in outbred and inbred Primary microcephaly:
high incidence of epilepsy
Abstract
Primary microcephaly (PM) is defined as a significant reduction in
occipito-frontal circumference (OFC) of prenatal onset. Clinical and
genetic heterogeneity of PM represents a diagnostic challenge. We
performed detailed phenotypic and genomic analyses in a large cohort
(n=169) of patients referred for PM, and could establish a molecular
diagnosis in 38 patients. Pathogenic variants in ASPM and WDR62 were the
most frequent causes in non-consanguineous patients in our cohort. In
consanguineous patients, microarray and targeted gene panel analyses
reached a diagnostic yield of 67%, which contrasts with a much lower
rate in outbred patients (9%). Our series includes 15 previously
unreported families and 11 novel pathogenic variants, and we identify
novel candidate genes including IGF2BP3, DNAH2, and TSR1. We confirm
progression of microcephaly over time in affected children. Epilepsy was
an important associated feature in our PM cohort, affecting 34% of
patients, with various degrees of severity and seizure types. Our
findings will help to prioritize genomic investigations, accelerate
molecular diagnoses and improve management of PM patients.