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DOI 10.1002/epi4.12525

Медицинская библиотека · Epilepsia open · 2021

Late diagnoses of Dravet syndrome: How many individuals are we missing?

Silvennoinen Katri, Puvirajasinghe Clinda, Hudgell Kirsty, Sidhu Meneka K, Martins Custodio Helena, Genomics England Research Consortium, Jones Wendy D, Balestrini Simona, Sisodiya Sanjay M

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Аннотация

We report new genetic diagnoses of Dravet syndrome in a group of adults with complex epilepsy of unknown cause, under follow-up at a tertiary epilepsy center. Individuals with epilepsy and other features of unknown cause from our unit underwent whole-genome sequencing through the 100 000 Genomes Project. Virtual gene panels were applied to frequency-filtered variants based on phenotype summary. Of 1078 individuals recruited, 8 (0.74%) were identified to have a pathogenic or likely pathogenic variant in SCN1A. Variant types were as follows: nonsense (stopgain) in five (62.5%) and missense in three (37.5%). Detailed review of childhood history confirmed a phenotype compatible with Dravet syndrome. Median age at genetic diagnosis was 44.5 years (range 28-52 years). Tonic-clonic seizures were ongoing in all despite polytherapy including valproate. All had a history of fever sensitivity and myoclonic seizures, which were ongoing in two (25%) and three (37.5%) individuals, respectively. Salient features of Dravet syndrome may be less apparent in adulthood, making clinical diagnosis difficult. Regardless of age, benefits of a genetic diagnosis include access to syndrome-specific treatment options, avoidance of harmful drugs, and monitoring for common complications.

Для цитирования: Silvennoinen Katri, Puvirajasinghe Clinda, Hudgell Kirsty, Sidhu Meneka K, Martins Custodio Helena, Genomics England Research Consortium, Jones Wendy D, Balestrini Simona, Sisodiya Sanjay M. Late diagnoses of Dravet syndrome: How many individuals are we missing?. Epilepsia open. 2021. DOI: 10.1002/epi4.12525.

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