Response to amoxicillin and perampanel in infantile Alexander disease

Type I Alexander disease (AxD) presents with paroxysmal neurodegeneration, refractory epilepsy, and encephalopathy in the first years of life and is associated with a poor prognosis. Although there is no treatment, mild symptomatic improvement has been reported in one case of adult Alexander treated...

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Detalles Bibliográficos
Autores: Boronat, Susana|||0000-0001-7096-5578, Turon Viñas, Eulalia|||0000-0001-6171-0282, Mac Manus, N., Díaz-Gómez, Asunción|||0009-0000-1216-1133, Vicente, Mónica, Ros-Castelló, Victoria|||0000-0002-9523-3012, Sierra-Marcos, Alba|||0000-0001-6570-9318
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:308688
Acceso en línea:https://ddd.uab.cat/record/308688
https://dx.doi.org/urn:doi:10.1002/epi4.13077
Access Level:acceso abierto
Palabra clave:Alexander's disease
Case report
Glutamate toxicity
Perampanel
β-lactam antibiotics
Descripción
Sumario:Type I Alexander disease (AxD) presents with paroxysmal neurodegeneration, refractory epilepsy, and encephalopathy in the first years of life and is associated with a poor prognosis. Although there is no treatment, mild symptomatic improvement has been reported in one case of adult Alexander treated with ceftriaxone, given its interaction with the mutant glial fibrillary acid protein (GFAP) responsible for the disease's pathogenesis. We describe a patient presenting with irritability starting at 2 months of age, initially attributed to gastroesophageal reflux. A ventriculoperitoneal shunt was placed at 3 months of age due to hydrocephalus secondary to aqueduct stenosis detected through an MRI scan, but the irritability persisted. At 5 months, a new brain MRI was performed due to irritability worsening, onset of abnormal ocular movements and seizures. In addition genetic testing was performed. AxD was diagnosed due to the mutation c.716G.