• Media type: E-Article
  • Title: Hereditary hemorrhagic telangiectasia associated with cortical development malformation due to a start loss mutation in ENG
  • Contributor: Villa, Davide; Cinnante, Claudia; Valcamonica, Gloria; Manenti, Giulia; Lanfranconi, Silvia; Colombi, Annalisa; Ghione, Isabella; Saetti, Maria Cristina; D’Amico, Mario; Bonato, Sara; Bresolin, Nereo; Comi, Giacomo Pietro; Ronchi, Dario
  • imprint: Springer Science and Business Media LLC, 2020
  • Published in: BMC Neurology
  • Language: English
  • DOI: 10.1186/s12883-020-01890-2
  • ISSN: 1471-2377
  • Keywords: Neurology (clinical) ; General Medicine
  • Origination:
  • Footnote:
  • Description: <jats:title>Abstract</jats:title><jats:sec> <jats:title>Background</jats:title> <jats:p>Hereditary hemorrhagic telangiectasia (HHT), also known as Rendu-Osler-Weber syndrome, is a rare disorder characterized by recurrent epistaxis, telangiectasias and systemic arteriovenous malformations (AVMs). HHT is associated with mutations in genes encoding for proteins involved in endothelial homeostasis such as <jats:italic>ENG</jats:italic> (endoglin) and <jats:italic>ACVRL1</jats:italic> (activin receptor-like kinase-1).</jats:p> </jats:sec><jats:sec> <jats:title>Case presentation</jats:title> <jats:p>Here we describe a 22-year-old male presenting with a transient episode of slurred speech and left arm paresis. Brain MRI displayed polymicrogyria. A right-to-left shunt in absence of an atrial septum defect was noted. Chest CT revealed multiple pulmonary AVMs, likely causing paradoxical embolism manifesting as a transient ischemic attack.</jats:p> <jats:p>The heterozygous <jats:italic>ENG</jats:italic> variant, c.3G &gt; A (p.Met1lle), was detected in the patient. This variant was also found in patient’s mother and in his younger brother who displayed cortical dysplasia type 2.</jats:p> </jats:sec><jats:sec> <jats:title>Conclusions</jats:title> <jats:p>The detection of cortical development malformations in multiple subjects from the same pedigree may expand the phenotypic features of ENG-related HHT patients. We suggest considering HHT in young patients presenting with acute cerebral ischemic events of unknown origin.</jats:p> </jats:sec>
  • Access State: Open Access