Your browser doesn't support javascript.
loading
Neuroimaging in Kabuki syndrome and another KMT2D-related disorder.
Stadelmaier, Rachel T; Kenna, Margaret A; Barrett, Devon; Mullen, Thomas E; Bodamer, Olaf; Agrawal, Pankaj B; Robson, Caroline D; Wojcik, Monica H.
Afiliação
  • Stadelmaier RT; Division of Newborn Medicine, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Kenna MA; Department of Otolaryngology and Communication Enhancement, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Barrett D; Emory University School of Medicine, Atlanta, Georgia, USA.
  • Mullen TE; Center for Mendelian Genomics, Broad Institute of MIT and Harvard, Cambridge, Massachusetts, USA.
  • Bodamer O; Division of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Agrawal PB; Division of Newborn Medicine, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Robson CD; Division of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Wojcik MH; Department of Radiology, Boston Children's Hospital, Boston, Massachusetts, USA.
Am J Med Genet A ; 185(12): 3770-3783, 2021 12.
Article em En | MEDLINE | ID: mdl-34369642
Recognition of distinct phenotypic features is an important component of genetic diagnosis. Although CHARGE syndrome, Kabuki syndrome, and a recently delineated KMT2D Ex 38/39 allelic disorder exhibit significant overlap, differences on neuroimaging may help distinguish these conditions and guide genetic testing and variant interpretation. We present an infant clinically diagnosed with CHARGE syndrome but subsequently found to have a de novo missense variant in exon 38 of KMT2D, the gene implicated in both Kabuki syndrome and a distinct KMT2D allelic disorder. We compare her brain and inner ear morphology to a retrospective cohort of 21 patients with classic Kabuki syndrome and to typical CHARGE syndrome findings described in the literature. Thirteen of the 21 Kabuki syndrome patients had temporal bone imaging (5/13 CT, 12/13 MRI) and/or brain MRI (12/13) which revealed findings distinct from both CHARGE syndrome and the KMT2D allelic disorder. Our findings further elucidate the spectrum of inner ear dysmorphology distinguishing Kabuki syndrome and the KMT2D allelic disorder from CHARGE syndrome, suggesting that these three disorders may be differentiated at least in part by their inner ear anomalies.
Assuntos
Palavras-chave

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Anormalidades Múltiplas / Doenças Vestibulares / Predisposição Genética para Doença / Proteínas de Ligação a DNA / Face / Síndrome CHARGE / Doenças Hematológicas / Proteínas de Neoplasias Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Female / Humans / Infant / Male / Newborn Idioma: En Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Anormalidades Múltiplas / Doenças Vestibulares / Predisposição Genética para Doença / Proteínas de Ligação a DNA / Face / Síndrome CHARGE / Doenças Hematológicas / Proteínas de Neoplasias Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Female / Humans / Infant / Male / Newborn Idioma: En Ano de publicação: 2021 Tipo de documento: Article