Beyond Gómez-López-Hernández syndrome: recurring phenotypic themes in rhombencephalosynapsis

Am J Med Genet A. 2012 Oct;158A(10):2393-406. doi: 10.1002/ajmg.a.35561. Epub 2012 Sep 10.

Abstract

Rhombencephalosynapsis (RES) is an uncommon cerebellar malformation characterized by fusion of the hemispheres without an intervening vermis. Frequently described in association with Gómez-López-Hernández syndrome, RES also occurs in conjunction with VACTERL features and with holoprosencephaly (HPE). We sought to determine the full phenotypic spectrum of RES in a large cohort of patients. Information was obtained through database review, patient questionnaire, radiographic, and morphologic assessment, and statistical analysis. We assessed 53 patients. Thirty-three had alopecia, 3 had trigeminal anesthesia, 14 had VACTERL features, and 2 had HPE with aventriculy. Specific craniofacial features were seen throughout the cohort, but were more common in patients with alopecia. We noted substantial overlap between groups. We conclude that although some distinct subgroups can be delineated, the overlapping features seen in our cohort suggest an underlying spectrum of RES-associated malformations rather than a collection of discrete syndromes.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Abnormalities, Multiple / pathology*
  • Adolescent
  • Adult
  • Alopecia / pathology*
  • Anal Canal / abnormalities
  • Anal Canal / pathology
  • Cerebellar Diseases / pathology*
  • Cerebellum / abnormalities
  • Cerebellum / pathology
  • Child
  • Child, Preschool
  • Craniofacial Abnormalities / pathology*
  • Esophagus / abnormalities
  • Esophagus / pathology
  • Female
  • Growth Disorders / pathology*
  • Heart Defects, Congenital / pathology
  • Holoprosencephaly / pathology
  • Humans
  • Infant
  • Infant, Newborn
  • Kidney / abnormalities
  • Kidney / pathology
  • Limb Deformities, Congenital / pathology
  • Male
  • Neurocutaneous Syndromes / pathology*
  • Phenotype
  • Rhombencephalon / abnormalities*
  • Rhombencephalon / pathology*
  • Spine / abnormalities
  • Spine / pathology
  • Trachea / abnormalities
  • Trachea / pathology
  • Young Adult

Supplementary concepts

  • Gomez Lopez Hernandez syndrome
  • VACTERL association