Respiratory and gastrointestinal dysfunctions associated with auriculo-condylar syndrome and a homozygous PLCB4 loss-of-function mutation

Am J Med Genet A. 2016 Jun;170(6):1471-8. doi: 10.1002/ajmg.a.37625. Epub 2016 Mar 23.

Abstract

Auriculo-Condylar Syndrome (ACS) is a craniofacial malformation syndrome characterized by external ear anomalies, hypoplasia of the mandibular condyle, temporomandibular joint abnormalities, micrognathia, and microstomia. Glossoptosis, masticatory abnormalities, orthodontic problems, and malocclusion occur in a majority of affected subjects. The clinical diagnosis is usually suggested by the pathognomonic ear appearance ("question mark ear"), consisting of a variable degree of clefting between the helix and earlobe. The genetic mechanisms underlying ACS have recently been identified. Both autosomal dominant and recessive inheritance of mutations in phospholipase C, beta 4 (PLCB4) and endothelin 1 (EDN1) have been reported along with autosomal dominant mutations in guanine nucleotide-binding protein (G protein) α inhibiting activity polypeptide 3 (GNAI3). We report 6 years of follow-up of a child with a clinical phenotype consistent with ACS due to a homozygous frameshift mutation in PLCB4. The baby presented feeding difficulties associated with failure to thrive and a complex sleep-related respiratory disorder, characterized by central and obstructive apnoeas. Our observations of this case further delineate the phenotype of ACS associated with autosomal recessive PLCB4 loss-of-function mutations, underscoring gastrointestinal dysfunction and severe sleep-related breathing abnormalities as additional features when compared to patients with heterozygous mutations with a presumed dominant negative effect. © 2016 Wiley Periodicals, Inc.

Keywords: PLCB4; auriculo-condylar syndrome; condylar hypoplasia; gastrointestinal dysfunctions; polysomnography; question mark ear; sleep apnoeas.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Comparative Genomic Hybridization
  • DNA Mutational Analysis
  • Ear / abnormalities*
  • Ear / physiopathology
  • Ear Diseases / diagnosis
  • Ear Diseases / genetics*
  • Ear Diseases / physiopathology*
  • Facies
  • Female
  • Genetic Association Studies*
  • Genotype
  • Homozygote*
  • Humans
  • Karyotype
  • Magnetic Resonance Imaging
  • Mutation*
  • Pedigree
  • Phenotype*
  • Phospholipase C beta / genetics*
  • Sequence Analysis, DNA

Substances

  • PLCB4 protein, human
  • Phospholipase C beta

Supplementary concepts

  • Auriculo-condylar syndrome