Exudative retinopathy, cerebral calcifications, duodenal atresia, preaxial polydactyly, micropenis, microcephaly and short stature: a new syndrome?

Am J Med Genet A. 2013 Aug;161A(8):1829-32. doi: 10.1002/ajmg.a.36021. Epub 2013 Jul 3.

Abstract

The association of Coats disease with intrauterine growth retardation, intracranial calcification, leukodystrophy, brain cysts, osteopenia, and gastrointestinal bleeding defines Coats plus syndrome caused by mutations in the CTC1 gene, encoding conserved telomere maintenance component 1. Here, we report on a child with exudative retinopathy, cerebral calcifications, duodenal atresia, preaxial polydactyly, micropenis, microcephaly, and short stature, in whom no mutations in CTC1 were found. Our patient shares some features seen in other diseases associated with telomere shortening including Hoyeraal-Hreidarsson and Revesz syndromes. We therefore measured telomere length by Flow-Fish which was normal. The association of duodenal atresia and microcephaly also suggested a diagnosis of Feingold syndrome. However, direct sequencing of MYCN was normal, and we did not detect any hemizygous deletion of the miR-17∼92 polycistronic miRNA cluster. To our knowledge, the phenotype we report on has not been described previously, leading us to speculate that this condition may represent a new syndrome.

Keywords: cerebral calcifications; duodenal atresia; exudative citroretinopathy; microcephaly; micropenis; prezxial polydactyly; short stature.

Publication types

  • Case Reports

MeSH terms

  • Abnormalities, Multiple
  • Brain Diseases / genetics
  • Brain Diseases / pathology*
  • Calcinosis / genetics
  • Calcinosis / pathology*
  • Child, Preschool
  • Duodenal Obstruction / genetics
  • Duodenal Obstruction / pathology*
  • Dwarfism / genetics
  • Dwarfism / pathology*
  • Exudates and Transudates
  • Genital Diseases, Male / genetics
  • Genital Diseases, Male / pathology*
  • Humans
  • Intestinal Atresia
  • Male
  • Microcephaly / genetics
  • Microcephaly / pathology*
  • Penis / abnormalities
  • Penis / pathology
  • Phenotype
  • Polydactyly / genetics
  • Polydactyly / pathology*
  • Retinal Telangiectasis / genetics
  • Retinal Telangiectasis / pathology*
  • Syndrome
  • Tomography, X-Ray Computed

Supplementary concepts

  • Familial duodenal atresia
  • Penis agenesis