Clinical and genetic complexity of Mitchell-Riley/Martinez-Frias syndrome

J Perinatol. 2014 Dec;34(12):948-50. doi: 10.1038/jp.2014.162.

Abstract

Mitchell-Riley syndrome/Martinez-Frias syndrome (MRS/MFS) is a rare, autosomal recessive disorder with multisystem involvement and poor prognosis. Most reported cases have been associated with homozygous or compound heterozygous mutations in the RFX6 gene, a transcriptional regulatory factor for pancreatic morphogenesis. Given the limited number of reported cases, the syndrome may be under-recognized. When the particular phenotype of MFS includes a mutation on the RFX6 gene and neonatal diabetes, it has been called Mitchell-Riley syndrome. Because of this, we propose that MFS/MRS is a symptom continuum or an RFX6 malformation complex. We report an infant with all of the key clinical features of MRS/MFS without a definable mutation in RFX6 gene, supporting the consideration of these features as a symptom complex, and raising the question of genetic heterogeneity.

Publication types

  • Case Reports

MeSH terms

  • DNA-Binding Proteins / genetics*
  • Diabetes Mellitus / diagnosis*
  • Diabetes Mellitus / genetics*
  • Gallbladder Diseases / diagnosis*
  • Gallbladder Diseases / genetics*
  • Hemochromatosis / diagnosis
  • Hemosiderosis / diagnosis
  • Humans
  • Infant, Newborn
  • Intestinal Atresia / diagnosis*
  • Intestinal Atresia / genetics*
  • Magnetic Resonance Imaging
  • Regulatory Factor X Transcription Factors
  • Tracheoesophageal Fistula / diagnosis*
  • Tracheoesophageal Fistula / genetics*
  • Transcription Factors / genetics*

Substances

  • DNA-Binding Proteins
  • Regulatory Factor X Transcription Factors
  • Rfx6 protein, human
  • Transcription Factors

Supplementary concepts

  • Martinez-Frias Syndrome
  • Mitchell-Riley Syndrome
  • Neonatal hemochromatosis