Novel DSP Spectrin 6 Region Variant Causes Neonatal Erythroderma, Failure to Thrive, Severe Herpes Simplex Infections and Brain Lesions

Acta Derm Venereol. 2019 Jul 1;99(9):789-796. doi: 10.2340/00015555-3203.

Abstract

Desmoplakin (DSP) and Desmoglein 1 (DSG1) variants result in skin barrier defects leading to erythroderma, palmoplantar keratoderma and variable [AQ4] other features. Some DSG1 variant carriers present with SAM syndrome (Severe dermatitis, multiple Allergies, Metabolic wasting) and a SAM-like phenotype has been reported in 4 subjects with different heterozygous DSP variants. We report here a patient with a novel DSP spectrin region (SR) 6 variant c.1756C>T, p.(His586Tyr), novel features of brain lesions and severe recurrent mucocutaneous herpes simplex virus infections, with a favourable response to ustekinumab. Through a review of reported cases of heterozygous variants in DSP SR6 (n = 15) and homozygous or compound heterozygous variants in DSG1 (n = 12) and SAM-like phenotype, we highlight phenotypic variability. Woolly hair, nail abnormalities and cardiomyopathy characterize patients with DSP variants, while elevated immunoglobulin E and food allergies are frequent in patients with DSG1 variants. Clinicians should be aware of the diverse manifestations of desmosomopathies.

Keywords: SAM syndrome; desmoplakin; metabolic wasting; severe dermatitis; desmoglein.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Brain Diseases / diagnostic imaging
  • Brain Diseases / genetics*
  • Child, Preschool
  • Dermatitis, Exfoliative / diagnosis
  • Dermatitis, Exfoliative / drug therapy
  • Dermatitis, Exfoliative / genetics*
  • Dermatologic Agents / therapeutic use
  • Desmoplakins / genetics*
  • Failure to Thrive / diagnosis
  • Failure to Thrive / genetics*
  • Genetic Predisposition to Disease
  • Genetic Variation*
  • Herpes Simplex / diagnosis
  • Herpes Simplex / genetics*
  • Herpes Simplex / virology
  • Humans
  • Ichthyosis / diagnosis
  • Ichthyosis / drug therapy
  • Ichthyosis / genetics*
  • Infant
  • Infant, Newborn
  • Male
  • Phenotype
  • Severity of Illness Index
  • Treatment Outcome
  • Ustekinumab / therapeutic use

Substances

  • DSP protein, human
  • Dermatologic Agents
  • Desmoplakins
  • Ustekinumab