DCAF17

Clinical Characteristics

The clinical features of this disease, per organ system, include:

Central nervous system

  • Intellectual disability
  • Dystonia
  • Choreoathetosis
  • Dysarthria
  • Extrapyramidal symptoms
  • Sensorineural deafness
  • White matter lesions
  • Decreased signal intensities in basal ganglia
  • Other rarer manifestations:
    • Psychosis
    • Hallucinations

Head and neck

  • Dysmorphic facial features (some patients), including:
    • Triangular face
    • Prominent ears
    • Prominent nasal root
    • Anodontia

Skin and hair

  • Alopecia
  • Loss of eyebrow hair

Endocrine

  • Diabetes mellitus
  • Hypogonadotropic or hypergonadotropic hypogonadism
  • Failure of secondary sexual development

Genitourinary

  • Small penis
  • Small testes
  • Hypospermatogenesis
  • Atrophic seminiferous tubules
  • Primary ovarian failure
  • Hypoplastic uterus
  • Streak ovaries
  • Rudimentary fallopian tubes
  • Vulvar hypoplasia

Laboratory features

  • Decreased testosterone
  • Decreased insulin-like growth factor 1
  • Decreased estradiol
  • Decreased thyroxine
  • Increased thyroid-stimulating hormone
  • Increased follicle-stimulating hormone
  • Hyperlipidemia

Other(s)

  • ECG Changes (nonspecific T-wave abnormalities)

The disease has a variable phenotype and a variable time of onset, with mental impairment commonly starting in early childhood and other symptoms starting in adolescence or early adulthood.