AP1B1

Clinical Characteristics

The clinical features of KIDAR include:

Skin abnormalities

  • Ichthyosis
  • Erythroderma
  • Palmoplantar keratoderma
  • Sparse hair, with or without a wiry texture
  • Thick yellow plate-like scale on scalp
  • Sparse or absent eyebrows
  • Excessive creases over palms and soles (in some patients)
  • Anhidrosis (in 1 patient)

Hepatic abnormalities

  • Hepatopathy (in some patients)

Gastrointestinal abnormalities

  • Enteropathy (in some patients)

Neurological abnormalities

  • Global developmental delay
  • Intellectual disability
  • Hyperreflexia (in some patients)
  • Sensorineural hearing loss – this can be profound requiring cochlear implantation
  • Photophobia (in some patients)
  • Structural brain abnormalities (in some patients):
    • Cerebral atrophy
    • Thin corpus callosum

Facial dysmorphism (variable, mild; in some patients)

  • Frontal bossing
  • Hairline recession
  • Ectropion

The following features were observed in a case report of an adult patient with KIDAR:

  • Severe corneal scarring
  • Gingival recession
  • Fissured tongue
  • Tooth loss and poor calcification of teeth

Laboratory abnormalities

  • Low serum copper
  • Low ceruloplasmin levels
  • Low plasma zinc
  • Elevated very-long-chain fatty acids (in some patients)
  • Liver profile (abnormal in some patients):
    • High alanine aminotransferase
    • High gamma-glutamyl transferase
    • High alkaline phosphatase
    • High bile acids
    • Low plasma albumin
  • CBC (abnormal in some patients):
    • Thrombocytopenia (episodic or persistent)
    • Anemia

Skin histology

  • Compact hyperkeratosis
  • Keratinocyte separation (focal) above the basal layer
  • Increased basophilic vesicles in the proliferative layers of epidermis
  • Large intracytoplasmic vesicles (on electron microscopy)

Duodenal biopsy and histology

  • Mild blunting of the villi
  • Mild crypt hyperplasia
  • Cytoplasmic vacuolation of the surface epithelium

Other

  • Failure to thrive
  • Dysmegakaryopoiesis on bone marrow biopsy