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Wolfram syndrome (DIDMOAD)

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A teenager with type 1 diabetes is found to have progressive vision loss (optic atrophy). Audiometry reveals sensorineural hearing loss. Urological workup shows diabetes insipidus.

AR; WFS1 (wolframin)

DIDMOAD mnemonic:

  • Diabetes Insipidus
  • Diabetes Mellitus (non-autoimmune)
  • Optic Atrophy
  • Deafness (sensorineural)

Other features: neurological degeneration, urinary tract abnormalities

  • Suspect with the combination of non-autoimmune (antibody-negative) diabetes mellitus and optic atrophy, the minimal diagnostic dyad
  • Document associated features: central diabetes insipidus (water deprivation testing), sensorineural hearing loss (audiometry), and urinary tract dilation
  • Confirm with WFS1 sequencing (autosomal recessive); CISD2 in Wolfram syndrome type 2
  • No disease-modifying therapy; care is supportive and multidisciplinary (endocrinology, ophthalmology, audiology, urology, neurology)
  • Insulin for diabetes mellitus; desmopressin for central diabetes insipidus; hearing aids for sensorineural hearing loss
  • Surveillance for progressive neurodegeneration, neurogenic bladder, and psychiatric manifestations
  • Genetic counseling: autosomal recessive, 25% recurrence risk for siblings

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