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Key Features

  • X-linked recessive disorder

  • Manifests in boys with spasticity, dystonia, and self-mutilating behaviors

  • Urinary uric acid to creatinine ratio or urine succinylpurines are useful screening tests.

Clinical Findings

  • Complete deficiency is characterized by CNS dysfunction and purine overproduction with hyperuricemia and hyperuricosuria

  • Choreoathetosis

  • Spasticity

  • Compulsive, mutilating lip and finger biting

  • Gouty arthritis

  • Urate ureterolithiasis

Diagnosis

  • Elevated uric acid-creatinine ratio in urine

  • Demonstration of enzyme deficiency in red blood cells or fibroblasts or by molecular analysis

Treatment

  • Hyperhydration and alkalinization are essential to prevent kidney stones and urate nephropathy

  • Allopurinol and probenecid may be given to reduce hyperuricemia and prevent gout but do not affect the neurologic status

  • Physical restraints are often more effective than neurologic medications for automutilation

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