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Pediatrics9 papers

Hyperinsulinism and hyperammonemia syndrome

Last edited: 4/15/2026

Overview

Hyperinsulinism and hyperammonemia syndrome is characterized by persistent hyperammonemia and hypoketotic hypoglycemia, often linked to functional hyperactivity of hepatic glutamate dehydrogenase (GDH) 1.

Diagnosis

  • Elevated blood ammonia levels (100-300 micromol/L) independent of protein intake 1.
  • Hypoketotic hypoglycemia, particularly postprandial 1.
  • Increased hepatic GDH activity (above normal range: 874 nmol/(min.mg protein)) with reduced GTP inhibition threshold 1.
  • Low plasma glutamine/blood ammonia ratio 1.
  • Management

  • First-line treatment: Diazoxide for managing hyperinsulinism 1.
  • Adjunctive therapy: Oral N-carbamylglutamate to decrease blood ammonia levels 1.
  • Aggressive supportive measures: Dialysis or exchange transfusion in severe cases, particularly in neonates 2.
  • Special Populations

  • Premature infants: Transient neonatal hyperammonemia (TNH) can occur and requires early aggressive treatment for favorable outcomes 2.
  • Key Recommendations

  • Evaluate hepatic GDH activity and GTP inhibition sensitivity in patients with persistent hyperammonemia and hyperinsulinism for diagnosis 1 (Evidence: Moderate).
  • Initiate diazoxide therapy for managing hyperinsulinism in patients with hyperinsulinism/hyperammonemia syndrome 1 (Evidence: Moderate).
  • In neonates with suspected transient neonatal hyperammonemia, promptly institute dialysis or exchange transfusion to prevent neurological damage 2 (Evidence: Weak).
  • References

    1 Huijmans JG, Duran M, de Klerk JB, Rovers MJ, Scholte HR. Functional hyperactivity of hepatic glutamate dehydrogenase as a cause of the hyperinsulinism/hyperammonemia syndrome: effect of treatment. Pediatrics 2000. link 2 Rothberg AD, Thomson PD, Andronikou S, Cohen DF. Transient neonatal hyperammonaemia. A case report. South African medical journal = Suid-Afrikaanse tydskrif vir geneeskunde 1982. link

    Original source

    1. [1]
    2. [2]
      Transient neonatal hyperammonaemia. A case report.Rothberg AD, Thomson PD, Andronikou S, Cohen DF South African medical journal = Suid-Afrikaanse tydskrif vir geneeskunde (1982)

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