Sodium Phenylbutyrate

What is Sodium Phenylbutyrate

Sodium phenylbutyrate is an adjunctive therapy in the chronic management of urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

It must be combined with dietary protein restriction and, in some cases, essential amino acid supplementation.

Sodium phenylbutyrate is indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life), as well as in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy. 

Brand Name

Buphenyl

Indications & Dosage

  • argininosuccinic acid syntherase (AAS) deficiency
  • carbamoyl phosphate synthetase (CPS) deficiency
  • ornithine transcarbamoylase (OTC) deficiency

For use as an adjunctive therapy to dietary protein restriction and amino acid supplementation in the chronic management of patients with neonatal-onset, or late-onset with history of hyperammonemic encephalopathy, urea cycle disorders ornithine transcarbamoylase (OTC) deficiency, carbamoyl phosphate synthetase (CPS) deficiency, or argininosuccinic acid syntherase (AAS) deficiency

Side Effects

  1. abdominal pain
  2. amenorrhea
  3. anemia
  4. aplastic anemia
  5. arrhythmia exacerbation
  6. bleeding
  7. constipation
  8. depression
  9. drug-induced body odor
  10. dysgeusia
  11. ecchymosis
  12. edema
  13. elevated hepatic enzymes
  14. gastritis
  15. headache
  16. hyperbilirubinemia
  17. hyperchloremia
  18. hypernatremia
  19. hyperphosphatemia
  20. hyperuricemia
  21. hypoalbuminemia
  22. hypokalemia
  23. hypophosphatemia
  24. leukocytosis
  25. leukopenia
  26. menstrual irregularity
  27. metabolic acidosis
  28. metabolic alkalosis
  29. nausea
  30. pancreatitis
  31. peptic ulcer
  32. rash
  33. renal tubular acidosis (RTA)
  34. syncope
  35. thrombocytopenia
  36. thrombocytosis
  37. vomiting

Contraindications

  • breast-feeding
  • children
  • corticosteroid therapy
  • edema
  • heart failure
  • hepatic disease
  • infants
  • neonates
  • pregnancy
  • renal disease
  • renal failure
  • renal impairment
  • sodium restriction
  • Azelastine; Fluticasone
  • Beclomethasone
  • Betamethasone
  • Budesonide
  • Budesonide; Formoterol
  • Ciclesonide
  • Colchicine; Probenecid
  • Corticosteroids
  • Corticotropin, ACTH
  • Cortisone
  • Deflazacort
  • Dexamethasone
  • Dichlorphenamide
  • Fludrocortisone
  • Flunisolide
  • Fluticasone
  • Fluticasone; Salmeterol
  • Fluticasone; Umeclidinium; Vilanterol
  • Fluticasone; Vilanterol
  • Formoterol; Mometasone
  • Haloperidol
  • Hydrocortisone
  • Methylprednisolone
  • Mometasone
  • Prednisolone
  • Prednisone
  • Probenecid
  • Sodium Benzoate; Sodium Phenylacetate
  • Triamcinolone
  • Valproic Acid, Divalproex Sodium