BNF for Children 2011-2012

494 9.8.1 Drugs used in metabolic disorders BNFC 2011–20129 Nutrition and bloodMaintenance treatment of hyperammonaemiain carbamylphosphate synthetase deficiency,ornithine carbamyl transferase deficiency (specialistuse only). By mouthNeonate 100 mg/kg daily in 3–4 divided dosesChild 1 month–18 years 100 mg/kg daily in 3–4divided dosesAcute hyperammonaemia in citrullinaemia,arginosuccinic aciduria (specialist use only). By intravenous infusionNeonate initially 600 mg/kg over 90 minutes followedby 25 mg/kg/hourChild 1 month–18 years initially 600 mg/kg over90 minutes followed by 25 mg/kg/hourMaintenance treatment of hyperammonaemiain citrullinaemia, arginosuccinic aciduria (specialistuse only). By mouthNeonate 100–175 mg/kg 3–4 times daily, withfood, adjusted according to responseChild 1 month–18 years 100–175 mg/kg 3–4times daily, with food, adjusted according toresponseL-Arginine (Non-proprietary)Tablets, L-arginine (as hydrochloride) 500 mg,Oral solution, L-arginine 100 mg/mLAvailable from ‘special-order’ manufacturers or specialistimporting companies, see p. 809Powder, L-arginine (as hydrochloride), net price 100 g= £12.27Prescribe as a borderline substance (ACBS). For use as a supplementin urea cycle disorders other than arginase deficiency, suchas hyperammonaemia types I and II, citrullinaemia, arginosuccinicaciduria, and deficiency of N-acetyl glutamate synthetaseInjection, L-arginine (as hydrochloride) 500 mg/mL,10-mL ampoules; 100 mg/mL, 200-mL ampAvailable from ‘special-order’ manufacturers or specialistimporting companies, see p. 809Note Other strengths may be available from ‘special-order’manufacturers or specialist importing companies, see p. 809Administration dilute to a concentration of 20 mg/mL withSodium Chloride 0.9% or 0.45%, or Glucose 5% or 10%; max.concentration 100 mg/mL; may be given orallyCARGLUMIC ACIDPregnancy manufacturer advises avoid unless essential—noinformation availableBreast-feeding manufacturer advises avoid—presentin milk in animal studiesSide-effects sweatingIndication and doseHyperammonaemia due to N-acetyl glutamatesynthase deficiency (initiated under specialistsupervision). By mouthNeonate initially 50–125 mg/kg twice dailyimmediately before feeds, adjusted according toplasma-ammonia concentration; maintenance 5–50 mg/kg twice daily; total daily dose may alternativelybe given in 3–4 divided dosesChild 1 month–18 years initially 50–125 mg/kgtwice daily immediately before food, adjustedaccording to plasma-ammonia concentration;maintenance 5–50 mg/kg twice daily; total dailydose may alternatively be given in 3–4 divideddosesCarbaglu c (Orphan Europe) ADispersible tablets, carglumic acid 200 mg, net price5-tab pack = £299.00, 60-tab pack = £3499.00. Label:13CITRULLINEPregnancy no information availableBreast-feeding no information availableIndication and doseCarbamyl phosphate synthase deficiency,ornithine carbamyl transferase deficiency. By mouthNeonate 150 mg/kg daily in 3–4 divided doses,adjusted according to responseChild 1 month–18 years 150 mg/kg daily in 3–4divided doses, adjusted according to responseCitrulline Powder (Non-proprietary)Powder, L-citrulline 100 gAvailable from ‘special-order’ manufacturers or specialistimporting companies, see p. 809Administration May be mixed with drinks or taken as a pasteSODIUM BENZOATECautions see notes above; neonates (risk of kernicterusand increased side-effects); interactions:Appendix 1 (sodium benzoate)Renal impairment see notes abovePregnancy no information availableBreast-feeding no information availableSide-effects nausea, vomiting, anorexia; irritability,lethargy, comaLicensed use not licensed for use in childrenIndication and doseAcute hyperammonaemia due to urea cycledisorders (specialist use only). By intravenous infusionNeonate initially 250 mg/kg over 90 minutes followedby 20 mg/kg/hour, adjusted according toresponseChild 1 month–18 years initially 250 mg/kg over90 minutes followed by 20 mg/kg/hour, adjustedaccording to responseMaintenance treatment of hyperammonaemiadue to urea cycle disorders; non-ketotichyperglycinaemia (specialist use only). By mouthNeonate 50–150 mg/kg 3–4 times daily, with food,adjusted according to responseChild 1 month–18 years 50–150 mg/kg 3–4times daily, with food, adjusted according toresponse