BNF for Children (BNFC) 2018-2019

according to plasma-ammonia concentration, the total

daily dose may alternatively be given in 3 – 4 divided

doses

IMPORTANT SAFETY INFORMATION

EMERGENCY MANAGEMENT OF UREA CYCLE DISORDERS

For further information on the emergency management

of urea cycle disorders consult the British Inherited

Metabolic Disease Group (BIMDG) website at

http://www.bimdg.org.uk.

lSIDE-EFFECTS

▶Common or very commonHyperhidrosis

▶UncommonBradycardia.diarrhoea.fever.vomiting

▶Frequency not knownRash

lPREGNANCYManufacturer advises avoid unless

essential—no information available.

lBREAST FEEDINGManufacturer advises avoid—present in

milk inanimalstudies.

lDIRECTIONS FOR ADMINISTRATIONDispersible tablets

must be dispersed in at least 5 – 10 mL of water and taken

orally immediately, or administered via a nasogastric tube.

lMEDICINAL FORMS

There can be variation in the licensing of different medicines

containing the same drug.

Dispersible tablet

CAUTIONARY AND ADVISORY LABELS 13

▶Carbaglu(Orphan Europe (UK) Ltd)

Carglumic acid 200 mgCarbaglu 200 mg dispersible tablets sugar-

free| 5 tabletP£ 299. 00 sugar-free| 15 tabletP£ 897. 00

sugar-free| 60 tabletP£ 3 , 499. 00

▶Ucedane(Lucane Pharma Ltd)

Carglumic acid 200 mgUcedane 200 mg dispersible tablets sugar-

free| 60 tabletP£ 3 , 300. 00

Citrulline

lINDICATIONS AND DOSE

Carbamyl phosphate synthase deficiency|Ornithine

carbamyl transferase deficiency

▶BY MOUTH

▶Neonate: 150 mg/kg daily in 3 – 4 divided doses, adjusted

according to response.

▶Child: 150 mg/kg daily in 3 – 4 divided doses, adjusted

according to response

lPREGNANCYNo information available.

lBREAST FEEDINGNo information available.

lDIRECTIONS FOR ADMINISTRATIONPowder may be mixed

with drinks or taken as a paste.

lMEDICINAL FORMS

There can be variation in the licensing of different medicines

containing the same drug. Forms available from special-order

manufacturers include: tablet, oral solution, powder

Powder

▶Citrulline (Non-proprietary)

L-citrulline 1 mg per 1 mgL-Citrulline powder| 100 gram £ 234. 27

BENZOATES

Sodium benzoate

lINDICATIONS AND DOSE

Acute hyperammonaemia due to urea cycle disorder

(specialist use only)

▶BY INTRAVENOUS INFUSION

▶Neonate:Initially 250 mg/kg, to be administered over

90 minutes, followed by 10 mg/kg/hour, adjusted

according to response.

▶Child:Initially 250 mg/kg, to be administered over

90 minutes, followed by 10 mg/kg/hour, adjusted

according to response

Maintenance treatment of hyperammonaemia due to urea

cycle disorders (specialist use only)|Non-ketotic

hyperglycinaemia (specialist use only)

▶BY MOUTH

▶Neonate:Up to 250 mg/kg daily in 3 – 4 divided doses,

dose to be taken with feeds.

▶Child:Up to 250 mg/kg daily in 3 – 4 divided doses, dose

to be taken with feeds or meals; maximum 12 g per day

lUNLICENSED USENot licensed for use in children.

lCAUTIONSConditions involving sodium retention with

oedema (preparations contain significant amounts of

sodium).congestive heart failure (preparations contain

significant amounts of sodium).neonates (risk of

kernicterus and increased side-effects)

lSIDE-EFFECTSAppetite decreased.coma.irritability.

lethargy.nausea.vomiting

SIDE-EFFECTS, FURTHER INFORMATIONGastro-intestinal

side-effects may be reduced by giving smaller doses more

frequently.

lPREGNANCYNo information available.

lBREAST FEEDINGNo information available.

lRENAL IMPAIRMENTCaution in renal insufficiency—

preparations contain significant amounts of sodium.

lDIRECTIONS FOR ADMINISTRATION

▶With oral useFor administrationby mouth, oral solution or

powder may be administered in fruit drinks; less soluble in

acidic drinks.

▶With intravenous useForintravenous infusion, dilute to a

max. concentration of 50 mg/mL with Glucose 10 %.

lPATIENT AND CARER ADVICE

Medicines for Children leaflet: Sodium benzoate for urea cycle

disorderswww.medicinesforchildren.org.uk/sodium-benzoate-

for-urea-cycle-disorders

lMEDICINAL FORMS

Forms available from special-order manufacturers include:

tablet, capsule, oral solution, solution for infusion

DRUGS FOR METABOLIC DISORDERS›ACETIC

ACIDS

Sodium dichloroacetate

lINDICATIONS AND DOSE

Pyruvate dehydrogenase defects

▶BY MOUTH

▶Neonate:Initially 12. 5 mg/kg 4 times a day, adjusted

according to response, increased if necessary up to

200 mg/kg daily.

▶Child:Initially 12. 5 mg/kg 4 times a day, adjusted

according to response, increased if necessary up to

200 mg/kg daily

lSIDE-EFFECTSMetabolic acidosis.oxalate metabolism

abnormal.polyneuropathy (long term use)

lPREGNANCYNo information available.

lBREAST FEEDINGNo information available.

lMEDICINAL FORMS

No licensed medicines listed.

614 Metabolic disorders BNFC 2018 – 2019

Blood and nutrition

9