lCAUTIONSAcute febrile respiratory illness (consider
delaying treatment).infusion-related reactions can occur.
severe respiratory disease
lSIDE-EFFECTS
▶Common or very commonArrhythmias.arthralgia.chest
pain.cough.cyanosis.diarrhoea.dizziness.dyspnoea.
fever.flushing.gastrointestinal discomfort.headache.
hypertension.hypotension.hypoxia.nausea.oedema.
respiratory disorders.skin reactions.tongue swelling.
tremor.vomiting
▶Frequency not knownHypersensitivity
SIDE-EFFECTS, FURTHER INFORMATIONInfusion-related
reactions often occur, they can be managed by slowing the
infusion rate or interrupting the infusion, and can be
minimised by pre-treatment with an antihistamine and an
antipyretic. Recurrent infusion-related reactions may
require pre-treatment with a corticosteroid—consult
product literature for details.lCONCEPTION AND CONTRACEPTIONContra-indicated in
women of child-bearing potential.
lPREGNANCYManufacturer advises avoid.
lBREAST FEEDINGManufacturer advises avoid—present in
milk inanimalstudies.
lDIRECTIONS FOR ADMINISTRATIONForintravenous
infusion, dilute requisite dose in 100 mL Sodium Chloride
0. 9 % and mix gently (do not shake); give over 3 hours
(gradually reduced to 1 hour if no infusion-related
reactions).
lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Solution for infusion
▶Elaprase(Shire Pharmaceuticals Ltd)A
Idursulfase 2 mg per 1 mlElaprase 6 mg/ 3 ml concentrate for
solution for infusion vials| 1 vialP£ 1 , 985. 00
Laronidase
lDRUG ACTIONLaronidase is an enzyme produced by
recombinant DNA technology licensed for long-term
replacement therapy in the treatment of non-neurological
manifestations of mucopolysaccharidosis I, a lysosomal
storage disorder caused by deficiency of alpha-L-
iduronidase.
lINDICATIONS AND DOSE
Non-neurological manifestations of
mucopolysaccharidosis I (specialist use only)
▶BY INTRAVENOUS INFUSION
▶Child: 100 units/kg once weeklylCAUTIONSInfusion-related reactions can occur
lINTERACTIONS→Appendix 1 : laronidase
lSIDE-EFFECTS
▶Common or very commonAbdominal pain.alopecia.
anaphylactic reaction.angioedema.chills.cough.
diarrhoea.dizziness.dyspnoea.fatigue.fever.flushing.
headache.hypotension.influenza like illness.joint
disorders.nausea.pain.pallor.paraesthesia.peripheral
coldness.respiratory disorders.restlessness.skin
reactions.sweat changes.tachycardia.temperature
sensation altered.vomiting
▶Frequency not knownCyanosis.hypoxia.oedema
SIDE-EFFECTS, FURTHER INFORMATIONInfusion-related
reactions often occur, they can be managed by slowing the
infusion rate or interrupting the infusion, and can be
minimised by pre-treatment with an antihistamine and an
antipyretic. Recurrent infusion-related reactions may
require pre-treatment with a corticosteroid—consult
product literature for details.lPREGNANCYManufacturer advises avoid unless
essential—no information available.
lBREAST FEEDINGManufacturer advises avoid—no
information available.
lMONITORING REQUIREMENTSMonitor immunoglobulin G
(IgG) antibody concentration.
lDIRECTIONS FOR ADMINISTRATIONForintravenous
infusion, dilute with Sodium Chloride 0. 9 %; body-weight
under 20 kg, dilute to 100 mL, body-weight over 20 kg
dilute to 250 mL; give through in-linefilter ( 0. 22 micron)
initially at a rate of 2 units/kg/hour then increase gradually
every 15 minutes to max. 43 units/kg/hour.lMEDICINAL FORMS
There can be variation in the licensing of different medicines
containing the same drug.
Solution for infusion
ELECTROLYTES:May contain Sodium
▶Aldurazyme(Genzyme Therapeutics Ltd)
Laronidase 100 unit per 1 mlAldurazyme 500 units/ 5 ml solution for
infusion vials| 1 vialP£ 444. 703.10Niemann-Pick type C disease
ENZYME INHIBITORS›GLUCOSYLCERAMIDE
SYNTHASE INHIBITORSMiglustat
lDRUG ACTIONMiglustat is an inhibitor of
glucosylceramide synthase.lINDICATIONS AND DOSE
Treatment of progressive neurological manifestations of
Niemann-Pick type C disease (under expert supervision)
▶BY MOUTH
▶Child 4–11 years (body surface area up to 0.48 m^2 ): 100 mg
once daily
▶Child 4–11 years (body surface area 0.48–0.73 m^2 ): 100 mg
twice daily
▶Child 4–11 years (body surface area 0.74–0.88 m^2 ): 100 mg
3 times a day
▶Child 4–11 years (body surface area 0.89–1.25 m^2 ): 200 mg
twice daily
▶Child 4–11 years (body surface area 1.26 m^2 and
above): 200 mg 3 times a day
▶Child 12–17 years: 200 mg 3 times a daylSIDE-EFFECTS
▶Common or very commonAppetite decreased.asthenia.
chills.constipation.depression.diarrhoea.dizziness.
flatulence.gastrointestinal discomfort.headache.
insomnia.libido decreased.malaise.muscle spasms.
muscle weakness.nausea.peripheral neuropathy.
sensation abnormal.thrombocytopenia.tremor.vomiting
.weight decreased
▶Frequency not knownGrowth retardation
lCONCEPTION AND CONTRACEPTIONEffective
contraception must be used during treatment. Men should
avoid fathering a child during and for 3 months after
treatment.
lPREGNANCYManufacturer advises avoid—toxicity in
animalstudies.
lBREAST FEEDINGManufacturer advises avoid—no
information available.
lHEPATIC IMPAIRMENTNo information available—
manufacturer advises caution.
lRENAL IMPAIRMENTAvoid if estimated glomerular
filtration less than 30 mL/minute/ 1. 73 m^2. Child under
12 years—consult product literature.BNFC 2018 – 2019 Niemann-Pick type C disease 611
Blood and nutrition9