Drugs List

Therapeutic Indications

Uses

Mucopolysaccharidosis IVA (Morquio A Syndrome)

Administration

For intravenous infusion only.

Patients weighing less than 25kg should receive a total volume of 100ml. When diluted in 100ml, the initial infusion rate should be 3ml/hour. The infusion rate may be increased as tolerated, every 15 minutes as follows: first increase the rate to 6ml/hour, then increase the rate every 15 minutes by 6ml/hour increments until a maximum rate of 36ml/hour is reached.

Patients weighing 25kg or more should receive a total volume of 250ml. When diluted in 250ml, the initial infusion rate should be 6ml/hour. The infusion rate may be increased as tolerated, every 15 minutes as follows: first increase the rate to 12ml/hour, then increase the rate every 15 minutes by 12ml/hour increments until a maximum rate of 72ml/hour is reached.

Contraindications

Hereditary fructose intolerance

Precautions and Warnings

Patients over 65 years
Restricted sodium intake
Breastfeeding
Pregnancy

Sodium content of formulation may be significant
Advise patient dizziness may affect ability to drive or operate machinery
Pre-medicate with antihistamines with or without antipyretics
Treatment to be initiated and supervised by a specialist
Presentations with sorbitol unsuitable in hereditary fructose intolerance
Dilute and use as an infusion
Resuscitation facilities must be immediately available
If spinal cord compression present/develops,use specific standard treatment
Monitor patient for infusion-associated reactions (IARs)
Interrupt therapy/reduce infusion rate if infusion-related reactions occur

Patients should receive pre-treatment with an antihistamine with or without antipyretics approximately 30 to 60 minutes prior to infusion.

Management of infusion reactions (IR) should be based on the severity of the reaction and include slowing or temporary interruption of the infusion and/or administration of additional antihistamines, antipyretics and/or corticosteroids. If severe IRs occur, immediately stop the infusion and initiate appropriate treatment. Re-administration after a severe reaction should be carried out with caution and close monitoring by the treating physician.

Pregnancy and Lactation

Pregnancy

Use elosulfase alfa with caution during pregnancy.

The manufacturer advises to avoid the use of elosulfase alfa during pregnancy unless clearly necessary. Animal studies do not indicate any direct or indirect harmful effects on pregnancy or foetal development. At the time of writing, there is no data available regarding the use of elosulfase alfa during human pregnancy. Potential risks are unknown.

Lactation

Use elosulfase alfa with caution during breastfeeding.

The manufacturer advises elosulfase alfa should only be administered to breastfeeding women if the potential benefit is considered to outweigh the potential risk. Animal studies have shown excretion of elosulfase alfa in milk, however presence in human breast milk is unknown. Systemic exposure via human breast milk is not expected but the effects in exposed infants are unknown.

Side Effects

Abdominal pain
Anaphylaxis
Antibody formation
Chills
Diarrhoea
Dizziness
Dyspnoea
Headache
Hypersensitivity reactions
Myalgia
Nausea
Oropharyngeal pain
Pyrexia
Upper abdominal pain
Vomiting

Presentation

Infusions of elosulfase alfa.

These products have been produced by recombinant technology using Chinese Hamster Ovary (CHO) cell lines.

Drugs List

Therapeutic Indications

Uses

Mucopolysaccharidosis IVA (Morquio A Syndrome)

Dosage

Adults

Children

Administration

For intravenous infusion only.

Patients weighing less than 25kg should receive a total volume of 100ml. When diluted in 100ml, the initial infusion rate should be 3ml/hour. The infusion rate may be increased as tolerated, every 15 minutes as follows: first increase the rate to 6ml/hour, then increase the rate every 15 minutes by 6ml/hour increments until a maximum rate of 36ml/hour is reached.

Patients weighing 25kg or more should receive a total volume of 250ml. When diluted in 250ml, the initial infusion rate should be 6ml/hour. The infusion rate may be increased as tolerated, every 15 minutes as follows: first increase the rate to 12ml/hour, then increase the rate every 15 minutes by 12ml/hour increments until a maximum rate of 72ml/hour is reached.

Contraindications

Hereditary fructose intolerance

Precautions and Warnings

Patients over 65 years
Restricted sodium intake
Breastfeeding
Pregnancy

Sodium content of formulation may be significant
Advise patient dizziness may affect ability to drive or operate machinery
Pre-medicate with antihistamines with or without antipyretics
Treatment to be initiated and supervised by a specialist
Presentations with sorbitol unsuitable in hereditary fructose intolerance
Dilute and use as an infusion
Resuscitation facilities must be immediately available
If spinal cord compression present/develops,use specific standard treatment
Monitor patient for infusion-associated reactions (IARs)
Interrupt therapy/reduce infusion rate if infusion-related reactions occur

Patients should receive pre-treatment with an antihistamine with or without antipyretics approximately 30 to 60 minutes prior to infusion.

Management of infusion reactions (IR) should be based on the severity of the reaction and include slowing or temporary interruption of the infusion and/or administration of additional antihistamines, antipyretics and/or corticosteroids. If severe IRs occur, immediately stop the infusion and initiate appropriate treatment. Re-administration after a severe reaction should be carried out with caution and close monitoring by the treating physician.

Pregnancy and Lactation

Pregnancy

Use elosulfase alfa with caution during pregnancy.

The manufacturer advises to avoid the use of elosulfase alfa during pregnancy unless clearly necessary. Animal studies do not indicate any direct or indirect harmful effects on pregnancy or foetal development. At the time of writing, there is no data available regarding the use of elosulfase alfa during human pregnancy. Potential risks are unknown.

Lactation

Use elosulfase alfa with caution during breastfeeding.

The manufacturer advises elosulfase alfa should only be administered to breastfeeding women if the potential benefit is considered to outweigh the potential risk. Animal studies have shown excretion of elosulfase alfa in milk, however presence in human breast milk is unknown. Systemic exposure via human breast milk is not expected but the effects in exposed infants are unknown.

Side Effects

Abdominal pain
Anaphylaxis
Antibody formation
Chills
Diarrhoea
Dizziness
Dyspnoea
Headache
Hypersensitivity reactions
Myalgia
Nausea
Oropharyngeal pain
Pyrexia
Upper abdominal pain
Vomiting

Overdosage

It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

The following number will direct the caller to the relevant local centre (0844) 892 0111

Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).

Further Information

Last Full Review Date: December 2019

Reference Sources

Summary of Product Characteristics: Vimizim 1mg/ml concentrate for solution for infusion. Biomarin Europe Ltd. Revised December 2018.