Elaprase

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Elaprase - Frequently
Asked Questions

This website is intended for healthcare professionals outside of the US and UK

What is the dosing schedule for Elaprase?

Elaprase is administered at a dose of 0.5 mg/kg body weight every week, by intravenous infusion, over a 3 hour period. This period of infusion may be gradually reduced to 1 hour if no infusion-associated reactions are observed. Given that dosing depends on body weight of the patient, it is therefore important to weigh the patient before each treatment.1

Can you round a Elaprase dose to the nearest whole vial?

It is advised that the volume of Elaprase needed for infusion is calculated using the following calculation:2

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How does Elaprase work?

Elaprase is designed to replace idursulfase (I2S) the enzyme that is deficient or absent in individuals with Hunter syndrome. Intravenous infusion with Elaprase provides Hunter syndrome patients with the exogenous idursulfase enzyme for uptake into cellular lysosomes. Cellular internalisation of Elaprase is possible due to Mannose-6-phosphate (M6P) residues on the oligosaccharide chains of the enzyme which allow it to bind specifically to the M6P receptors on the cell surface. Following internalisation of the Elaprase, the enzyme is targeted to intracellular lysosomes and subsequently catabolises accumulated GAG.1

Can Elaprase infusions take place at home?

In countries where the legislation allows infusion at home may be considered for patients who have received several months of treatment in the clinic and who are tolerating their infusions well. Home infusions should be performed under the surveillance of a physician or other healthcare professional.1

When is it best to start treatment with Elaprase?

It is generally agreed that early initiation of treatment with Elaprase can be beneficial for somatic aspects of Hunter syndrome however this is not currently reflected by treatment guidelines.3 Current data from studies of the treatment of young children with Elaprase have found similar results both in terms of the safety and efficacy of this treatment when used in patients over 5 years of age.

How should I store Elaprase?

Do not use this medicine after the expiry date which is stated on the label and carton after EXP. The expiry date refers to the last day of that month. Store in a refrigerator (2°C – 8°C). Do not freeze or shake.

Elaprase does not contain preservatives; therefore, after dilution with saline, the diluted solution should be used immediately. If immediate use is not possible, the diluted solution should be stored refrigerated at 2 °C to 8 °C (36 °F to 46 °F) for up to 24 hours.2

How should I dispose of any unused Elaprase?

Do not use this medicine if you notice that there is discolouration or presence of foreign particles. Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. Any unused medicinal product or waste material should be disposed of in accordance with local requirements. These measures will help protect the environment. Keep this medicine out of the sight and reach of children.2

Is liver or renal function affected by treatment with Elaprase?

Idursulfase is taken up by selective receptor-mediated mechanisms involving binding to mannose 6- phosphate receptors. Upon internalisation by cells, it is localised within cellular lysosomes, thereby limiting distribution of the protein. Degradation of idursulfase is achieved by generally well understood protein hydrolysis mechanisms to produce small peptides and amino acids, consequently renal and liver function impairment is not expected to affect the pharmacokinetics of idursulfase. There is no clinical experience in patients with renal or hepatic insufficiency.1

What were the common adverse events associated with Elaprase?

The most commonly observed adverse events with Elaprase treatment were infusion-related reactions, which include cutaneous reactions (rash, pruritus, urticarial, and erythema), pyrexia, flushing, wheezing, dyspnea, headache, vomiting, abdominal pain, nausea, and chest pain. The frequency of infusion-related reactions decreased over time with continued treatment. If an anaphylactoid/anaphylactic reaction occurs, the infusion should be immediately suspended and appropriate treatment and observation initiated.1

References:

  1. Elaprase Summary of Product Characteristics. 2018. Last accessed August 2018.

  2. Elaprase Guidelines for Weight-Based Dosing and Infusion 2016.

  3. Whiteman DAH, Kimura A. Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future. Drug Design, Development and Therapy. 2017;11:2467-2480.

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