2 The technology

2 The technology

Description of the technology

Pegaspargase (Oncaspar, Baxalta [now part of Shire Pharmaceuticals]) is a polyethylene glycol conjugate of Escherichia coli (E. coli)‑derived L‑asparaginase.

L‑asparaginase is a bacterial enzyme that depletes circulating asparagine, an essential amino acid on which leukaemic cells, incapable of synthesising asparagine, depend. This leads to cell death.

Marketing authorisation

Pegaspargase received its marketing authorisation in January 2016. It is indicated as 'a component of antineoplastic combination therapy in acute lymphoblastic leukaemia in paediatric patients from birth to 18 years, and adult patients'.

Adverse reactions

The most common side effects with pegaspargase (which may affect more than 1 in 10 people) are allergic reactions (including serious allergic reactions), hives, rash, high blood sugar levels, pancreatitis, diarrhoea, and abdominal pain. For full details of adverse reactions and contraindications, see the summary of product characteristics.

Recommended dose and schedule

Pegaspargase is administered as either an intramuscular or intravenous infusion.

Summary of product characteristics

Pegaspargase is usually used as part of combination chemotherapy protocols with other antineoplastic agents.

Paediatric patients and adults ≤21 years

The recommended dose of pegaspargase in patients with a body surface area ≥0.6 m2 and who are ≤21 years of age is 2500 IU (equivalent to 3.3 ml pegaspargase)/m2 body surface area every 14 days.

Children with a body surface area <0.6 m2 should have 82.5 IU (equivalent to 0.1 ml pegaspargase)/kg body weight every 14 days.

Adults >21 years

Unless otherwise prescribed, the recommended posology in adults aged >21 years is 2000 IU/m2 every 14 days.

Clinical practice

The protocols for the ongoing UKALL trials, on which current clinical practice is based, recommend a dosage of 1,000 IU/m2.

The UKALL trials have demonstrated that in clinical practice, dosing frequency depends on the patient's age, the phase of treatment in which pegaspargase is given (induction, consolidation, intensification, and so on), and the length of each phase.

The average length of a course of treatment depends on the individual UKALL treatment protocols for patients in different age groups.

Price

The acquisition cost of pegaspargase is £1,296.19 per vial (excluding VAT; price confirmed by company).

For paediatric and young adult patients, a course of pegaspargase costs between £5,144 (intermediate/standard-risk patients) and £15,246 (high-risk patients), assuming that patients complete the treatment (with no hypersensitivity) as per the UKALL 2003 protocol.

For adult patients, a course of pegaspargase costs between £6,034 (for those aged 41 years or over) and £7,544 (for those aged 40 years and under), assuming that patients complete the treatment (with no hypersensitivity) as per the UKALL14 protocol, and don't have a transplant.

Costs are based on a dose of 1,000 IU/m2 as used in clinical practice, which equates to 1 vial of pegaspargase per dose. Although the summary of product characteristics dose is higher (2,000 to 2,500 IU/m2), only 1 vial would be used per treatment administration. Costs may vary in different settings because of negotiated procurement discounts.

  • National Institute for Health and Care Excellence (NICE)