Atypical haemolytic uraemic syndrome
The Pharmaceutical Benefits Scheme subsidises eculizumab for the treatment of patients of atypical haemolytic uraemic syndrome (aHUS).
Section 100 arrangements
Authority to provide eculizumab (Soliris®) for the treatment of atypical haemolytic uraemic syndrome (aHUS) is available under section 100 of the National Health Act 1953.
This item is only available to a patient who is attending either:
- an approved private hospital
- a participating public hospital, or
- a public hospital
The hospital provider number must be included on the application form.
The Schedule of Pharmaceutical Benefits on the PBS website outlines restrictions for prescribing eculizumab (Soliris®) to patients.
Patients starting treatment with eculizumab (Soliris®) must meet all aspects of the initial criteria.
Patients will be approved for a maximum of 24 weeks treatment and must be assessed for their response to treatment at its completion. Eligible patients may continue to receive 28 weeks of treatment as 'extended initial 1' treatment.
At the completion of an 'extended initial 1' treatment course, patients must be assessed for their response to treatment. Eligible patients may continue to receive 28 weeks of treatment as 'extended initial 2' treatment.
Patients who have demonstrated an adequate response to a total of 80 weeks of combined initial treatments are eligible for continuing treatment.
You must provide serial hematological results every 3 months while the patient is receiving treatment.
Patients will be approved for a maximum of 28 weeks of continuing treatment.
Recommencement of treatment
Patients who have demonstrated an adequate response to treatment prior to a break in treatment may recommence if they experience a disease flare.
Life-threatening and fatal meningococcal infections have occurred in patients treated with eculizumab (Soliris®).
Meningococcal infections can rapidly become life threatening if not recognised and treated early.
Patients must comply with the current recommendation for meningococcal vaccination in patients with complement deficiencies. This is directed by the Advisory Committee on Immunization Practices (ACIP).
You must immunise patients starting treatment with eculizumab (Soliris®) against meningococcal. You must provide a vaccination certificate with each application.
For additional guidance on the management of the risk of meningococcal infection, refer to the Product Information for eculizumab (Soliris®).
You must monitor your patients for early signs of meningococcal infections and evaluate immediately if you suspect infection.
Schedule item details
Dose - eculizumab (Soliris®) is presented as a vial containing 300 mg/30 mL.
For patients 18 years of age and older, eculizumab (Soliris®) therapy consists of:
- 900 mg weekly for the first 4 weeks, followed by
- 1,200 mg for the fifth dose 1 week later, then
- 1,200 mg every 2 weeks thereafter
For patients younger than 18 years of age, eculizumab (Soliris®) should be administered based on body weight, as per the following table.
Dosing recommendations in patients less than 18 years of age:
|Patient body weight||Induction||Maintenance|
|40 kg and over||900 mg weekly x 4 doses||1,200 mg at week 5,
then 1,200 mg every 2 weeks
|30 kg to less than 40 kg||600 mg weekly x 2 doses||900 mg at week 3,
then 900 mg every 2 weeks
|20 kg to less than 30 kg||600 mg weekly x 2 doses||600 mg at week 3,
then 600 mg every 2 weeks
|10 kg to less than 20 kg||600 mg weekly x 1 dose||300 mg at week 2,
then 300 mg every 2 weeks
|5 kg to less than 10 kg||300 mg weekly x 1 dose||300 mg at week 2,
then 300 mg every 3 weeks
Eculizumab (Soliris®) should be administered on, or within 2 days of, the recommended dosage regimen time points.
Authority prescriptions should be written as:
- the actual number of vials required per dose, and
- the appropriate number of repeats to provide either:
- 24 weeks of initial treatment
- 28 weeks of ‘extended initial 1’ treatment
- 28 weeks of ‘extended initial 2’ treatment, or
- 24 weeks of continuing treatment
Patients must meet the relevant criteria as indicated in the PBS restrictions and be eligible for the Pharmaceutical Benefits Scheme.
Lodging an application
All applications must be made by, or in consultation with:
- a paediatric nephrologist
- a nephrologist
- a paediatric haematologist, or
- a haematologist
All applications must include sufficient information from the patient's file. The test results, which apply to each patient’s circumstance, are listed in the relevant form.
For a patient starting initial treatment, use the Atypical Haemolytic Uraemic Syndrome (aHUS) – eculizumab Initial PBS authority application Supporting information form (PB119).
For a patient starting extended initial 1, or extended initial 2 treatment, use the Atypical Haemolytic Uraemic Syndrome (aHUS) – eculizumab Extended initial PBS authority application Supporting information form (PB175).
For a patient continuing treatment, use the Atypical Haemolytic Uraemic Syndrome (aHUS) - eculizumab Continuing PBS authority application Supporting Information form (PB125).
For a patient recommencing treatment, use the Atypical Haemolytic Uraemic Syndrome (aHUS) - eculizumab Recommencement PBS authority application Supporting Information form (PB176).
Applications should be faxed to the fax number dedicated to the aHUS program. This number is 1800 785 672. We will then contact the prescriber by telephone. For this program it is not necessary to mail the completed application to Complex Drugs Programs.
For more information contact us on the Complex Drugs enquiry line.