Treating aHUS

Treatments

Plasma Exchange/Infusion

Your physician may carry out a procedure known as plasma exchange. The purpose of this is to remove plasma from your body that contains the faulty proteins and replace it with fresh plasma containing non – faulty proteins.
This procedure normally lasts a few hours and you will need a line to be inserted into one of your larger blood vessels for it to be carried out.

Eculizumab and Ravulizumab

Eculizumab (Soliris©) and Ravulizumab are medications currently licensed for use in aHUS.
They work by blocking the action of complement and therefore preventing it from damaging the body’s own cells.
Eculizumab and Ravulizumab are given as an infusion that usually lasts up to an hour for most patients, but can be longer if the infusion is for a child.
For eculizumab, after the first doses have been given, the regular long-term dose interval is every two weeks in patients over 10kg and every three weeks for children under 10kg
Historically Eculizumab was given for the remainder of your life but this may change in the future depending on the results of a clinical trial currently running to if it is safe to stop Eculizumab / Ravulizumab infusions, and what monitoring should be done when the infusions stop.

YOU SHOULD NOT STOP RECEIVING YOUR ECULIZUMAB / RAVULIZUMAB INFUSIONS WITHOUT FIRST OF ALL DISCUSSING THIS WITH YOUR DOCTOR.

Long - term monitoring on Eculizumab / Ravulizumab

When you are receiving Eculizumab / Ravulizumab treatment, your physician willcarry out some regular tests to ensure that the drug is working and that there is noevidence ofthe disease returning.

These will include:

Full blood count – to look at the level of platelets and haemoglobin in your blood
Urea & Electrolytes–to assess your kidney function
LDH – this should return to normal levels once the disease is no longer active or has been suppressed by Eculizumab/Ravulizumab
Urine Protein-Creatinine Ratio (PCR)–measures the amount of protein beingexcreted in urine which can be an indicator of renal damage
Complement studies–to ensure that Eculizumab is suppressing your complement activity (currently we cannot measure this for patients onRavulizumab but we hope to develop this test in the future)

Dialysis

aHUS can cause severe damage to the kidneys and dialysis may be required to do thejob of the kidneys.
Like plasma exchange, this will also take a few hours and will also require a line to beinserted into your blood vessel.
It is not unusual to need more than one session of dialysis and in a small number ofcases this may continue for a few weeks or months.
In someinstances, the damage to the kidneys does not get better and dialysis may berequired in the longer term.

Transplantation

Patients with end-stage kidney disease resulting from aHUS will usually be assessedfor their suitability for a kidney transplant.
Unfortunately aHUS can occur in a transplanted kidney as the faulty complement proteins are still produced by the liver.
Eculizumab can be given to prevent the recurrence of aHUS in the transplanted kidney