Useful Information

On this page you will find links to further information that may be of use to patients with aHUS and their families.

Additional information can be viewed on our useful links page.

Homecare Infusion Service

  • Alexion fund a free service that allows patients on Eculizumab for aHUS to receive their infusions in their own home. It may even be possible for you to have your infusions in your workplace or in school when appropriate.

  • If you would like to find out more about this service or are having any issues or problems with it then please contact the aHUS Specialist Nurses at ahus.nurses@nhs.net.

Meningitis Prevention

  • Eculizumab interferes with your body’s ability to deal with the bacteria that can cause meningitis. The following measures will be taken to reduce the chances of you developing this disease whilst on the drug.

    • Your local team will ensure you receive the relevant meningitis vaccinations.

    • A blood test will be done to check your response to the vaccination. If needed you may be given a further booster dose.

    • Your physician will also prescribe long-term antibiotics. It is vitally important that you continue to take these for as long as you remain on Eculizumab.

    • A throat swab will be done to check if you are an asymptomatic carrier of the bacteria.

  • Once all precautions are carried out the risk of you contracting meningitis is extremely small but cannot be completely eradicated.

  • You should make yourself aware of the signs and symptoms of meningitis and seek advice as soon as possible if you are concerned. Information on this can be found at

http://www.nhs.uk/conditions/Meningitis/Pages/Introduction.aspx

  • The National aHUS service produces cards that you can carry that provide information for health care staff on the risk of meningitis and the actions to be taken.

  • If you would like one of these cards then please contact us at ahus.nurses@nhs.net.

Genetics and aHUS

  • In the majority of cases, aHUS is caused by a genetic fault in a component of the complement system.

  • These faults can be in 2 areas of the system:

  • The protector proteins that usually stop complement attacking the body’s own cells

    • Factor H

    • Factor I

    • CD46, also known as Membrane Co-factor Protein

  • The proteins that make up complement

    • C3

    • Complement factor B

  • In addition there are some rare genetic variants where Eculizumab therapy is not effective.

All patients with a suspected diagnosis of aHUS will have genetic screening performed  

  • A genetic fault is found in approximately 50% of patients referred.

  • Where a known genetic fault is identified, genetic screening can then be offered to family members such as siblings, parents or offspring. This can be done locally or individuals or families can receive an appointment to meet with physicians from the National aHUS Service in Newcastle.

  • At risk individuals will be given advice and counselling on what to look out for and how to seek assistance if required.

  • Work is still being undertaken worldwide to identify further genetic faults associated with aHUS.

Auto-antibodies

  • Alongside genetic testing, samples will also be checked for the presence of auto-antibodies.

  • This is currently a research test carried out at the complement research laboratory at Newcastle University.

  • In aHUS, auto-antibodies will attack the protector proteins, Factor H and Factor I, and stop them from working properly.

  • It is possible to have both a genetic fault and an auto-antibody.

PatientView and RaDaR

  • The National aHUS service is required to provide regular updates to the commissioners at NHS England on the progress and outcomes of all patients who come under the care of the service.

  • Under the shared care agreement between the local team and the National aHUS Service, physicians will therefore be asked to provide regular patient updates including latest blood results.

  • In order to help with this, your local physician will ask you to register for the following services. Registration is completely voluntary and will not in any way prevent your access to Eculizumab.

 

PatientView

  • PatientView is a system that takes information such as blood results from your local hospital’s computer system and allows you to view them from your home computer or electronic device.

  • Once you are registered for the service then other medical practitioners involved in your care, such as your GP, can also see your results.

  • It will also allow RaDaR to collect up to date information about your condition alongside results from other people with the same disease.

  • Further information can be found at

www.patientview.org

 

RaDaR

  • RaDaR is the National Registry of Rare Kidney Diseases and is an initiative run by the Renal Association in the United Kingdom.

  • Its aim is to bring together information on patients with a rare kidney disease such as aHUS, thereby improving our understanding of these diseases and therefore outcomes.

  • Further information can be found at

http://rarerenal.org/radar-registry/

 

The National aHUS Service

  • These registrations will allow the team at the National aHUS service to receive reports detailing your latest blood results and will speed up the process of collecting this data.

  • Please be reassured that your personal data will not be shared with any other parties and all outcome measures will be anonymised in any reports generated.

  • Registration is not compulsory but will speed up the process of getting the required information to the National aHUS Service and reduce the number of requests we will have to send to your local physician.

TTP

  • Thrombotic Thrombocytopenic Purpura (TTP) is a rare blood disease that can present in a similar way to aHUS.  

  • It is also characterised by a low haemoglobin and platelet count, raised LDH and fragmentation on blood film.

  • Neurological problems are the most common symptoms at presentation though the kidneys can be affected.

  • TTP is caused by a deficiency of an enzyme known as ADAMTS13.

  • ADAMTS13 is responsible for breaking down von Willebrand Factor in the blood and a deficiency of it will lead to clumps of platelets forming in the small blood vessels.

  • Plasma exchange is the recommend treatment for TTP and not Eculizumab.

  • In most patients, it is important to distinguish between aHUS and TTP before Eculizumab can be used to treat aHUS.

  • The best way to do this is to measure the level of ADAMTS13 in the blood.

  • Further information on TTP can be found at

http://ttpnetwork.org.uk/

Typical HUS

  • ‘Typical’ haemolytic uraemic syndrome may also be called STEC HUS, E-Coli HUS or diarrhoea positive HUS.

  • It is a condition caused by infection with bugs known as shiga-toxin Enterococci.

  • The most common of these in the UK is E.coli O157.

  • These bugs cause severe gastro-intestinal upset usually characterised by watery, bloody diarrhoea. They also attack the cells that line the blood vessels in the kidney causing the symptoms of HUS and damage to the kidneys.

  • Although this illness can be severe and some patients may require some dialysis until their kidney function improves, the majority of patients will recover completely and have no long-term problems.

  • In order to rule out typical HUS, the medical team will collect samples of faeces and a blood sample to check for any evidence of infection with the bugs that cause it.

  • Eculizumab is not known to be of benefit in the treatment of typical HUS and the drug will usually be stopped if any of these tests are later found to be positive.