NKF Letters26 October 2011
Subject: aHUS and AHUSUK
As we agreed in our telephone conversation this morning I am following up with a summary of what we talked about.
aHUSUK has been formed by patients with aHUS and their families to provide a support group for people who encounter this disease.
aHUS or atypical Haemolytic Uraemic Syndrome is a rare disease. It has been discovered that its causes are genetic in origin particularly in the blood complement system (Factors H and I). As a result if a patient’s immune system is triggered, it becomes uncontrolled and leads to clotting/red blood cell destruction which destroys the kidneys (and other organs).
In its typical form HUS is associated with E Coli 0157 and it is why in E Coli outbreaks victims experience kidney failure .But if the HUS episode is controlled full recovery is possible with no recurrence.
In its atypical form other agents may trigger an episode, e.g. pregnancy or even a transplanted organ. Therefore it is unlikely that aHUS patients with ESRF will be able to have a successful transplant, as another episode of HUS could be triggered and destroy the kidney graft.
Until recently, treatment for aHUS has been basically plasmas exchange for those pre dialysis patients and dialysis if the recurring episodes progressively reduce and eliminate kidney function. Because the blood complement defect is created by the liver, a few combined liver kidney transplants have been successfully carried out.
But a drug has been developed which can control the blood complement over reaction and prevent aHUS patients experiencing kidney failure. Also for those on dialysis it provides an opportunity for a transplant on a level playing field with others needing a kidney transplant. And for many this would mean the first chance to be on the transplant list.
The drug is called eculizumab and as an orphan drug it is very expensive at the present time. It has been given approval by the FDA in the States and the EMA has recommended it for approval which is likely to be in the next couple of months. Then it is up to member countries to decide on whether and how to use it as the treatment of choice.
As a rare disease NICE will have permitted a direct application to the Advisory Group for NHS Specialised Services(AGNSS) to evaluate the need for it become a nationally commissioned and funded special service for aHUS in England. Their recommendation will need approval from Department of Health. aHUSUK are helping to provide the “patient voice” when AGNSS’ evaluation begins next Monday(31st)
A lobbying group (aHUS Action) has been formed to raise awareness of aHUS in the political community and those aHUS patients who are aware have been helping with this by writing to MPs about their experience. A Seminar about aHUS for MPs and Peers is to be held in Westminster on 17th January 2012. All aHUS patients and their families can attend (see below for information about invitations)
The “aware” patients are also supporting the case, along with their family members, friends, colleagues and others by signing an e petition calling for eculizumab as the treatment for aHUS in England. The petition link is http://epetitions.direct.gov.uk/petitions/16053
There are very few aHUS patients in the UK, about 170 or so and of them just over 60 are dialysis patients. Following the first ever aHUS Patients Conference held in the International Centre for Life, Newcastle upon Tyne in June (a video of which can be viewed at www.aHUS.org.uk) the small number of patients and families who attended expressed a need for a Patient and Families Support Group specific for aHUS.
On the 10 September, in the Royal Society of Medicine in Wimpole Street, London with the families of six patients attending, a Support Group was formed and is called aHUSUK. Officers and trustees were elected temporarily until the first general meeting which is planned to be held on Saturday 28 January 2012 (venue to be decided).
At the moment we know about so few patients of the overall small number with aHUS who are dispersed throughout the UK. We are looking at ways of getting aHUSUK’s existence known and engaging as many as possible to not only join with our small community and attend the inaugural AGM, but also to help with getting a first class treatment for the disease. In particular we know of very few of the 60 or so aHUS patients on dialysis.
So we would appreciate your help in letting people know about aHUSUK and giving them the opportunity to join with us if they wish. There are aHUS patients who may know nothing yet about the exciting developments in treatment. They may wish to attend the Parliamentary Seminar mentioned above details/invitations of which can be got from aHUSUK at my e mail address (the website at aHUS.co.uk will eventually become the portal for aHUSUK)
We would also appreciate the help of the renal community at large with our mission of getting the best treatment for aHUS patients by signing the e petition at the link above. It only takes a minute or so and every signature will be of help to us.
Thanks for any help you can give us and if there is any more information you need please let me know.
The National Kidney Federation cannot accept responsibility for the views expressed by others in these letters pages.