The aHUSUK newsetters were a part of the work of that charity before it ceased to be . Answers for aHUS will continue keeping aHUS patients up to date. This blog news item covers several current topics.
Rare Disease Day 2017
28 February will be Rare Disease Day this year and its theme is ” rare disease research”. Although Answer for aHUS is not planning its own event, it will be supporting others.
One of the key global aHUS events is the creation of an aHUS Rare Disease Day Video where aHUS folk from around the world are being invited to participate. (The featured image gives an idea of what is involved ) . Each question page will be joined up into a video to be used thoughout the world on Rare Disease Day. More information on how to participate can be found here
“I raised £8.50 for aHUS buying a fridge freezer from John Lewis”
“I raised £3.40 for aHUS just booking a hotel for the weekend through Booking.com”
“ I raised £1.00 for aHUS doing the weekly shop at Waitrose
“I raised 17p for aHUS buying a book on Amazon”
Fundraising the easy way is just what Easyfundraising is ,if you are buying something online anyway why not use Easyfundraising to raise funds for aHUS research.
It’s simple and 26 people already are using aHUS as the cause they support to raise money for aHUS research and they have raised nearly £300. With more involved whether it’s a few pounds or few pence at a time together we could raise thousands. Why not sign up and support our research cause.
|Next generation Eculizumab is on its way
aHUS patients throughout the UK have benefited from the complement inhibitor Eculizumab. It blocks the complement cascade that it is so destructive for those affected. Patients are receiving doses of between 300 and 1200 ml at fortnightly intervals. The next generation of Eculizumab currently called ALXN 1210 has been approved by the EMA to be trialled and the Expert Centre have said that patients in the UK are to be included in that trial.
The new version of Eculizumab has a longer effective life and so a longer interval between infusions are possible. ALXN 1210 will be given every 8 weeks which means only 7/8 treatments a year.
Those currently receiving first generation eculizumab will not be included in the trial which will be for new on setting patients only; but if the trial proves successful it could be that by 2020 all will be eligible for ALXN 1210 who need it for as long as they need it .
|Withdrawal from Eculizumab Study
The study of eculizumab withdrawal which was required as part of the NICE decision for it to be used for aHUS is still going through the application process. The study group will have a representative from Answers for aHUS as a member, and the funding authority from the NHS, the NHIR, will also have input from an aHUS patient. aHUS patients’ views about this research have been taken into account since the Study Leader, Prof Neil Sheerin, spoke about it at the 2015 aHUS conference in London. The Study has achieved provisional approval meaning that it will go ahead once some of the financial and other detail is clarified.
|aHUS Patients active role in aHUS Research
As can be seen from the Eculizumab Withdrawal Study these days patients are being called upon to be more than passive data points when it comes to research. A blog on the Answers for aHUS website revealed what UK patients thought were the priorities for research click here to read. This research “agenda” was presented to a group of UK aHUS Investigators at a meeting held in September and had an influence on a programme of research topics they came up with to collaborate on. This programme of research led by Prof Nick Webb of Manchester Children’s Hospital and Dr Marie Scully of UCL Hospital (who talked about her work at the last aHUSUK AGM) will involve patients in the study teams to be set up; and volunteers from the Answers for aHUS community may be sought to participate. More information to follow.
|aHUS Alliance – Global Research Agenda
Len , UK’s representative on the aHUS alliance ( www.ahusallianceaction.org) , has recently had an article published in the Orphanet Journal of Rare Diseases. The article is about the way in which the alliance’s affiliated organisations from around the world created the first patient agenda for aHUS research; and how the Global aHUS Registry Scientific Advisory Board took it on board in developing its research programme. The article can be read (it is free to view) by following this link http://www.ojrd.com/content/11/1/154/email?from=email .
|aHUS Patient to give patient talk at Kidney Research UK Fellows Day 2017
Kelly, an aHUS patient in Wales, is scheduled to a talk about her experience of having and living with aHUS at KRUK’s Annual Fellows Day in Swansea later this year. This is another great opportunity for increasing awareness about aHUS; but it also reflects the closeness that Answers for aHUS and KRUK are forming.
|Expert Centre for aHUS
By now most affected by aHUS should be aware of the “ ahus centre of excellence” ( we have called it by many names) has been established at the National Renal Complement Therapy Centre in Newcastle upon Tyne University Hospital Trust. Led by Dr David Kavanagh and Prof Neil Sheering , following Prof Tim Goodship’s retirement , the centre has been recruiting staff and setting up its new accommodation in the Royal Victoria Hospital ,the “ RVI “, in Newcastle upon Tyne city centre.
From what Answers for aHUS has heard the Centre apart from its role in approving and funding eculizumab for patients, it will become a focal point for aHUS patients support and information and will have a website , newsletters and host patient days. It will also distribute the aHUS patients’ cards created by aHUSUK but in time will issue its own card.
For any Rare Disease having a disease expert centre is a major asset for accessing and developing treatment. Under Newcastle’s leadership the future for aHUS patients is a lot brighter.
The Centre will also be seeking a panel of patients/carers from around the county to feedback views about the service it provides so again the Answers for aHUS community will have a chance to become involved. More information to follow when the Centre is ready to communicate direct to patients and their families.
Expert Centre’s Nurses
This snippet of news has been recived from the Newcaste Nurses
“The National Centre for aHUS in Newcastle now has two Specialist Nurses working within the team and part of our role is to develop patient involvement with the service and ensure that the service we provide meets the needs of aHUS patients in England. We have recently sent out surveys* and the responses received will help to design the services we provide to you in the future. Thank you in advance for your help.
We can be contacted at firstname.lastname@example.org
Joanne Stout & Angela Watt”
*the survey has had an excellent response and will provide useful feedback to improve service and research data to be reported at National Kidney Week in Liverpool in April.