Along with the simple messages “you are not alone” and “we can find a way” on Rare Disease Day 2019 The Global aHUS Patients’ Research Agenda is officially launched by the aHUS alliance.
It has been over four years in the making. Five Rare Disease days have happened since the idea began.
It has been crafted using
- a “suggest fest” by an alliance affiliates focus group in 2015
- questions in an alliance Global Poll about aHUS in 2016
- a rare disease day video by the alliance in 2017
Through it all, almost 200 aHUS patients and carers have revealed:
“What matters most to me in aHUS research”
The draft version of the Agenda has been around in the public domain for a while, but it needed a meeting of the alliance affiliates to approve the draft and decide to adopt it formally. The aHUS alliance affiliate organisations met in Nijmegen in the The Netherlands in November 2018.
The affiliates decided to restructure the fifteen topics into themes and then formally adopted it as The Global aHUS Patients’ Research Agenda
This is the final approved version:
- aHUS Causes and precautions
1.1 Are the predisposing genetic and triggering factors of aHUS fully catalogued and understood and will it help to know how variable are the risks of these between individuals?
1.2 When it comes to genetic testing of aHUS family members what is best – to know or not to know -and what can be done with the knowledge?
- aHUS Diagnosis
2.1 Is there a diagnosis sweet spot which can be found before a developing thrombotic microangiopathy turns into a catastrophic episode of aHUS?
- aHUS Treatment
3.1 Is there an optimal way in which a complement inhibitor can be delivered to suit an individual’s need?
3.2 Can a clinically effective therapy be developed that is affordable for all aHUS patients?
3.3 Is it more cost effective, as well as clinically effective, for the management of an aHUS patient’s treatment to undertake genetic testing?
3.4 Can a complement inhibitor be stopped safely when not needed by some aHUS patients and what makes them different?
3.5 Is there a significant difference in outcome between having a complement inhibitor before or after a kidney transplant?
3.6 Will there be a cure eventually?
- aHUS Impact – clinical/psychological
4.1 Are there long-term studies of outcomes of those in remission whether treated by a complement inhibitor or not?
4.2 Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial onset?
4.3 Does the anxiety and self-esteem of aHUS patients vary significantly between treatment types and what can be done to reduce and boost them respectively?
- aHUS Impact- Socio-Economic
5.1 How does living with aHUS impact on education and work?
5.2 Do aHUS families have all the correct information to make informed family planning decisions?
5.3 How many aHUS patients are there in my local area, my country and the world and how do they differ?
The alliance will now use this version to let the aHUS research world know what research answers aHUS patients want.
The alliance will also let aHUS patients know about future research it finds using this Agenda.
The Agenda is for aHUS patients everywhere.
A short video has been created to accompany the launch of the Global aHUS Patients’ Research Agenda and can be seen HERE
In it there are some messages from some well known aHUS researchers from around the world saying:
YOU ARE NOT ALONE
WE CAN FIND A WAY
Good will come together