Review of the aHUS patient research agenda (Part 1)

During December and January last, there has been a review of each of the 15 topics in the patients research agenda. Many of the topics were included in the first agenda which was created nearly 9 years ago. The most recent version has been around for over 5 years. So it was time to look at it again to see what progress has been made.

Usually a task like this would require what is known as a “literature search” . This is where published articles on the named topic are searched for on the internet. Sifted for relevance and the conclusions of each summarised to determine the current to state of play.

aHUS alliance Global Action has not got the capacity to do that so we asked an expert to summarise an answer for each of the topics. For those who have read all the articles ( all the topics below are linked to the relevant website article) it will come as no surprise that the expert we asked was none other than AI app Chat GPT. AI responses are based on searches that the “robot” undertakes.

Yes it is cheating a bit but if any viewer feels inclined to do a literature search and publish what is found they are more than welcome to do so!

Below is summary of the answers to the current agenda. It is a mixture of mainly positive answers , some negative and some qualified. The overall impression is that patients’ concerns are being addressed either by clinicians , academia, industry ( manufacturers and genetic testing labs) , and patients themselves.

So what next?

Firstly do we still need to have a research agenda and are patients already satisfied with the answers they have to empower them for managing their aHUS?

Are all aHUS researchers satisfied that they know what is important to aHUS patients?

If no then the agenda clearly needs some rationalisation and reformatting and to get as wide spread communication reach to the wider aHUS community.

So more work to be done before any decisions can be made. See Review of the aHUS patients research agenda ( Part 2)

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?
-No to fully catalogued
-Yes to understood
-Yes to help to know
2When 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?
-No don’t know what
is best
-Yes some uses are evident
3Is there a diagnosis sweet spot which can be found before a developing
thrombotic microangiopathy turns into a catastrophic episode of aHUS?
-Yes it is in the period
up to 7 days
from presentation of a
TMA manifestation to a specialist clinician
4 Is there an optimal way in which a complement inhibitor can be delivered to
suit an individual’s need?
5 Can a clinically effective therapy be developed that is affordable for all
aHUS patients?
6 Is it more cost effective, as well as clinically effective, for the management
of an aHUS patient’s treatment to undertake genetic testing?
7Can a complement inhibitor be stopped safely when not needed by some aHUS
patients and what makes them different?
-Yes to stopped safely
-Difference only partially
8Is there a significant difference in outcome between having a complement
inhibitor before or after a kidney transplant?
9Will there be a cure for aHUS eventually?Probably
10 Are there long-term studies of outcomes of those in remission from aHUS
whether treated by a complement inhibitor or not?
11Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial
aHUS onset?
12Does the anxiety and self-esteem of aHUS patients vary significantly between
treatment types and what can be done to reduce and boost them respectively?
-Yes to vary significantly
-Yes there are things
that can be done
13How does living with aHUS impact on education and work?Varies between acute
phase and recovery
14Do aHUS families have all the correct information to make informed
family planning decisions?
No not always
15How many aHUS patients are there in my local area, my country and the world
and how do they differ?
Not known only estimates available

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