The aHUS Alliance, an umbrella group of aHUS advocates and patient groups in over 30 nations, launched its aHUS Global Patients’ Research Agenda on Rare Disease Day 2019. An international project developed over 4 years, the aHUS Global Patients’ Research Agenda lists 15 central questions grouped into 5 main categories:  Causes and Precautions, Diagnosis, Treatment, Impact: Clinical/Psychological, and Impact: Socio-Economic.
The aHUS Global Patients’ Research Agenda was crafted using input from an alliance affiliates group in 2015, survey data from the 2016 aHUS Global Poll, and international entries from aHUS patients and caregivers for the aHUS Alliance 2017 Rare Disease Day video project.

The importance of the aHUS Global Patients’ Research Agenda was described in this aHUS Alliance video, with the agenda itself available as a print friendly pdf document offered in 3 languages:  EN, ES, and FR.

Click to WATCH the VIDEO

Q1 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?

Q2 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?

Q3 Is there a diagnosis sweet spot which can be found before a developing thrombotic microangiopathy turns into a catastrophic episode of aHUS?

Q4  Is there an optimal way in which a complement inhibitor can be delivered to suit an individual’s need?

Q5 Can a clinically effective therapy be developed that is affordable for all aHUS patients?

Q6 Is it more cost effective, as well as clinically effective, for the management of an aHUS patient’s treatment to undertake genetic testing?

Q7 Can a complement inhibitor be stopped safely when not needed by some aHUS patients and what makes them different?

Q8 Is there a significant difference in outcome between having a complement inhibitor before or after a kidney transplant?

Q9 Will there be a cure eventually?

Q10 Are there long-term studies of outcomes of those in remission whether treated by a complement inhibitor or not?

Q11 Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial onset?

Q12 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?

Q13 How does living with aHUS impact on education and work?

Q14 Do aHUS families have all the correct information to make informed family planning decisions?

Q15 How many aHUS patients are there in my local area, my country and the world and how do they differ?