aHUS health, well-being and work

Article No. 355

13 July 2020

Among the the topics included in the Global aHUS Patients’ Research Agenda are the following :
Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial onset?
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?
How does living with aHUS impact on education and work?
Recently an article about aHUS Fatigue featured on this website ( Read HERE). Research revealed that prior to treatment extreme tiredness was an evident symptom of an aHUS onset; but with treatment and time,  patients could return to the kind of fatigue that normal people live with. This suggests that it is a temporary ailment and not a side of effect of their complement inhibitor.
A report of that study of aHUS Fatigue was published in Kidney International Reports and can be read at the following link
But there was more to that publication than just the “aHUS Fatigue” ailment.
Apart from the FACIT – Fatigue instrument, which the Alexion Registry uses to gather data about Patient Reported Outcomes, another set of questionnaires is used to gather patients’ experience of other symptoms of aHUS and its treatment. These questionnaires cover incidents of symptoms associated with serious illness , and also  what these aHUS patients do  e.g work and other activities.
Using a study cohort of 511 patients and the same subcategories created for the aHUS FACIT Fatigue analysis, i.e. those on eculizumab BEFORE enrollment, in the Registry , those starting eculizumab treatment AFTER enrollment and those NEVER receiving eculizumab,  their reported outcomes are described and compared.
Starting with data about temporary and possible on going symptoms/ailments,  including Anxiety (which will be looked at separately. Patients were asked to indicate , from a predetermined list of possible symptoms,  which of them they might have had  an incident of in the time since they last completed the questionnaire.
The generic list includes Abdominal pain, Anxiety, Confusion, Diarrhea, Easy bruising/ Abdominal bleeding, Fatigue ,Headache, Irritability, Nausea/Vomiting, Shortness of breath , Swelling, Weakness, Jaundice and Other.
From that list the top three most reported symptoms were Fatigue, Headache and Weakness. The following Table 1 displays the percentage of patients reporting such incidents at baseline and in a follow up  questionnaire.
Table 1

Fatigue Baseline Latest
Never 55.7 61.8
Before 63.4 66.3
After 95 65,0


Headache Baseline Latest
Never 51.9 53.5
Before 55.2 55.6
After 70 65


Weakness Baseline Latest
Never 43.3 39.2
Before 47 44
After 70 45

More patients (70% plus) in the starting treatment after enrollment group reported these symptom incidents. Fatigue at 95% was clearly the most likely symptom that aHUS patients would experience when ill.
Though such incidents drop as health returns, only fatigue and weakness fall to the levels experienced by those never ,or ,receiving eculizumab before  enrollment.  Headaches although falling ,remain much higher. The similarities between never and ever on eculizumab suggest the symptoms are more to do with the effect of aHUS than the treatment.
Patients also reported incidents of the all the other symptoms but fewer than half of them did so. For brevity ,in Table 2 ,these have been put into “decile” groupings e.g.  0-10 % etc to show the overall relative incidence reported after treatment . Incidents of all symptoms fall with recovery from illness with some reported as significantly down compared with pre-treatment levels at the start of treatment.
Table 2

0 to 10% At start Direction Significant
Jaundice 30 Down Yes
Other 10 Down No


10-20% At start Direction Significant
Chest pain 30 Down No
Confusion 25 Down No


20 to 30% At start Direction Significant
Abdominal pain 35 Down No
Diarrhoea 50 Down Yes
Bruising 35 Down No
Nausea/Vomitting 60 Down Yes
Swelling 55 Down Yes


30% plus At start Direction Significant
Irritability 65 Down No
Shortness of breath 55 Down No

The symptom “Anxiety” has been kept separate from the other ailments reported above because it is referred to separately in the patients’ research agenda. The patient reported outcome questionnaire does not include the psychological condition of self esteem ( nor its inverse state “depression”) but it does include data about aHUS patients anxiety.
Table 3 shows that prior to treatment with an aHUS onset, more than half of patients experience it. It falls after after treatment , but not quickly and it seems to stay at a level where 1 in 3 patients report feelings of anxiety even as time passes. It seems  it is not dependent on whether patients have been or never been on eculizumab so that it would appear not to be a side effect.
Table 3

Overall  32.7 31.9
Never 32.5 29.2
Before 31.2 33.0
After 55 45

This data does not indicate how severe these incidents are, nor what, if any, interventions there have been. It can be perfectly normal to feel anxious for any number of reasons. It is when it becomes constant,or debilitating that it becomes a problem. General anxiety disorder is estimated to  affect 5% to 18 %  of the population.
Given the continued levels reported there may be something more specific to aHUS given the experience of onset and its serious  threat to the patient’s life. Anyone’s confidence can be rocked after that. Increasingly there are reports that there will be a surge of stress in those survivors of COVID 19 with the trauma of  experiencing the severe near death form of the  illness. So there could be something in that. Those that recover can be reassuring to those in the midst and aftermath of that encounter.
Further follow up  by this study will reveal what course this symptom will take in the long term. But for now hopefully patients will be receiving help if needed or take control by pursuing self- help if they choose.
A third questionnaire completed by participants in the Alexion Registry relates to work and other activity.
It is rare , if at all, to see such data reported about aHUS patients. Table 4  below gives details of how 511 of the patients in the study spend their time. This is the overall position as the researchers found little difference between those who were ever or never receiving eculizumab.
Table 4 

Activity No. of Patients % of total
Working full time 108 21.1
Working part time 56 11.0
Students 117 22.9
Unemployed not aHUS related 31 6.1
Unemployed aHUS related 53 10.3
Retired 26 5.1
Other : mostly self employed,homemaker or disabled 120 23.5
Total 511 100

Over half of aHUS patients are working or in education, possibly nearly two thirds , if some of those in the “other“  category who are self employed and homemakers are included. This is surprising but welcoming data which shows what normality  aHUS patients are reaching.
Having said that  16 % reported being unemployed just under 2 in 3 of them giving  aHUS as the reason. Some of the “others” regard themselves as disabled. 1 in 20 aHUS patients are retired.
Two articles written by two aHUS patients, Wim and Kamal, come to mind to show how they chose to recover from their aHUS experience ,whether ever or never on eculizumab. The articles below may be of inspiration to other aHUS patients today who are  just starting off their recovery.


There is still more to look at in this study and Global Action will return to it.

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