aHUS:Time is a great healer

Article No.356

17 July 2020

Two recent website articles delved into the data provided in an article by the Alexion aHUS Registry about Patient Reported Outcomes for over 500 aHUS patients.( Link to  those articles follow
aHUS Patient Fatigue  and aHUS Health Well being and Work)

The data revealed that patients say  that they can,in time, recover from their encounter with aHUS. Measures of fatigue show aHUS patients can return to levels of fatigue felt by the general population. Similarly incidents of other symptoms fall in time,  but ” anxiety” ,in particular, can linger for some though that would be hardly surprising given what had happened to them.

Data also showed that the  majority of aHUS patients became economically and educationally active.

All very positive and encouraging.

These patients were also asked to score their general health on a five point scale from Poor to Excellent. Table 1 below shows the over all scores at baseline and at last follow up.

Table 1

General Health Baseline % Last follow up %
Excellent 10.0 11.2
Very Good  23.7 28.2
Good  35.4 37.1
Fair  23.9 19.2
Poor 7.0 4.3
Total  100 100

At baseline 69.1% of patients reported they had good to excellent health and with more recovery time that increased to 76.5%. With those in poor health dropping from 7% to 4.3%. All of which is encouraging too.

In Table 2 a breakdown of the three subcategories used:  patients, never on eculizumab, on eculizumab before registry enrollment and on eculizumab after enrollment provides the direction of health status, improving , same or worsening from baseline.


General Health Never  Before After
Improving  29.7 35.1 55.0
Same  50.9 45.2 35.0
Worsening 19.3 19.7 10.0

The general health state of those in the “never“ and “before“ groups remain relatively stable but those on eculizumab, “before” enrollment reported a higher improvement in health. Not us much as those whose encounter with aHUS and treatment with eculizumab was more recent and whose improvement in health is much more marked. Nevertheless,  overall  a very positive picture is emerging.

Something that many of those with aHUS 20 years or even 10 years ago would not have thought likely. Better diagnosis and targeted treatment in the post eculizumab generation has been beneficial in avoiding kidney disease.

The data, although encouraging,  does show that a minority of patients are  not flourishing yet.

The fatigue scores those in the “after” group, who are on dialysis or received a transplant have median scores  (36  and 40) below those who have not been ( 44) despite being at similar levels at baseline ( 30,31 and 32.5).

At the time of onset,  aHUS ,as the “H” suggests, involves the destruction of red blood cells needed to carry oxygen to the rest of the body, hence the extreme anemia felt by patients. Those  whose kidneys fail also lose the ability to create the hormone  erythropoietin which is needed to stimulate red blood cell production in the bone marrow. A double blow to them .Apart from blood transfusions those with chronic kidney failure rely on a recombinant human-made version frequently referred  to as EPO. Once such patients have a transplant the new kidney begins the production of natural erythropoietin again.

Kidney function continues to play its part in the relative health of aHUS patients. The fewer that go into kidney failure the better the average aHUS patient is likely to become.

This  Alexion Registry study has been the first and so far  most significant analysis of aHUS patients burden and  quality of life. Although it was the smallest cohort, the recovery from low point of illness was very noticeable in the those who received eculizumab after enrollment.

Having said that ,although not improving as much , the  stability in their recovery attained by the other two patient groups, is another positive message to take away from this study.

It may be a few more years before follow up data is presented again. And a longer term picture of what happens to aHUS patients , as reported by aHUS patients, can be seen. A key benefit from this “longitudinal” research.

Along with clinical interventions a lot also depends on individual patient’s reaction and response in their recovery. But also, as is often said,  time is a great healer.