aHUS what is really happening?

Listening to some aHUS people on social media there is a sense that all are not satisfied with what is happening to them healthwise .

This is what underpins two topics in the Patients Research Agenda that matter to aHUS Patients.

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?

These questions reflect what patients are thinking AFTER having had aHUS and the physical and mental toll that can follow. Either because of a legacy of the disease or treatments needed.

The second question relates to the different treatment types – essentially dialysis , transplants and complement inhibition and how that impacts on mental health.,

The first question is about having a specific drug therapy.

There is a common understanding that no treatment therapy is without risks. Although all evaluations of new drug treatment look to see what benefits he patient, they also look to see what risks there might be for those using the therapy.

Risks can be severe and serious or can be a minor inconvenience. There are many terms to describe them from “side effects to serious adverse events.” “Adverse” sounds worse that “side” but they are effectively the same.

Identifying these risks begins in the clinical trials on a small representative number of the kind of patients who eventually will use the therapy. Those side effects reported by , or for, patients have to be declared at the time any therapy is evaluated for use. It does not mean that all potential effects have been identified during the trial. Either because more time is needed or some will be experienced by different patients who will be using it.

After the marketing authorisation of a drug it does not stop there.

It is why for all drugs licensed for use by humans ( and animals) there is some kind of follow up observation of what happens to those who receive treatment over time. To continually weigh up the risks and benefits.

So how is this follow up done?

Traditionally it is through something called Post Marketing Surveillance ( PMS) . PMS may be done voluntary by the drug manufacturer or may be imposed as a condition of approval of a license to market a drug.

Data is collected by the licensing authority. The sources are usually

  • following what happens to those in the original trials usually up to five years ( if the participants volunteer to continue ):
  • setting up Patient or Disease Registries to follow up what happens to other patients over time ( usually for 10 years )
  • Spontaneous adverse event reporting to the Licensing organisation e.g FDA or EMA by any patient or their health care provider

All are providing additional or continuous evidence of the safety of the treatment as well as its quality and efficacy based on experience in practice over time.

Eculizumab and ravulizumab are both subject to such surveillance. Data is available from the PNH and aHUS clinical trials and PNH and aHUS Registries as well as an Adverse Events reporting systems and databases set up by the FDA or EMA or other national processes..

From the first PNH trial, surveillance has been continuing for nearly twenty years, including spontaneous adverse for at least 14 years. The latter is collected on a drug by drug basis for all disease patients using eculizumab and ravulizumab. Patient data will include both those with PNH or aHUS.

According to the FDAs database cumulatively there has been over 44,000 adverse events and 4000 deaths reported to them about PNH and aHUS patients using eculizumab. There may have been more but reporting has dropped off significantly, by two thirds, during the “COVID years”. Or since ravulizumab?

It seems though that what is apparent in the public domain from PMS is of little help to the new aHUS patient facing an important clinical decision right now . The data bases above do not separate aHUS from PNH patients. PNH patients on average are much older than aHUS patients. Eculizumab has not been as efficacious for treating PNH as it has been for aHUS. Something that has been put right for PNH patients in the ravulizumab dosing regimen.

When all is said and done, however, after all this time and additional data gathered neither FDA, EMA nor any other country;s licensing authority has concluded any thing differently to when they initially approved eculizumab as safe to use. Its benefits outweighing the risks that come with it.

So why the concerns in the aHUS patient research agenda and the concerned noise in the aHUS social media. These patients have had experience. From life.

And this is another way of looking at things. Listening to what patients are saying about the reality of their health status and their health care. Are those in the social media providing useful data but it is not being captured?

Real World Data, or RWD, is becoming another way to monitor safety and collect confirmatory evidence that all is well. Previously this has been a method used at the approval stage of a new drug. It is only beginning to gain traction in the post approval stage too.

Like PMS, clinical trials and registries are data sources. But electronic health records and claims are different sources of evidence.

The key difference is Real World Data also calls for Patient Generated Health Data, or PGHD

PGHD is different to spontaneous adverse events reporting. In the latter, for example, there is no reporting by those patients who have no adverse events.

However, the practicalities of gathering data to give a more holistic picture of real life experience from patients are not without their challenges. The solutions lie in online reporting technology. All of which needs to be analysed and turned into information for decision making and action. Deploying on line monitoring and Artificial Intelligence I applications.

Easier said than done,but it is the future for all diseases , even for aHUS

But in the meantime aHUS alliance is using Rare Disease Day 2023 to highlight this important development and in a small way gather data from aHUS patients about an aspect of living with aHUS. It is about patients’ awareness rather than a clinical study. By patients for patients.

Details of how to participate are given in this previous article and also reproduced below.

Examples of other patients contributions can be seen on the aHUS alliance Facebook page and Instagram ( see link to Facebook and Instagram at bottom of the screen) .


We are asking for those who want to participate in this year’s Rare Disease Day video, to send the following.

For those who have been treated with a complement inhibitor :

  – Your Photo

  •  Name and where you are from
  • Answer “Yes”, “No”, or “I don’t know” for the following side effects /long term aHUS events listed ( see photo below too)
Side effect experiencedDue to aHUSDue to complement inhibitor
Other* ( specify if wish to)  

*specify any other side effect or long term aHUS event other than the above

Also answer answer “yes” or “no ” to the question “Do you know how to report a side effects”

For those who have not had a Complement Inhibitor :

Your photo

Name and where you are from

Answer “yes” ” no ” or “don’t know” to the following questions. ( see photo below)

Eculizumab is sold in my country

Ravulizumab is sold in my country

My Government funds treatment

Private insurance funds at least some of the cost

Jeff will then create a slide for you that will be posted on the aHUS Alliance page and the atypical HUS Families group.  And it will be included in the video/slideshow that will debut on Rare Disease Day.  In order to raise awareness leading up to Rare Disease Day we are hoping you will start sending in your submissions right away (don’t wait until the end) so we can post one or two a day from now until Rare Disease Day.

We welcome ALL those affected by aHUS from our global community to join in this project.

You can email your submission to Jeff Schmidt at jeff@ahusallianceaction.org

Submissions are due by the end of 2/17/2023 to allow time to put this together, but the earlier you can turn in your entry the better!

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