Hey I have a daughter with aHUS treated with ravulizumab and every week before her next infusion her behaviour changes and no body is interested.
Hi who is the the “no body ” not interested?
Her doctors and nurses for one.
Ok we’ll come back to their reaction to what appears to be a adverse event but tell us more about her behaviour change.
Well in the week before her infusion is when her behaviour changes and she is more unhappy and miserable and she is not normally like that.
OK when does that behaviour change back?
Oddly during her infusion she seems happier and once it is over she is back to her normal self. She does not mind having the infusion. So why is this happening ? Is it drug related?
Well that would be for the manufacturer to answer.
They are not interested they ask for a survey to be completed every six months but their questions don’t apply to this.
Well they have a duty to be interested but we will come back to that. Let’s think about it how it could be due to the drug.
Think what?
Basically there are two concerns the drug itself and the way and where it is administered. Lets start with the drug.
OK.
The ingredients of the drug are same for the duration of the dose interval. For three weeks she behaves normally and for one week her behaviour changes. It is still the same ingredients. So the level of active drug changes during the dose interval. Highest level after infusion and falls at a uniform rate during the interval until it reaches the trough level before it is topped up again.
Trough level?
Yes the level at which the drug can still effectively do its job. There is usually a safety margin so there is more than enough drug to prevent a mini haemolysis- a disease break through. From time to time doctors may seek a blood test so that the trough level is measured. Has your daughter’s trough level been tested?
I don’t know.
If it had and its OK then you would hear nothing further. If it isn’t then the dose level would be increased.
Why would it not be enough when it had been OK?
This is likely to happen on or around when the weight related dose step is reached. Has your daughter’s weight changed. And have the infusions moved from 4 week to 8 week intervals.
She is growing but only the doctor would know that.
Did you notice any difference when your daughter shifted from when the lower dose interval ended one week and when the higher dose interval begun .
Dont think so?
Well if is not the drug ingredients and it does not fluctuate with changing dose level then that leaves the administration. Does she have a port or needle insert? Is it done at home or in hospital ?
I see what you are doing here you think there might be some pre infusion anxiety. Why?
It happens. May relate to how its done. Through a port at home would be less stressful for any child than by needle insert in an hospital environment.
But there are others who tell me the children display the same behaviour. We have talked about it on social media.
Disease social media groups are important for mutual support and empathy. even sympathy So someone is listening. So lets return to the disinterest shown by your doctors.
When I raise the issue I am told that no one else has raised this issue before and there were no events of this kind in the clinical trial.
Well every adverse event has to start with a first and you have found others telling you they have have seen it too. But why would your doctors know about these others. But why aren’t they curious about it and from general clinical experience, there are many other conditions which require young children to have regular infusions it is not just aHUS children. Why are they not suggesting things that could be done that work elsewhere.
Mmmm. Like what?
Well it is not only aHUS children that this may be happening to many conditions are treated with regular infusion of a drug. In fact you mention other aHUS families, when you chat to them do you talk about anything you try to improve your children’s behaviour. Which interventions work.
I don’t know we just watch it happening there is nothing can be done.
Except look on line. ask when this happens in other conditions more common than aHUS or report it to the manufacturer and see what is the response.
We cannot do it.
Your country has a way to do it and it would encourage you to do it. Also anyone around the world can report adverse events to the US Food and Drug Administration. Alexion are committed to its investigation . If all four families do so that would be a help. And maybe others will join in too.
If there was a physical reaction to a needle insert that would be an adverse event why not a behavioural reaction. Then it is a case of how often this happens in child complement inhibitor recipients
I guess that would be getting somebody to notice.
Yes which is what you wanted. The five of you can then become public patient advocates for this adverse event and maybe you’ll find others from time to time.
Would it not take a lot of time and effort.
Not really it is up to you all you want is someone’s attention
Public Patient Advocate I have never thought of myself as being one.
It may only one aspect of aHUS but you could in time be an authority on this aspect of aHUS if you wanted . It is not as if you do not want her to have the treatment you just want her to cope with it better.
.
Article No: 824
