A day in an aHUS life

Article No. 401

11 December 2020

When it comes to those who have aHUS in their lives , everyone has some particular days which stand out to be remembered. 11 December 2013 was one those days for the author and the following extracts from a series of articles written by a reluctant advocate describes those 24 Hours.
It was a cold, crisp and frosty morning in Manchester, England on 11th of December 2013. Above the sky was vivid blue when it could be glimpsed through the fog that shrouded much of the UK that day. Road conditions were poor as I set off to the city.
Once inside the National Institute of  Health and Care  Excellence  office in the centre of Manchester , we were taken to the 20th floor of the “Tower” and the view from there confirmed that, above the fog, the day was bright and clear into the distance but all the well known Manchester landmarks were invisible.
The Evaluation Committee meeting began with the Chair explaining  how the meeting would run. After introductions and declarations of conflict of interests the witnesses were addressed in turn. Giving  evidence, apart from ourselves, was Prof. Tim Goodship and a Dr Rodney Gilbert from Southampton Hospital, the NHS Specialised Services leaders ( including Dr Edmund Jessop  2019 Winner of EURORDIS’ “Policy Maker  Award”) , and a team from Alexion. The academic guys from Sheffield University, who were expert in health economic evidence critique, were also there. The Evaluating Committee was made up of clinical experts from a range of medical roles, some lay members, and were supported by officials from NICE itself , some of whom we had already met.
No patient presentation had to be made, instead one of the Evaluation Committee’s lay members  summarised the case from our patient submission. His conclusion was the evidence was “rich and robust” and that “it made a compelling case for a call on NHS resources”.
WOW ! The only issue now was that I and the other patient representative said nothing that would detract from that view! The only criticism of our report was that there not enough stories about patients who had received eculizumab. That was true but few had accessed it so far and only some of those who were trialists were known to us at the time.
It had been difficult to find aHUS patients, as we had told NICE at the outset, and eculizumab had not been approved so by definition such patients were few and far between. However my aHUSUK  patient expert colleague was a recipient of eculizumab but was not included in the report as it had been completed before she joined us. She was able to tell the Committee how ill she had been with loss of kidney function and needing dialysis after plasma exchange became less and less effective. She had been included in the eculizumab trial and was given doses of the drug. Soon her aHUS came under control and she recovered some kidney function. She came off dialysis and began to feel better. With better health she returned to work and got married. She was also starting a family. That is what eculizumab can do.
When asked for any concluding comments I remembered what my daughter had said at the first AGNSS meeting. Eculizumab for those on dialysis meant FREEDOM. Freedom from living a life around a dialysis machine, Freedom to eat and drink as we all do, Freedom to work and have a full life again. It goes without saying that those newly onseting would no longer need to know what that aHUS life would be like.
Unexpectedly the expert from Sheffield University in his critique mentioned an article on the aHUSUK website about a visit to Milan to find out more about the pioneering work of Dr Gianluigi Ardissino in adjusting and tapering the doses of eculizumab in his patients ,and with some withdrawing from treatment once stable. This of course meant, unlike the “licensed full dose for life” on which the Committee had to consider the drug , that the average cost per patient would be much less. We could not have brought that up, it was not part of our evidence, but now it was part of the discussion and some doubt about the true treatment cost had crept in. Eculizumab when needed for as long as needed . It should not be wasted at that price.
The morning session went very quickly and there was a break for lunch. The meeting was going very well. In part of the afternoon session  Alexion was  asked to talk to the Committee without anyone else present; such was the commercially sensitive nature of the talk about its price for eculizumab.
It was a relief when the meeting ended and “witnesses were released”. I do not know if it had been the coldness of the day or the dryness of the air-conditioning, but by the beginning of the afternoon my voice was disappearing and became croaky! Maybe it was nature’s way of saying “ shut up and say no more, the patients’ voice had already done its job”.
The Committee would spend the rest of  meeting making its decision in private. Our main concern was about what Alexion had said during its closed session, it could be very detrimental.
We had been told that the next step would be the release of the Evaluation Decision document ( the “product” of this stage) . It was likely to be published sometime in late January 2014 ( after our third Christmas as a charity).
Personally none of that seemed to matter to me for now because during the lunch session a message had got to me (I was not one for mobile phones!) . It was from my wife. The hospital had called. There could be a kidney for my daughter.
There was no rush!
It may seem odd that on the very same day that NICE was making its decision on whether eculizumab should be made available for  aHUS  patients throughout England  that my daughter could be about to receive it to support a kidney  transplant.
Some might say that this was because of the CPAG decision to extend scope of eculizumab to dialysis patients. That was a possibility.
Except my daughter had been called as a potential recipient twice before. Those calls came before the CPAG decision. On those occasions the kidneys were thought to be too marginal for her and they  might be have been donated to the “reserve patient” . Reserves are always called up and most are  disappointed  and stand down.
So why had this happened at all?
Our knowledge about eculizumab’s existence went back to before 2004 . We were not told its name but in passing in the hospital corridor her transplant surgeon from her first transplant mentioned to my daughter that something was coming which could be of help. In 2004 work began for a transplant listing work up but was stopped when a doctor said that he would seek  Prof. Tim Goodship’s advice. Nothing more was heard, but at every clinic she asked how the listing was going, only to be told that her notes said that this doctor was still looking into it.
My daughter had not been on the transplant list before her first transplant , that  same doctor from the near derelict hospital which she was treated in had  failed to do so. So her first transplant was with a kidney donated by her mother. That was on the Thanksgiving Day 1999. By Millenium Eve the decision was made to remove the failed graft. aHUS  recurrence was the cause but it was clear the clinicians treating her had no idea until we told them that it was a possibility and that she should be given plasma exchange. We were ignored and dismissed. But we  continued and eventually she was given fresh frozen plasma, and not PEX, to treat the TMA. Although they, now looking back, very recklessly zapped her  whole immune system for rejection , putting her at risk of infection , it was too late. It then emerged that the promised management of her  transplant between the derelict hospital and the one where the transplant took place had failed because of politics between two organisations which were going through a managerial change.
Had she been on the transplant list the fact that the graft failed within six months, it did not last 6 days,  would have meant she would have retained her waiting time credit, which would be important in a future  allocation decision ,all other things being equal.
Even if she had been put on the transplant list in 2004 she would have accrued 7 years waiting time. When we asked Prof Goodship at the Wimpole Street meeting in 2011 to create aHUSUK , what would happen to aHUS patients who had not been on the transplant list . He said they would go to the bottom of the list. So even if a decision was made to provide eculizumab my daughter could be looking at a further five year wait. That seemed unjust as those aHUS dialysis patients who had been listed, but suspended and not transplanted ( which was protocol ) could have accrued enough time to be top of the list when their temporary suspension was lifted. What should be a fair and just system of allocation was, in practice, flawed.
So when we left that first aHUSUK meeting in London we were determined to address that injustice. For that we had to switch to private patient engagement advocacy.
We began by writing to the hospital quoting the six month rule. We were told with some certainty that would only apply if someone had been on the kidney donor list. Correspondence continued until we asked whether it was accidental (negligence) or deliberate  (willful) whether she had not be treated fairly by that Doctor. The correspondence continued and escalated  through  2012 so by the end we were having to write through the Chief Executive of the hospital such was the nature of the “complaint” it had become.
The hospital was saying that it was my daughter’s fault she had not  been listed because someone had written in her notes that she had said that “she had not wished to go BACK ON the list” . It was that “note” on which the hospital was defending its position. But when we pointed out she could not have said that ” because she had never been ON the list” the Hospital relented and agreed to approach the donor list authorities to put right her waiting time, which that authority did. Private patient advocacy can work but it is not easy. It takes an enormous amount of time to do ( and we were also active in public advocacy too) .There can be much resistance to your view ,however right you may be . It also emerged that the doctor who everyone thought had been reviewing the advice for now eight years had not been doing that at all, but had neglected to tell anyone. All this could have been sorted out years before if communication had been up to professional standard. The precedent having been set it  would change the policy and benefit all aHUS patients similarly affected.
The other factor that may have had a bearing on the call up before eculizumab was approved for dialysis patients, was that during the correspondence with the hospital one doctor said that we should apply for a Individual Funding Request, IFR, for eculizumab.  No IFR had been approved for an adult aHUS patient, and certainly not one for an adult aHUS dialysis patient. But we agreed to try and were asked to make the case so the hospital could make the request. I remember it took two days of research and draft rewriting  to produce  a two page case of why an IFR should be made in my daughter’s case. We were not confident after we gave it to the hospital when the hospital staff told us that “no one at the hospital could have made the case as well as we had done”, but the application went ahead.
This was all happening concurrently with the “reorganisation” of the NHS which  was taking place in 2013 and decision making committee structures  were changing.  The same reorganisation which had forced eculizumab going to NICE for re evaluation. We waited and waited. Our MP had become involved and was prompting the decision making group for a decision. Finally it was approved in May 2013 and with my daughter’s waiting time credit given too , the transplant work up which began in 2004 was concluded in June 2013.
It had not been an easy journey.
Very soon after the donor listing confirmation letter ,the first call came. Then the second and now the third call.
Would this be the one?
There was no rush!
Strange  how the media portrayal of transplant stories is of a quick action drama , with blue lights flashing , does not seem to be so in the real world.
I finished the NICE meeting. My wife and my daughter went to watch our grandson’s  school play as previously arranged.My daughter had been to the hospital for the usual pre- transplant check for antibody compatibility etc.
Until the “go ahead decision”  it was not thought odd that a  prophylactic  eculizumab infusion had not been done as per protocol for an aHUS patient transplant.
In the early evening she returned only to be told she could go home to wait and return in the early hours of the following morning. There was going be a transplant .
Our  family went to the hospital as requested at 2am and got settled into a room on the transplant ward. Still no eculizumab infusion and we began to mention it to the staff who seemed unconcerned. Then we got the news that she was to be next in theatre and a porter would arrive soon to take her down to theatre.
We were alarmed after all the time spent getting access to the drug she needed no one seemed to care that she was not to be given it. I spoke to the Sister in charge of the ward and told her  that according to protocol she was to receive eculizumab before surgery. With the usual afront to being challenged I was told that they knew what they were doing , they did it all the time. I pointed out that was not the case as this was the first UK cadaver donated kidney transplant supported by eculizumab ever, so this was different.
It made no difference they would not listen. In less than 24 hours I had been relegated from an expert witness  being listened to at a NICE meeting about aHUS and eculizumab , to being a numpty over anxious parent who did not know what he was talking about. Yet I was the one who knew what the official clinical protocol was. I was on a committee that established it.
This demonstrates that no matter how detailed and correct a clinical protocol can be decided upon by experts and eminent medical practitioners , it could all be for nothing in practice,  let down by the weakest link in the delivery chain. That Sister , that night , was the weakest link. She did not know she was, she was used to the common routine not the rare and innovative. No one had told her. Now  she  would not listen.
We were just as we were in the first transplant in 1999. “ Plus ca change plus c’est la meme chose”
Except there was a new interim aHUS Expert Centre in Newcastle. aHUSUK had designed a patient card with contact details . We got the telephone number from it and asked to be helped. Within a few minutes we had a call back , it was Prof Tim Goodship he was on duty I had only seen him less than 12 hours earlier at the NICE meeting.
We told him what was happening and he said he would contact the hospital.
Meanwhile the porter arrived and my wife confronted the health care staff refusing to let our daughter to go to theatre until Prof Goodship had spoken to the hospital.
Time passed and then we heard that her operation had been rescheduled and that the pharmacy had now been asked to supply the drug. After nearly 13 years of waiting it is hard to believe that we would ever put anything in the way of a transplant but that was what we had been forced to do.
The drug came eventually and infusion begun. It had not finished infusing when another porter came to take her to theatre, so off she went with the saline drip following her trolley.
Now the situation changed as it became what all families facing a transplant feel. Would the operation go well, would the grafted kidney work. The risks in recovery while immunosuppressed and the regime of medicines to take daily ( transplant patients in the media never seem to have to face that most dramatic of changes).
In essence that is all aHUSUK had ever wanted for aHUS dialysis patients. Just to get them on a level playing field with other transplant patients. We knew that life with a transplant came with baggage but it was nothing like the burden of dialysis. Even the two week eculizumab infusion for life was more endurable than sticking needles in arms five or six times a week  to link up to a dialysis machine. It was to be freedom as the Trustee for aHUS Patient Voice , who was now in theatre, had said
But at that time for the reluctant advocate it was just about sitting and waiting for the operation to be over. When all had been said and done that had led to that moment, that other realisation enters  transplant patients’ and their families’ thoughts.  Someone somewhere had lost their life today and their family were having the worst day of their lives.
They would be remembering  those 24 hours for a much different reason today.
11th December 2013 – a bitter sweet day.”
Seven years have passed post transplant.
There was an aHUS life before 11 December 2013 and another life since.
There are so many thanks to be given to so many people for improving aHUS patients’ lives, but on that day one life made a such a difference to an aHUS life and it will not be forgotten.
Note: The updated book “A reluctant advocate” will be published in the New Year.

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