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 she was treated 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 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 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?
Link to 11th December 2013 click here.
