The aHUS Alliance was invited by the aHUS Registry Scientific Board to say what matters most to patients in aHUS research. The alliance’s response to that invitation is well documented in an Orphanet Journal article, which can be seen by clicking here. At the top of the alliance list was knowing what the outcomes were of transplants with or without eculizumab before an operation. This was because the alliance was both mindful that of the challenges faced by patient organisations in getting aHUS dialysis patients, needing a transplant, within scope of access to eculizumab, as new onsets with aHUS were; as well as those whose health authorities would only accept therapeutic access on re-onset following transplant. It also wanted the best evidence on which such patients should be counselled about risks not just of the operation, but also about re-onset.
The SAB subsequently made that topic the priority research project within the Registry’s research programme.
The results of that study are now nearing completion, and will be published later this year.
The alliance has become aware of an abstract on the preliminary findings of the study which were revealed at a European renal conference last year.
The high level figures show that the Registry held data on 295 aHUS patients who had had a transplant ( about 25% of patients enrolled) but not all of them had sufficient data to understand their eculizumab treatment for at least 1 year beyond their transplant. As a result only 147 patients were included in the study.
Of these there were 69 who had received eculizumab prior to the operation and 78 who did not.
-Of the 69 only one patient had not received aHUS diagnosis prior to transplant.
– -Of the 78 , 44 had been diagnosed with aHUS but 34 had not and only found our post-transplant.
What had happened to this latter group is no surprise because patient organisations have been aware of a small number of anecdotes about it occurring, but it was a surprise that it was so prevalent, at around at least 15% of all aHUS transplants. It may well have been that their doctors had not identified the cause of their kidney failure, maybe thinking it was typical HUS; or they may have missed that a TMA was the root cause.
Whatever it may have been, these patients did not fare well due to the unexpected onset and time to re diagnosis and make the drug available to help them. 6 ( 20%) of these patients lost their kidney graft and returned to chronic dialysis . 40 % of them who had need of acute dialysis recovered kidney function with eculizumab; and the remainder were effectively treated to avoid the need for dialysis.
The next worse off group were those 44 who were diagnosed with aHUS, but did not get the drug until re-onset, although fewer of them needed some dialysis 5 (11%) of them lost their kidney graft.
Those treated prophylatically fared the best although half of the 6 patients (9%) who needed needed dialysis despite having eculizumab, lost their kidney graft.
These preliminary results are challenging some of the conventional wisdom that prevails about eculizumab and transplants within the aHUS community. Advice about the risks of aHUS and timing of treatment to patients may need to be rethought.
The alliance would like to hear the views of aHUS patients on dialysis facing a transplant operation, particularly if they are from countries whose policies only permit access to eculizumab with aHUS onset after the operation. Also it would be great to hear from any transplant patient who only discovered they had aHUS following a transplant. The alliance can be contacted on firstname.lastname@example.org or perhaps leave a comment on any posted link to this article on the alliance Facebook page.
The featured image is of Dianne Illingworth, an aHUS Dialysis and Transplant Patient from the UK , who recently passed away from complications of her treatment. Dianne bravely agreed to help with the successful campaign for eculizumab to be made available in the UK for all patients when needed, for as long as is needed.This article is dedicated to her memory.