The alliance has frequently featured articles about kidney transplants for those with aHUS. It was its top topic identified as a priority for research that matters to aHUS patients , when asked by the SAB (Scientific Advisory Board) of the international aHUS Registry.
The impact of eculizumab in countries where access is allowed has reduced the number of aHUS patients who end up on chronic kidney dialysis and needing a transplant.
Nevertheless it remains a question on the aHUS Patients Global Research Agenda because dialysis patients on the waiting list needing eculizumab for a successful transplant are frequently discriminated against when it comes to access policy in many countries. Some countries will only permit access on recurrence of aHUS. So dialysis patients, who have already experience kidney loss from an aHUS onset are rightly concerned about whether not getting prophylactic eculizumab has a detrimental effect on transplant outcomes.
Transplant outcomes can be looked at from both the quality of the graft function and its longevity.
The results of the aHUS Registry Study were published in KIdney International earlier this year and a report about it featured on our website , which can be seen here. The results revealed that better kidney graft function was achieved with prophylactic eculizumab therapy. OK function was still maintained when eculizumab was prescribed as rescue therapy when patients had been previously diagnosed with aHUS. Those who had not been previously diagnosed with aHUS as the cause of their kidney failure but who had a second episode following transplant fared much less well. Their graft function was not much better than being on dialysis.
The longevity of the grafts in the aHUS Registry are not reported although the undiagnosed with poor function did tend to return to kidney dialysis quite quickly.
The data on renal function outcomes did persuade one country’s (Australia) policy makers to revise their policy on offering prophylactic eculizumab.
Again earlier this year a researcher from the Transplant Unit at Newcastle upon Tyne Hospitals presented some data on U.K. aHUS transplants with and without eculizumab. Dr Emily Glover revealed during UK Kidney Week her findings on the 5 year survival of transplants . 5 year longevity is a key milestone in monitoring transplant success.
Emily reported that in the period 1985 to 2017 there had been 51 aHUS transplants without eculizumab.In the period 2011 to 2017 , 14 aHUS Patients got eculizumab as a rescue therapy and there were 33 prophylactic transplants. The ratio of Male to Female patients in all three categories was 1:2 and in the eculizumab era the average patient age was 43 compared with 28 in the historic group.
5 year survival in the UK’s general transplant population is 86% in deceased donor transplants and 93% in living donor. The survival rate at 5 years in the historic group was 35% , 78% in the rescued group and in the prophylactic group it was 90%.
So in terms of longevity there is significantly better outcome from employing a prophylactic eculizumab therapy strategy. Those getting it as a rescue therapy do OK but their graft is more harmed by the experience.
The two studies together show that prophylactic eculizumab in aHUS transplants results in kidney grafts working better for longer.
