aHUS community answers the call

Article No 384

4 October 2020

On the 3 August 2020 , the aHUS alliance Global Action sent out a call to the aHUS community appealing for help from patients/carers with experience of both eculizumab and ravulizumab treatment..
Volunteers  were needed to participate in a Global Action research study on the comparative impact on patients from using those two technologies. Some wrote about the experience and others talked about it too. There was an enthusiasm  to communicate what had happened and what mattered  to them.
What they wrote and said was copied to a summary page and then analysed. The comments were structured by transition process, infusion process ,efficacy, side effects , work school etc, cost and  “other issues” along with an overall view. The differences and similarities identified were then summarised.  Finally Global Action extracted quotes from participants which demonstrated the collective point made.
Eventually a report on the study emerged. With the  title ”


A copy of the report can be seen using this link Ecu and Ravu experience
The collective evidence, given by those who know what each treatment is like ,provides an authentic and authoritative case to  healthcare decision-makers that :

“ From the evidence provided by those with experience of both eculizumab and ravulizumab treatments patients see ravulizumab as a positive and progressive step change to their treatment. Although not perfect yet, it has much to commend it and is welcomed by aHUS patients   “

“Not perfect” because  some are still or even more affected by the side effects which they experienced with eculizumab infusions.
Although not raised by those in the study there  are  still some answers needed on Ravulizumab‘s suitability for short term “de novo” aHUS use . Also for pregnancy aHUS treatment , which has been OK’d for eculizumab use. This is because the process by which Ravulizumab is recycled to provide longer efficacy involves an agent, the  neonatal fc receptor ( FcRn) , which also plays a part maternal immunity transfer. Hence the jury is out.
The Global Action research report is both unique and “a first”. It is a unique study because it is research done by aHUS patients/carers about aHUS patient/carers for aHUS patients/carers. And a first because  it’s the first time the Global Action made such a call for help with research and got such a resounding response.
Most aHUS research is done by clinicians and pharma. Some such research these days cite the research that matters to aHUS patients/ carers which makes up their Global aHUS Research Agenda.
This study takes “patients included” one step further and has been successful. Maybe even more can be done. Global Action found talking to the volunteers in this way a valuable experience.
The aHUS alliance Global Action would like to thank all those who volunteered and participated in the research. What they have done by answering the call will make a difference for others.
For a new therapy to be approved it much be at least as, if not more, clinically effective than the alternative . It helps too if it is more cost effective. Finally it should something that is welcomed by the patients who need it for as long as they need it. aHUS patients say they welcome ravulizumab for a freer and easier life.

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