Article No. 406
22 December 2020
As the year is drawing to a close and the holidays are fast approaching, it is not unheard of for aHUS Global Action to reflect on the content of its website during the past year.
Less reflection needed this year because of the introduction of its newsletter The Global Advocate which brings viewers’ attention to news about aHUS. ( Subscribe HERE if you have not already done so).
There have been 98 news articles this year ( 99 when this is published) and the number of news blogs published since we started has passed 400. Probably, this website is not surpassed for wide ranging aHUS content.
But what has most interested those in the aHUS community in 2020?
Surprisingly among the most viewed articles is a blog from 2018, Qu’est-ce que le syndrome haemolitic et uremiue atypique ( SHUa )? As this is a global website, articles in languages other than English have been encouraged. The French article, popular when first published, had seen viewings grow in 2019 and then more so in the last 12 months. Probably “Monsieur Google’s” search engine has found it often for a “SHUa” search.
Other language articles have featured during the year, e.g. on Germany and Norway. More are welcome.
Another article from the past , from 2017 in fact, continues to be found by Google, probably because it is a frequently asked question on the internet i.e. Can the price of eculizumab be justified? It was written for the 2017 Rare Disease Day video follow up.
Perhaps unsurprisingly the other well read articles this year are about COVID19 and its link to aHUS.
The website had just featured Rare Disease Day and had a series of articles with a “Spotlight on Ravulizumab” and aHUS Genetics when COVID19 began to permeate.
The first COVID19 article was in our “Trials Watch” series. aHUS Trials Watch 10 was written because of an amusing incident at a Complement conference in Toronto.
Written on the 8 March it was about a New York clinician who had created a trial to use eculizumab to treat COVID19. In it aHUS Global Action first speculated on whether COVID 19 was a trigger for an Infection TMA.
The next article about it linked rare disease to COVID19 and the different attitude by society to the latter when the worst of that disease outcome was similar to aHUS.
It was later said by experts that COVID19 and aHUS were like parallel diseases. But what a difference in awareness.
It makes you think but there is more and more talk about “Long COVID”. Is that any different to what aHUS patients feel long after their aHUS has abated. “Long aHUS”!? They “get it” when they can get it was an article which drew comparisons in society’s approach towards the common and rare.
Another early article about COVID and aHUS brought out the likely issues of concern for patients. Simply titled aHUS and COVID 19 included some clinicians’ early thoughts and advice on COVID and aHUS.
Another page, rather than a news blog, with a similar title is aHUS and COVID19 ,which provided a diary of events and much more information during the year.
Although articles about research featured regularly during the year, aHUS Global Action also did more of its own research. Our research was published in website articles.
The first which should be mentioned is the most comprehensive search of literature to catalogue drug technologies with a potential relevance to aHUS. Researched over several weeks by author Linda Burke, the status of complement inhibitor drugs in 2020 can be see in the article 2020 Atypical Drug Discovery Review, Released by the aHUS alliance
How are you doing- Patient Reported Outcomes was a four part article about self reported outcomes by patients in the Alexion Global aHUS Registry. Using data asked for , and approved by the Scientific Advisory Board of the registry about patient reports completion compliance, the article showed how compliance had deteriorated over the five years from the commencement of the Registry.
Most Rare diseases do not have registries, so it is disappointing to find that valuable data is not being captured when a Registry exists. Whilst there may be good intentions at the beginning, as time goes by there is a drop off in enthusiasm . Recommendations were fedback to Alexion about improving patient reported outcomes capture performance in its Registry from a patient perspective but nothing has been heard since.
Access to eculizumab is an important issue to aHUS patients and aHUS Global Action set out to find what had been the access experience in the world outside of USA and Europe. The results were published in the article Access to Eculizumab.
The current status in 12 countries was reported along with information about the enablers and barriers which had been identified.
aHUS Global Action delved into the estimated prevalence levels of aHUS, focusing on the USA patient population. Using some published information about aHUS eculizumab treatment levels in 2011-2016 in a database of patients, representing 20% of the USA population, Global Action re estimated the number of aHUS patients in the USA.
No one knows the exact number but there is a strong case to think that there are in excess of 2000. Certainly substantially more than the outdated figures quoted using a 2010 prevalence rate of 2 patients per million, that is just over 600 patients. The article USA aHUS Patients – 2000 or more and rising really challenged the status quo, but surprisingly did not seem to make so much of a reaction.
Alexion will know how many aHUS patients are having, or have had, eculizumab treatment. In 2019 Alexion sold $2 billion worth of eculizumab in the USA alone for all conditions it benefits, as well as as for aHUS. Even at the highest list price that would mean that there were at least 4000 eculizumab patients in the USA.
It would not be unreasonable to say half of them were aHUS patients. 2000 still means it is very rare but there are more and more surviving each year.
A key bit of research involved those who had experience of both eculizumab and Ravulizumab. The results of written feedback from and interviews with a representative group of patients were captured in a report and conclusions drawn from what patients and carers said . The article aHUS Community Answers the Call includes that report.
The year finished with another aHUS Global Action research project in progress about the aHUS Diagnosis Process. Again the aHUS community is rallying to the call which was made in the article Help needed to join the aHUS dots
The survey questionnaire will be open until 19 January 2021. If you have not participated yet, the survey form can be accessed in the article.
So aHUS Global Action will be hitting the ground running in 2021 with a key research project enabled by the support from the aHUS community.
But first a break for the holiday season. Wishing all a restful time and health and well being in the New Year.