Category Archives: vCJD

Sporadic CJD cases in the UK bleeding disorders community reported


A recent paper from the Centers for Disease Control and Prevention (CDC) covers the death of two people with inherited bleeding disorders from Sporadic Creutzfeldt-Jakob disease (sCJD) in 2014. Both of the people concerned were women. One had Type 3 von Willebrands and the other carried the Haemophilia B gene. These are the first deaths from sCJD in the bleeding disorders community anywhere in the world.

Most of the concern in the bleeding disorders community around CJD has been about variant Creutzfeldt-Jakob disease (vCJD). This is thought to be the human equivalent of bovine spongiform encephalopathy (BSE), known as mad cow disease.  Many of our members were informed in 2004 that they are at increased risk from vCJD for public health purposes.  This risk relates to being treated with blood products made from plasma donated by people in the UK who had eaten BSE implicated beef or went on to develop vCJD.  Although in 2008 vCJD implicated prions were found at post-mortem in the spleen of a man with severe haemophilia A who died of other causes, nobody with a bleeding disorder has ever developed vCJD.  To put that in context, a study found that 1 in 2000 people in the UK carry the implicated prion, which is the equivalent to 32,500 people.  At the moment there have been 178 cases of vCJD in the UK.

The news from the CDC related to a different form of CJD, sCJD appears to occur at random in the population at the rate of about 100 a year in the UK (fewer than 10 in Scotland). It mainly affects people of middle age and older and has been known about since the 1920s. It is a rare condition which is why there being two cases in recipients of blood products is unusual enough to trigger an investigation. It is not known whether these cases are related to being treated with plasma derived clotting factor products or whether these cases are coincidental. The CDC paper says, “Assuming an annual incidence rate of sCJD of 1.5–2.0 per million population the occurrence of 2 cases of sCJD in this total population may not imply a causal link between the treatment and the occurrence of the disease.”

There is active surveillance both in the UK and internationally for both sporadic and variant CJD. In the UK the National CJD Research and Surveillance Unit is based in Edinburgh and examines all cases of CJD in detail.  In these new cases they determined that they were sCJD (rather than vCJD ) by looking at how the disease progressed and where the CJD associated prions were found.

Prion diseases such as vCJD and sCJD are not well understood. There are still a lot of unanswered questions.  Haemophilia Scotland have highlighted the CDC paper to the Scottish Government, the Scottish Inherited Bleeding Disorders Network, and are looking into any potential legal implications.  We will be carefully monitoring the situation and will report any further developments on our website and to our members.

Further information about these cases can be found in a briefing paper produced by the European Haemophilia Consortium (ECH). There is more information about CJD on the NHS Choices website.  The best place to go with specific medical questions about your health is your Haemophilia Centre.

Last chance to have your say in our #NeedsAssessment


We are closing the needs assessment survey on Thursday morning (1st December).  We’ve had a really good response so far but the more people who take part the more useful our results will be.  We are running the needs assessment in partnership with the Scottish Inherited Bleeding Disorders Network so the results will influence the services we offer but also the services of the Scottish Haemophilia Centres.

There are some parts of Scotland that are a little under-represented at the moment.  We are keen that the results reflect everyone in Scotland.  So if you are reading this in Argyll & Bute, Dumfries & Galloway, East Renfrewshire, Orkney, Stirling, or The Western Isles please make a particular effort to take part.

If you do take part then you might also win one of three FitBits that we are giving away.  They would make a great early Christmas present for yourself or could let you cross at least one name of that Christmas shopping list!

Click here to take the survey

New study on inhibitor risks expected soon

EHC - RT - Inhibitors - Feb 2016 001

Yesterday, the European Haemophilia Consortium (EHC) held its best attended Round Table meeting yet.  Almost 80 people came from all over Europe to discuss an important study into inhibitors which is expected to be published very soon.  The SIPPET Study is the largest randomized controlled trial every conducted in haemophilia.  216 people with haemophilia have been helping the researchers investigate whether there is a difference in the chances of developing an inhibitor depending on whether someone is being treated with a plasma derived or recombinant clotting factor product.

This issue is so important because inhibitors are the most significant problem with current treatments for haemophilia.  They develop when the someones immune system reacts against the clotting factor product they have been treated with.  Having an inhibitor means more bleeds, more joint damage, and even a increased risk of death while it persists.

Approximately 30% of people who receive regular treatment for haemophilia will develop an inhibitor.  Of these roughly one third will clear it without treatment and two thirds will require treatment.  Haemophilia Centres use Immune Tolerance Induction (ITI) to try to tackle inhibitors. This involves giving relatively large amounts of treatment to train the immune system to recognize the product without reacting against it.  Even in Europe there are many countries where not everyone who needs ITI can get it.  We are fortunate that Scotland is one of the countries with unrestricted access to ITI for those who need it. ITI is successful in 60%-80% of people.  That leaves 20%-40% of people who get an inhibitor which can cause long term problems for them.

Although the SIPPET Study data has not been published the initial indications (based on the abstract which has been published) are that it will conclude that there is a higher risk of developing an inhibitor when using a recombinant product when compared with a plasma-derived product, although there is a risk with both classes of treatment. Without the relevant data, clinical organisations and patient groups haven’t yet been able to develop recommendations about how best to respond.  However, there was some speculation at the meeting that any advice that was developed might look at using plasma-derived products for people at a higher risk of developing an inhibitor for other reasons (such as genotype, ethnicity, or family history).  Most inhibitors develop between the 20th and 30th day that someone takes treatment.  Any new approach might be expected to concentrate on that higher risk period.  That means that these decisions could have the biggest impact for previously untreated people, like children or babies, and minimally treated people, such a adults with mild haemophilia.  However, until the data is available these are just educated guesses.

There are several reasons that it might be unlikely that there will be recommendations to switch, wholesale, from recombinant to plasma-derived products.  Not least of there is the concern that plasma-derived products are inherently more vulnerable to new blood borne pathogens such as viruses or prions.  This consideration is particularly acute in Scotland, and the rest of the UK, because of concerns about vCJD.  Although there are no cases of infections with a blood borne virus or vCJD from the products currently available in Scotland, nobody wants to be complacent.  It is also worth remembering that the volumes involved in recombinant treatment are lower which has advantages when treating children.

What was very encouraging was that clinician after clinician at the meeting stressed the importance of giving patients and parents the information they need to understand their choices.  There was also a strong commitment to developing any guidance or recommendations in partnership between clinicians and patients.   In the UK, the United Kingdom Haemophilia Centre Doctors’ Organisation (UKHCDO) Inhibitors Working Party will play a leading role. Here in Scotland, we will work through the new Scottish Inherited Bleeding Disorders Network to respond to changing knowledge about inhibitors and adapt any recommendations from UKHCDO, the EHC, or the World Federation of Hemophilia (WFH) for use in Scotland.  There is currently no date for the publication of the SIPPET study but we will keep you informed as soon we know more.

Find out more about the treatment of inhibitors by reading this paper.

EHC - RT - Inhibitors - Feb 2016 002




Dan Farthing-Sykes
CEO, Haemophilia Scotland

BBC Radio Scotland covers Contaminated Blood Disaster

BBC Radio Scotland's Edinburgh Studio

BBC Radio Scotland’s Edinburgh Studio

This morning BBC Radio Scotland covered the recent increase in activity in the contaminated blood campaign.

In particular, they highlighted yesterday’s Report from the All Party Group on Haemophilia and Contaminated Blood, today’s backbench debate at Westminster, and news that three campaigners have launched a legal challenge to the current financial support arrangements on the grounds of discrimination.  With a date for the publication of the Penrose Inquiry Final Report expected within the next couple of weeks, now is a critical time for raising awareness of the campaign again.

Today, BBC Radio played a report which included the moving concluding scene from the Factor 9 play by Dogstar.  Hayley Millar then interviewed prominent campaigner, Bruce Norval, in their Inverness studio.  Bruce explained how pooling of blood plasma donations in the manufacturing process increased the risk of infection from HIV and Hepatitis C, and exposed people to risks from vCJD.  He was clear that, in his view, the APPG report’s proposal amounted to “replacing five failed organisations with one failing organisation.”

The interview then turned to Dan Farthing-Sykes, representing Haemophilia Scotland, to ask about the Penrose Inquiry.  Dan said,

“The Penrose Inquiry is just the latest in a long line of Inquiries since the Scottish Parliament was established.  We’ve had Internal Inquiries, we’ve had Committee Inquiries, we’ve had an Expert Panel, and now we’ve finally got our Public Inquiry.  What we are really hoping for at Haemophilia Scotland, is that this will be the end of the Inquiries and the beginning of actually sorting this out.  As yesterday’s report from the APPG makes clear, there are all manner of unfairnesses and inadequacies in the current support arrangements.  We are really hoping that when we eventually get the Penrose Report, that will be the catalyst for really sorting this out and making sure that people, as the report made clear, are no longer living in poverty.”

We are grateful to the campaign group, Tainted Blood, for providing this clip of the BBC Radio Scotland Coverage.

More Information

The history of the campaign in the Scottish Parliament


Patient Interest Legal Team see Factor 9

Stuart, Laura Anne, Simon, Lynn, Susan, Bill and Matthew after another amazing performance of Factor 9.

Stuart, Laura-Anne, Simon, Lynn, Susan, Bill and Matthew after another amazing performance of Factor 9.

During the Penrose Inquiry the patient interest was ably represented by a small team of lawyers.  We were delighted that some of them were able to join us today to see Factor 9 at the Edinburgh Fringe.

Simon, Laura-Anne and Lynn (pictured) all worked incredibly hard to really understand how the disaster had affected people and what we want we needed the Inquiry to hear.  It was fascinating to discuss how the play highlights important elements of the contaminated blood story with them.

The play continues to get great reviews and to astound audiences. The intimate space really adds to the impact of what is already a very powerful piece of theatre.

If you would like to see it there are just 11 more performances in this run so make sure you don’t miss out and book today.

Daniel Portman from Games of Thrones backs Factor 9 at the Fringe

Daniel Portman from Games of Thrones came to see the Factor 9 play at the Edinburgh Fringe today.

In this video you can see what a powerful impact the pay has and that he is almost pleading with you to come and see it.  It really is that good.

So why not do as he suggests and get your ticket today?
Tickets for Factor 9 at the Edinburgh Fringe

The first line of the review by Yasmin Sulaiman on The List says it all, “Prepare to be physically shaken by Factor 9.”

This slideshow requires JavaScript.

MPs report on risk from vCJD

The All Party Parliamentary Group on Haemophilia and Contaminated Blood has members from the House of Commons and the House of Lords

The House of Commons Science and Technology Committee have made a detailed report in relation to the risk from vCJD

We know many of our members have been told that they are at risk for public health purposes (the at risk group) in relation to vCJD.  It is important to remember that there have been no cases of anyone contracting vCJD from a blood product. However, the prion associated to the condition can be transmitted by blood and implicated prions have been discovered at post-mortem in a man with Haemophilia who died of other causes.  With that in mind it is important that the community stays vigilant.

Today’s report from the House of Commons Science and Technology Committee is called After the storm? UK blood safety and the risk of variant Creutzfeldt-Jakob Disease and it makes some important recommendations.

The committee says that they “cannot be confident that prions are not present in the blood supply” and therefore “consider it imperative that a precautionary approach to this risk be maintained until further evidence becomes available.”

They also recognise that “Pathogens are constantly emerging and evolving; novel pathogens will therefore always pose a threat to the blood supply. In the past, it has often taken multiple cases of transfusion-transmitted infection before these threats have been recognised and mitigated. This will remain the case as long as risk mitigation measures remain pathogen-specific.”  They go on to “urge the Government to take steps to support the development of broader spectrum technologies with the potential to mitigate the risk of both known and unknown pathogens.”

To always assume that there might be a new and undiscovered threat to blood safety and to act accordingly is an important lesson from the contaminated blood disaster and it is very welcome to see an influential committee adopt this position.

The report is critical of the amount of support the Westminster Government has given to the testing and adoption of the DuPont’s prion inactivation product in relation to the decontamination of surgical instruments.  The committee also stresses the need for better access to the rare vCJD samples to promote the development of a vCJD blood test.  They also recommend that “the Government ensures that a large-scale vCJD blood prevalence study be initiated in the UK within the next 12 months.”

However, the recommendations which will probably be of most interested to people with bleeding disorders are recommendations 16 to 19 which deal specifically with people who are in the at risk group.  These recommendations call for people in the at risk group to have developments explained to them in person; for the prototype vCJD blood test to be offered to people in the at risk group; for improved monitoring of the at risk group; and for Government to be more involved in decisions about who is in the at risk group.  These recommendations are reproduced below for your ease of reference.

Read more