Category Archives: EHC

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.

New documentary tell #HaemophiliaStories from across Europe


The European Haemophilia Consortium (EHC) has been working with acclaimed director Goran Kapetanovic.  He has created a powerful documentary which reveals the variety of experiences people have of living with bleeding disorders across the continent.

His 53 minute documentary, “What is it like to live in Europe with haemophilia?” is available to all for free online on the Haemophilia Stories website.

We hope that everyone who cares about haemophilia and related bleeding disorders will enjoy the film and share it as widely as possible.

For the high quality version of the documentary and to see individual stories please go to Haemophilia Stories website.

EHC meeting calls for the eradication of Hep C for people with bleeding disorders

EHC - European Parliament - Dan

Haemophilia Scotland CEO, Dan Farthing-Sykes, on his way into the European Parliament for the EHC Round Table meeting on HCV and Haemophilia.

Yesterday was the latest of the European Haemophilia Consortium’s (EHC) fantastic round table meetings.  Held in the European Parliament the meeting gathered patients and experts from all over Europe to discuss Hepatitis C and Haemophilia.

It was clear from the discussion that Scotland’s record on treating hepatitis C is good.  In many ways Scotland is leading the way with Ireland and Portugal.

The meeting heard how the success rates of the new Direct Acting Antiviral (DAA) treatments, along with their improved tolerability and simpler administration, has made the goal of eradicating hepatitis C in people with bleeding disorders achievable.  The health economics mean that it is already a cost effective step to take.  However, it will only be achieved where clinicians and patients work together to make an effective case to Government.

As well as some extremely informative clinical presentations there were two excellent presentations from people who were infected.

You can read our full report from the meeting here.

World Haemophilia Day 2016 #WHD2016 marked by launch of Malawi Diagnosis project and awareness raising at science festivals



Yesterday (17 April) was World Haemophilia Day.  Our sister organisations all over the world were marking the day with an amazing range of activities.

Here at Haemophilia Scotland, we like to use World Haemophilia Day to raise the profile of bleeding disorders in Scotland and of the work we are doing to improve treatment and care in the rest of the world.

This year we are launching our Diagnose Malawi Appeal and are raising awareness at science festivals.

Diagnose Malawi Appeal

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We are working in partnership with the Society of Haemophilia and Allied Disorders (SHAD) in Malawi to improve diagnosis, treatment, and care in their country.

All over the world 1 in 10,000 babies is born with Haemophilia A.  However, despite having a population of well over 16 million there was nobody in Malawi with a laboratory diagnosed bleeding disorder when the project started.

We have received funding from the Scottish Government to run the Malawi Diagnosis Project with SHAD.  There will be a public awareness campaign in Malawi, and training for hospital staff, to try and find suspected cases of bleeding disorders.  New clinics will then offer testing, and where appropriate, treatment.

As part of our funding application we pledged to raise some of the money we need through community fundraising.  Money spent on this project goes a long way.  For example, running a single test will cost £15.  By the end of the project we hope that between 216 and 360 people will have been tested. Our first fundraising target of £1,500 is the equivalent of paying for the first 100 test. However, your money will be used to support the whole project.

The easiest way to donate to the appeal is on Just Giving. Alternatively, you can donate fifteen pounds by texting ‘MALA31 £15’ to 70070.  If you’d like to give a different amount just change the amount in your text. We will be posting more about the project during our ‘Malawi May’ when there will also be a cultural event to find out more about the county we are working with.

Following the success of Walk RED Malawi last year there will be another event this year.  Details are being finalised. Please put the 17th of July in your diary. If you are interested in finding out more please email Emma Black to be put on the Walk RED Malawi mailing list.

Science Festivals

To raise awareness of bleeding disorders in Scotland we have been taking part science festivals.  We worked with Blackwell’s Bookstore to run a very successful crime scene investigation event as part of the Edinburgh International Science Festival.  All day, amateur sleuths were cracking clues to solve a murder, while improving their knowledge of bleeding disorders.

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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

EHC Tender & Procurement Survey Results Published in Haemophilia Journal

Haemophilia Journal

The European Haemophilia Consortium (EHC) has announced that the scientific journal Haemophilia has published the results of their 2014 ‘Survey of coagulation factor concentrates tender and procurement procedures in 38 European countries.’

The preliminary results of the survey were presented during a World Haemophilia Day event organised by the EHC in Dublin on 16 April which Haemophilia Scotland attended.

The key conclusion of the survey and article is that,

The involvement of both clinicians and patient organizations greatly improves the outcome of a tender or procurement process, as does the presence of a legal framework that governs the process.

This significantly strengthens the calls Haemophilia Scotland has been making for Scottish patients to be involved in all tendering and procurement decisions.  We believe this is also a key lesson from the Penrose Inquiry evidence.

Today is World Haemophilia Day – building a family of support

World Haemophilia Day 2015 HS

The 17th of April every year is World Haemophilia Day.  This year’s global theme of ‘building a family of support’ is something we are strongly in favour of at Haemophilia Scotland.  Our mentoring and events programmes are all about helping the community come together in Scotland to provide mutual support.

Yesterday, we attended the European Haemophilia Consortium World Haemophilia Day event in Dublin.  We really appreciate this chance to come together as a family of patient organisations and talk about the big issues we all face – in this case the importance of patients in tendering for clotting factor products.

Today, we are delighted to have been invited to join the team at the Glasgow Haemophilia Centre for their World Haemophilia Day celebrations.  Glasgow is famed for its hospitality – as everyone who comes to the World Federation of Hemophilia Congress in 2018 will discover.  Today that hospitality means we will be enjoying a vintage tea party!

On Saturday the 25th we are holding our own World Haemophilia Day Event.  A dedicated band of fundraisers are “Walking RED Malawi” from Blantyre to Glasgow Green.  They are walking in tribute of the historic ties between Scotland and Malawi, first forged by David Livingstone who used to walk the same route to attend University.  All funds raised will support our work with the newly established patient association in Malawi.  It is not too late to sign up if you would like to take part.

New research shows the patient voice is key when buying clotting factor products

IMG_0359Today, patient representatives from all over Europe gathered in Dublin for a prestigious event to mark World Haemophilia Day.

The European Haemophilia Consortium shared exciting new data on the value of patient and clinical involvement in the procurement of clotting factor products.  A recent pan-European survey not only found that conducting a national tender lowered prices but, strikingly, that tenders where patients and clinicians took leading rolls achieved the best value of all.

The meeting also learned that the adoption of a new EU Directive on procurement (2014/24/EU) and the licensing of new, longer acting products, means that tender criteria in many countries will have to be rewritten. This provides a fantastic opportunity for countries who run tenders to formally include patients and clinicians on their tendering boards.

Clotting factor procurement is a matter for the Scottish Government.  However, for the last three tenders Scotland has purchased products as part of a UK tender.  Haemophilia Scotland is calling for Scottish patients to have a formal role in the procurement process. The Scottish Government has already announced that it will establish a National Haemophilia Committee in Scotland (to be known as the National Managed Clinical Network for Inherited Bleeding Disorders). That will give patients a voice in decisions about how Haemophilia Services in Scotland are delivered. We believe that the Scottish patient voice must be heard when procurement decisions are made as well.

The Penrose Report revealed that English patients had access to products which didn’t transmit Hepatitis C much earlier than in Scotland.  We believe that decisions like that wouldn’t happened if Scottish patients were properly involved. Putting Scottish patients at the heart of the procurement process is an important lesson from the findings of the Penrose Inquiry.

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EHC meeting asks if there anything we can do to reduce inhibitor risk

The EHC run very professional meetings

The EHC run very professional meetings

The European Haemophilia Consortium (EHC) have organised another one of there excellent Round Table meetings. These events give patient representatives an invaluable chance to hear from some of the leading clinicians on a key issue facing people with bleeding disorders in Europe.

The March 2015 meeting tackled the issue of inhibitors in Haemophilia A. If your immune system responds to a clotting factor product and stops it working it is called an inhibitor. About 30% of people who regularly take clotting factor products will develop an inhibitor.

A lot of the discussion was about whether different types of products or treatment regimes had any impact on the risk of developing an inhibitor – especially when someone hasn’t had treatment before. Previously Untreated Patients are often abbreviated to PUPs in these discussions.

Recombinant or Plasma Derived Products

We heard that the largest retrospective study, the Rodin Cohort Study, does not show a conclusivly whether there is a difference in inhibitor risk between plasma derived or recombinant products.

Therefore, a randomised clinical trial, known as the SIPPET Study, has been started. SIPPET has looked at an effective sample of 270 people with Haemophilia from all over the world. It found a 29% of people developed an inhibitor. It is testing the idea that plasma derived products might be half as likely to cause an inhibitor. However, the study is still ongoing and hasn’t produced an answer yet.

Second or Third Generation Products

The Rodin Study indicated that there might be an increased inhibitor risk from second generation products with a full length molecule.  Studies have been conducted in France, the UK, and Canada to try and confirm the finding. The combined studies showed a statistically significant increased risk for previously untreated patients who were on Kogenate. However, this data needs further checking for potential bias so the results are being treated with a lot of caution. Haemophilia Scotland were informed about this issue at the annual meeting with the Scottish Haemophilia Centres and assured that Kogenate is not and will not be used for previously untreated patients in Scotland. At the moment there isn’t a clear explanation for why there might be an increased risk. One of these studies also suggested a risk associated to ReFacto AF but the low numbers of people on the product means that these figures aren’t secure and need further investigation.

European Monitoring

The EUHASS project looks at the question of inhibitors a different way. It is a Europe wide adverse events surveillance system. The Centres which talk part regularly report on the number of inhibitors and the number of patients reaching their 50th treatment (as an indication of how many people are at risk of an inhibitor). They found a slightly lower rate of inhibitors at about 26%.

There was a suggestion that it would take a trial which randomly assigned products to previously untreated patients to give stronger results. However, concerns were raised about whether this would be possible while still giving patients fully informed choice.

Patients and Patient Organisations

Thomas Sannie, from the Association Francaise des Hemophilies (AFH) argued that patients are vital to surveillance after market authorisation of any product. This requires engaged and well informed patients who are prepared to make reports. He described the work of the AFH on a project that developed tools to help make reporting adverse events, including inhibitors, part of the culture of the bleeding disorders community in France. This includes a new website which guides patients through the process.

In Scotland the YellowCard Centre Scotland accepts reports of adverse events and side effects from patients and healthcare professionals. However, the system could certainly learn from how the French project has made their equivalent much more user friendly.

You can find out more about this meeting on the EHC website.

Thomas Sannie explains how patient reporting has been improved in France.

Thomas Sannie explains how patient reporting has been improved in France.

Now is a great time to talk to supportive MSPs

Motherwell was looking grand in the sunshine, there was even a band playing in Merry Street.

Motherwell was looking grand in the sunshine, there was even a band playing in Merry Street.

Now that the Scottish Parliament is in recess the MSPs have returned to their constituencies and regions to deal with local issues and prepared for the referendum.  That means that now is a great chance to meet your local MSPs without having to travel to the Scottish Parliament.

Today I was in Motherwell seeing Richard Lyle MSP who is a great friend of Haemophilia Scotland as well as been a member of the Scottish Parliament’s Health and Sport Committee.

We discussed how to keep his colleagues interested and informed in the run up to the publication of the Penrose Report in the Autumn.  I also updated him on our campaign for an open market for longer acting clotting factor products. As usual Richard was extremely helpful and full of useful advice and ideas.

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