Category Archives: Research

What research question would do most to #StopTheBleeding?

Stop the bleeding

A Bleeding Disorders priority setting partnership (PSP) has been formed in conjunction with The James Lind Alliance and they are asking for your help.

The PSP is compiling a list of the 10 most important unanswered questions about bleeding disorders.  Once finished, the list will be used to promote research and attract funding.  It’s is very important that research goes into areas which have an impact on the lives of individuals and families with inherited bleeding disorders.  That is why the PSP want to encourage those who live with the conditions to feed in their ideas and propose research questions.

All you need to do to take part is complete a short online survey.

Stop The Bleeding Survey

The JLA is a non-profit making initiative which is a branch of the National Institute for Health Research (NIHR). The experience of the JLA with conducting PSPs is helping us shape and organise this project. You can find details about the JLA and other PSPs here.

Haemophilia Scotland is not a member of the PSP Steering Group but we are supportive of their objectives.

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

European Medicines Agency investigation of Direct Acting Antivirals

European Medicines Agency - EMA

Whenever a new product is adopted there is a possibility that a the larger numbers patients with different histories and settings will reveal effects that weren’t apparent during the clinical trials.  That is why post marketing monitoring and initiatives such as the Yellow Card scheme are so important.

The new Direct Acting Antiviral treatments for hepatitis C have been very successful in helping many Haemophilia Scotland members achieve Sustained Virological Responses (SVR).  Many of our members had a history of unsuccessful and grueling treatment involving interferon. So far, members have reported some, comparatively mild, side-effects but overall have responded well to the new treatments.  Nevertheless, it is important to remain vigilant.

Globally, there have been some anecdotal reports of hepatitis B re-activation in some patients who have been infected with both hepatitis B and C viruses and who were treated with direct-acting antivirals for hepatitis C. Hepatitis B re-activation is the return of active infection in a patient whose hepatitis B infection had been chronic.

In April 2016, a study was published regarding the risk of liver cancer (hepatocellular carcinoma) returning in patients who were treated with these direct-acting antivirals for hepatitis C. The study suggests that some patients were at risk of their cancer coming back earlier than in those patients with hepatitis C who were not treated with direct-acting antivirals.

To investigate these reports the European Medicines Agency (EMA) began a review in March 2016.  The current status of their review is “Under Evaluation”.  However, their website does provide a summary, key facts, and all documents for those who would like to know more. The scope of the EMA review extends to assessing the risk of liver cancer with these medicines.

Commenting on their review the EMA states that, “While the review is ongoing, patients should speak to their doctor or pharmacist if they have any questions or concerns.”

Sources include Haemophilia World.

UKHCDO respond to SIPPET study into inhibitor risk


Dr Dan Hart is the Chair of the UKHCDO Working Group on Inhibitors.  He is pictured here explaining inhibitor risk to the Haemophilia Scotland Gathering and Conference in 2016.

The United Kingdom Haemophilia Centre Doctors’ Organisation (UKHCDO) has released a statement in response to the SIPPET Study.

The SIPPET Study is a randomized clinical trial which looked at inhibitor risk related to class of clotting factor product.  It has involved 77 centres in 19 countries across 5 continents. The study isn’t specific to individual products but compares recombinant products as a class to plasma derived products as a class. Inhibitor development is the most challenging complication of current haemophilia treatment.

The UKHCDO statement gives the SIPPET findings as,

Those treated with recombinant FVIII concentrates had a 44.5% rate of all inhibitors compared to 26.8% in plasma-derived concentrates with the “high-titre” rates being 28.4% in recombinant versus 18.6% in plasma-derived products.

The statement points out that these risks are higher than the findings of a study that the UKHCDO published in 2015 in previously untreated people using recombinant Factor VIII.

This UK study identified an overall inhibitor rate of 29% with the important subgroup of those with a “high-titre” inhibitor being 14.7%.

The UKHCDO statement concludes by outlining what effect this new information will have on the practice in UK Haemophilia Centres.

UK clinicians should counsel parents about the implications of known inhibitor studies if the presenting clinical scenario allows. Recombinant FVIII concentrates remain an acceptable standard of care for PUPs, with plasma-derived concentrates considered on an individualized basis.

The statement refers to parents because these considerations are most relevant to patients who have had less than 30 days of exposure to clotting factor products.  In most cases these people are children but it will also include some people with mild or moderate bleeding disorders.  This new advice will apply to Haemophilia Centres in Scotland unless the Scottish Inherited Bleeding Disorders Network recommend that any variations are appropriate in due course.

Anyone who would like to discuss their treatment, or the treatment of their child, in the light of today’s statement should talk to their Haemophilia Centre as usual.

You can read the UKHCDO statement in response to the SIPPET Study here.

Dr Dan Hart has high hopes for new haemophilia treatments


Regular readers will remember that Dr Dan Hart kindly came to our Gathering Conference this year in Dunblane to speak about inhibitor risks.

Part of the feedback from the event was that you’d like to hear more about upcoming developments in haemophilia treatment.

Media Planet have just posted an article by Dan on their Health Awareness site in which he talks about his hope for the new haemophilia treatments, including Extended Half-Life (EHL) products.

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

The Pharmaceutical Industry wants your views

The survey should take between 10 and 15 minutes

The survey should take between 10 and 15 minutes

The Association of the British Pharmaceutical Industry (ABPI) is the industry body for research-based bio-pharmaceutical companies.  The ABPI issue guidelines that, amongst other things, govern what contact pharmaceutical companies can have with patients and their families.  This means it is particularly important that they understand bleeding disorders.

They have commissioned an independent researcher to find out more about what patients and carers think of the treatments for rare diseases that their members develop.  All bleeding disorders are rare but the survey will be particularly interesting for people with rarer factor deficiencies or platelet disorders.

The results of this survey will be published and use to raise the profile of rare diseases and orphan medicines.

There are two surveys to choose from;

The survey’s close on the 25th of April.

Interferon-free hepatitis C treatments now being developed

New treatments continue to be developed to combat the scourge of Hepatitis C Virus (HCV), which is estimated to have infected about 95% of UK haemophiliacs receiving blood products up to the mid-1980s, when heat-treated and later ‘recombinant’ clotting factors became available; virtually eliminating the risk today of contracting hepatitis through injection of anti-haemophiliac clotting products.


The Hepatitis C Trust is an excellent source of information on the latest developments in hepatitis C treatments.

The latest step forward, which has been widely reported in medical literature and online media, is an experimental drug duo that in clinical trials on 362 people with chronic hepatitis C indicated a 69% success rate in clearing hepatitis C, without causing the severe side-effects often associated with current standard interferon based therapy.

The German led research indicated that two new drugs – faldaprevir and deleobuvir – when taken with ribavirin provided an effective treatment for hepatitis C. Significantly, this was achieved without the need to inject interferon; part of the current therapy that has proven difficult for many patients to tolerate. Several other pharmaceutical companies are also actively pursuing the development of interferon-free drug combinations for the treatment of hepatitis C.

Eliminating injected interferon from treatment regimens is a highly desirable goal in hepatitis C management, which makes this news very welcome. However, it should be stressed that the trial results varied across genotypes, with genotype-1b patients being particularly responsive.

More information on the new treatments can be found on the Heptitis C Trust website:

Scoping Exercise into Living with HCV Contaminated Blood

At the Haemophilia Scotland event in the Scottish Parliament last April, Alex Neil MSP, Cabinet Secretary for Health and Wellbeing, announced that there is to be a scoping exercise looking at what it is like for people in Scotland who live with Hepatitis C.
The study will not only focus on health issues such as the side-effects of treatment for Hep C,but also more general factors like financial consequences (for example, insurance), and other areas of life that might be affected including: relationships, social stigma, employment and access to welfare benefits or other supports.

The project is being managed by the Scottish Infected Blood Forum on behalf of the Scottish Government. Haemophilia Scotland is fully involved by having a place on the Project Reference Group.

This group allows for expert advice and influence on the way the scoping exercise progresses. A questionnaire is being developed which will be available soon. It is hoped that as many people as possible who are infected or affected by Hep C will take part.
While not wishing to raise any false expectations in these times of austerity, the results of the scoping exercise will be used by the Scottish Government to guide its thinking and plans for making provision to meet the needs of Scottish citizens affected by this serious viral infection.

When the questionnaire is available, Haemophilia Scotland will do its part to promote the scoping exercise and encourage people to have their say so watch this space.