As many of you will know, Crizanlizumab (Adakveo) was recommended as a new disease modifying treatment for sickle cell anaemia by National Institute of Clinical Excellence (NICE) and NHS England on a Managed Access Agreement. This means that the treatment was recommended to eligible patients under an agreement to collect more data about it.

On the 26^th May 2023, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) made a recommendation to revoke the conditional marketing authorization for crizanlizumab, a once-a-month, humanized anti-P-selectin monoclonal antibody infusion indicated for the prevention of recurrent vaso‑occlusive crises (pain crises) in sickle cell disease patients aged 16 years and older.

Findings from a recent study did not confirm that there was a clinical benefit of the drug in successfully reducing the number of painful crises requiring a healthcare visit or treatment at home in patients with sickle cell disease and so the benefits do not outweigh the risks.

John James, CEO of Sickle Cell Society said

“Whilst the recommendation of the European Medicines Agency has been made, the decision of the Medicines Healthcare products Regulatory Agency (MHRA) for England is still awaited.

We would like to reassure UK patients who are currently taking Crizanlizumab (Adakveo) as part of the Managed Access scheme, that it is safe. However, if you have any questions, we recommend that you speak to your Consultant Haematologist who will determine the best options for each patient depending on their individual circumstances.”

The CHMP’s recommendation will be provided to the European Commission (EC) and a final decision will be made by the EC in the next two months.

As soon as we know the decision of the MHRA for England, we will update you again.

The post SCS response to European Medicines Agency (EMA) and Committee for Medicinal Products for Human Use (CHMP) updates on Crizanlizumab appeared first on Sickle Cell Society.

The purpose of this study is to explore the safety, tolerability and effectiveness of a study drug called VIT-2763 for sickle cell disease (SCD). Specifically, we want to investigate its effects on the breakdown of red blood cells and inflammation associated with sickle cell disease.

VIT-2763 targets and blocks a protein in the body called ferroportin. Ferroportin allows the body to transport iron to the blood stream. Iron is important in the production of haemoglobin, the protein that transports oxygen in red blood cells. It is expected that, by blocking ferroportin, VIT-2763 may lower the iron level in the blood. This might reduce the concentration of abnormal haemoglobin (sickle cells) in the red blood cells, and it may reduce the destruction of red blood cells caused by SCD.

VIT-2763 is being developed by Vifor International Inc., the sponsor of this study. It has been tested before in healthy volunteers at multiple dose levels and was well-tolerated by study participants. VIT-2763 is also being tested in people with another blood disorder called thalassemia.

To take part, you must

• Be 18 to 60 years of
• Have SCD, including HbS/S or HbS/βT0 genotype (but NOT HbS/βT+ genotype or HbSC disease). The study doctor can tell you what kind of SCD you have if you are not sure.
• Have had 1-10 vaso-occlusive crises within 12 months prior to study screening. These are also called ‘sickle cell pain crises’ or ‘VOC episodes’.
• Not have chronic liver disease or a history of liver Other criteria will also be reviewed to see if you can take part.

The study

-The expected duration is max 16 weeks (up to 4 weeks screening period, 8 weeks of treatment and 4 weeks safety follow-up)

-The study involves 7 outpatient visits.

-Eligible participants may receive compensation for their participation in the study, plus reasonable travel expenses.

-24 participants will take part in the study

More Information

You can also Download a PDF of the Clinical Trial Leaflet Here which gives full information on the trial.

If you are interested in finding out more, or participating in the study then please contact your nearest Clinical Trial Location.

Clinical trial locations

-Barts Health NHS Trust – Please Contact: Tasnima Ferdousi  Phone: 020 324 60261

-King´s College hospital NHS foundation Trust– Please Contact: Katia Mercone, Phone: 020 329 95772

-Homerton University Hospital NHS Foundation Trust – Please Contact: Jagrul Miah, Phone: 020 851 05117

-Royal Liverpool University Hospital – Please Contact: Samuel Badu,  Phone: 015 170 63397

-Manchester University NHS Foundation Trust – Please contact: Charlene Hyde, email: NMH.Trials@mft.nhs.uk

-Imperial College healthcare NHS Trust – Please Contact: Camelia Vladescu, Phone: 020 331 31195

The post CSL Vifor VIT-2763 Clinical Trial in SCD appeared first on Sickle Cell Society.

Wearing her House of Lords Robes, Baroness Benjamin will be carrying the magnificent Sovereign’s Sceptre with Dove, traditionally known as the Rod of Equity and Mercy”, which is presented to the King during his investiture before being crowned sovereign to represent his spiritual role.

Dame Benjamin said, “I feel honoured and privileged to be part of the historic coronation ceremony. To be selected to carry the Sovereign’s sceptre with dove, which represents spirituality, equity and mercy, is for me very symbolic as it’s everything I stand for and sends out a clear message that diversity and inclusion is being embraced.”

John James, CEO of Sickle Cell Society, said, “We are so proud that our Patron Baroness Benjamin has been selected for this very special role, representing the Windrush Generation and the diverse nation which we have become. All of us at the Sickle Cell Society send her our congratulations on this special role and our very best wishes for the day.”

Another of our Patrons, Prof Dame Elizabeth Anionwu, recently appointed to the Order of Merit, has also been selected to play a key role in the British Coronation Ceremony as she will carry the Orb during the coronation Ceremony of His Majesty King Charles the Third in the proceedings.

The Sovereign’s Orb, representing the monarch’s power and God’s granting of that power to the monarch, is a round hollow sphere of gold adorned with a cross.

The Orb was designed by the royal goldsmith, Sir Robert Viner, for the Coronation of King Charles II. In 1671, Colonel Thomas Blood attempted to steal the Sovereign’s Orb along with the other Crown Jewels.

During the Coronation, the Archbishop of Canterbury places the Orb in the monarch’s right hand; then, the Orb is placed on the altar before the monarch is crowned.

In 1979, Anionwu became the United Kingdom’s first sickle-cell and thalassemia nurse specialist, helping establish the Brent Sickle Cell and Thalassaemia Counselling Centre with consultant haematologist Milica Brozovic. In 1998, by then a Professor of Nursing, Anionwu created the Mary Seacole Centre for Nursing Practice at the University of West London.

She holds the Order of Merit, was appointed a Dame Commander of the Order of the British Empire and is a Fellow of the Royal College of Nursing (RCN). She retired in 2007, and in 2016 she published her memoirs, Mixed Blessings from a Cambridge Union.

John James, CEO of Sickle Cell Society, said, “Congratulations go to our Patron, Prof Dame Elizabeth Anionwu, for the part she will play in the Coronation of King Charles III. All of us at the Sickle Cell Society are so proud that a Sickle Cell and Thalassaemia specialist nurse has been included in the ceremony, and we look forward to supporting her in representing the Sickle Cell community during the Coronation.”

The Rt Revd Rose Hudson-Wilkin will present Queen Camilla with The Queen Consort’s Rod.

In a statement released by the Palace, Hudson-Wilkin said, “I am surprised, excited and honoured to have been asked to play a part in this historic once-in-a-lifetime occasion. As I make my presentation, both their Majesties will remain in my prayers as they seek to serve the nation and the Commonwealth.”

John James, CEO of Sickle Cell Society said, “This is the first time that female Bishops will play a part in a Coronation service, so we are absolutely delighted that one of the female Bishop’s selected is a woman of colour, recognising the diverse population of the UK and also a Patron of the Sickle Cell Society. We send her our congratulations and our support on the day.”

We are so proud of all of our Patrons for the role they are playing in today’s proceedings. We send them our very best wishes. As a Society, we send our congratulations to His Majesty King Charles the Third and Camilla, The Queen Consort.

The post Congratulations to our Patrons participating in the King’s Coronation! appeared first on Sickle Cell Society.

We would encourage anyone who is interested in finding out more about the study and Sickle Cell to attend.

The Redress Study is also running a Poetry Competition, open until the 14th May, with a £50 Amazon voucher up for grabs for the best poem.

Register for the event here.

Download a PDF with info via the following links:

Event Programme

Poetry Competition

The post Redress Study Launch Event – Saturday 20th May appeared first on Sickle Cell Society.

The post It’s in our Genes appeared first on Sickle Cell Society.

About The PM Society Awards

The PM Society Awards has been running for 37 years, it is an event in the healthcare calendar that showcases the best creative work the industry has to offer. It is the longest running and largest awards event in the industry calendar, taking place annually in March and recognizing creativity, innovation and impact across a range of creative communications work.

The post Code Red Awareness Campaign Shortlisted in the PM Society Awards appeared first on Sickle Cell Society.

We celebrated Valentine’s Day about five weeks ago. Did you know it is reportedly the most celebrated day around the world besides New Year?  Saint Valentine’s Day is apparently named after a saint called Valentinus who it is said was imprisoned for performing weddings on soldiers forbidden to marry and for ministering to Christians who were persecuted under the Roman Empire.  Legend has it that he healed the daughter of his jailer and before his execution he wrote “from your Valentine” as a farewell to her!  By the 15^th Century, 14 February had become associated with romance and the tradition of courtly love and had pretty much evolved to what happens now – an occasion when couples (current or would-be!) express their love by presenting each other with flowers, chocolates, romantic gestures and cards known as ‘valentines’.

Why Care about Type?

From the type of people we might have been attracted to this past Valentine’s Day to the way we look and behave, there’s a lot we inherit from our parents through genes, including our haemoglobin genotype.  This tells you the two genes (i.e. codes) – one inherited from each parent – that determine your type of blood haemoglobin.  Haemoglobin is the substance in your blood that gives blood its red colour and carries oxygen around your body.  The type of haemoglobin genes you inherit or pass on can play an important role in determining whether you or your children are affected by two serious inherited blood conditions – sickle cell disease and thalassaemia.

Sickle cell disease is a serious inherited blood condition that can cause severe pain, anaemia and organ damage.  It mainly affects people who originate from Africa, the Caribbean, Asia, the Middle East and the Mediterranean.  However, sickle cell is not a ‘Black’ disease and can affect ‘White’ people too, though less frequently.  The reason sickle cell is more common amongst the ethnicities described above is because the sickle cell gene was a mutation to protect against malaria and being a ‘carrier’ of one copy of the gene (also known as being ‘trait’) offered some protection.  People with the trait survived malaria and could have children with other survivors, thus making sickle cell prevalent in areas that had malaria.  Thus, it is not a condition people should feel stigma about.  Also, please remember trait or not, everyone should protect themselves when in malaria areas of the world!

Out of the 15 million people estimated to have sickle cell disease worldwide, around 10 million live in Africa of which approximately 4 million are in Nigeria.  In the UK, an estimated 15,000 people have sickle cell disease and in 2019/20,  262 new babies were born with sickle cell and 8247 were ‘carriers’ or ‘trait’ (Data from NHS Sickle Cell and Thalassaemia Screening Programme Data Report 2019/20).

Thalassaemia is a condition most common among people originating from India, Pakistan, Bangladesh, Cyprus and China.  People can inherit Beta Thalassaemia major which affects their ability to produce enough red blood cells. This causes severe anaemia and organ damage and they need to be on regular blood transfusions throughout life.

The usual and most common type of haemoglobin gene people inherit is Haemoglobin A. Unusual haemoglobin genes include Haemoglobin S (known as ‘sickle haemoglobin’), Haemoglobin C and beta thalassaemia.   People can only get sickle cell or thalassaemia if they inherit two unusual genes for haemoglobin, one from each parent.  In the UK 1 in 4 West Africans are sickle cell ‘trait’.

Post Valentine’s be the perfect partner!

On 14 February 2023, many people will have established new relationships or progressed further with old ones.  Particularly for young couples who have not yet had children, now might be the right time to raise awareness of these two inherited blood conditions so individuals can consider finding out their ‘haemoglobin genotype’, as each time two people with trait have a baby there is a 25% chance the baby could be born with sickle cell disease or thalassaemia.  A simple blood test will determine if you carry a gene for sickle cell or thalassaemia and tell you your haemoglobin genotype.  For someone with sickle cell anaemia this will be ‘SS’ and for someone with sickle cell trait this is ‘AS’.  However, if people inherit the other unusual haemoglobin genes they will have other types of sickle cell disease apart from sickle cell anaemia, such as ‘SC Disease’ (common amongst Ghanaians) and ‘sickle-beta thalassaemia’.

There is an NHS Sickle Cell and Thalassaema (antenatal and newborn) Screening Programme which offers all pregnant women a screening blood test which is then offered to the father-to-be if the woman is found to be a carrier. The Programme also screens newborn babies for sickle cell.  In England only 60% of men are currently accepting their invitation for sickle cell screening and so more men need to step up!  If the father-to-be also carries the sickle cell gene then the ‘at-risk’ couple is given all the information that enables them to make an informed choice about the pregnancy.  If you are already a couple and you know that you both carry one of the genes for sickle cell or thalassaemia  then you should present to your GP early in your pregnancy or contact maternity services or your nearest NHS Sickle Cell and Thalassaemia specialist counselling services directly. You should also tell healthcare professionals if you want counselling and prenatal diagnosis (tests to see if the unborn baby has the condition) and not assume that all healthcare professionals will know what you want!

Preconception Testing

Testing for your haemoglobin genotype before pregnancy is called ‘preconception testing’. You can ask your GP for this blood test before you and your partner decide to start a family and especially if you know other family members who are carriers or who have sickle cell. You and your ‘Valentine’ can then discuss any risks and the choices that are right for you!  Recent discussions the Sickle Cell Society has had with students and young adults who have not yet had children indicate that there is now more awareness of sickle cell and that many are keen to find out their genotype before starting relationships. They are also keen to know options available to ‘at-risk’ couples. The Sickle Cell Society and UK Thalassaemia Society do a lot of outreach to educate the general public about sickle cell and thalassaemia  as more awareness will also help to remove the stigma associated with these two conditions.   The Societies are also planning to do even more preconception outreach so those planning children will be well informed.

Now the valentine cards, chocolates and flowers are over why not learn more about sickle cell disease and thalassaemia so you can make informed choices (about testing) if and when the need arises?  By so doing, you just might be considered that perfect partner after all!

Below are websites with lots of information to help you:

Sickle Cell Society

www.sicklecellsociety.org

Email: info@sicklecellsociety.org

UK Thalassaemia Society

www.ukts.org

Email: office@ukts.org

The post This Valentine’s Did You Find Your Type? appeared first on Sickle Cell Society.

You can download the leaflet here: Read the Leaflet Here

The post Living with Sickle Cell or Beta Thallasaemia Trait Leaflet appeared first on Sickle Cell Society.

The post Rare Disease Day 2023 appeared first on Sickle Cell Society.

We have now been informed that preliminary findings from the ongoing worldwide clinical study called STAND (NCT038147460) indicates no statistically significant difference between Crizanlizumab 5mg/kg Crizanlizumab7.5mg/kg and placebo in making a difference to a sickle cell crisis. However, these findings are inconsistent with previous trial results from the SUSTAIN (NCT01895361) trial. As a result of this regulatory bodies such as the European Medicines Agency (EMA) will undertake a review of Crizanlizumab to evaluate the impact of these contrasting results on its currently authorised use.

We would like to reassure those who are taking Crizanlizumab , or who may be contemplating taking it, following discussions with their Health Care Professional, that it is safe. However, if you have any concerns, we recommend that you speak to your Consultant Haematologist in the first instance who will determine the best option for each patient depending on their individual situation.

We will provide more information when we have it.

The post SCS response to European Medicines Agency (EMA) and Novartis updates on Crizlanlizumab appeared first on Sickle Cell Society.