A Hull teenager with severe haemophilia has become the first person in the country to be treated with a new drug, which is much easier to administer.
The patient commenced his treatment with Marstacimab at Hull University Teaching Hospitals NHS Trust – making us the first Trust in the UK to use this drug – and the results have been very successful.
Haemophilia is a rare condition that affects the way blood clots, meaning those who have it bleed more easily than other people. It cannot usually be cured but treatment can help control the symptoms. Marstacimab is a weekly injection that targets a protein in the blood clotting process, and helps to prevent or reduce bleeding in patients with haemophilia A or B.
Haematology consultant, Dr Jireh Ann Batac has been instrumental in securing the use of this drug in our hospitals. It’s currently funded by the NHS to treat patients with severe haemophilia B. The main benefit is the way its administered, causing less discomfort for the recipient and making the process much easier.
She said: “This patient has severe haemophilia B, severe autism and development delay. Before this drug, the treatment was always intravenous (IV). This medication means they don’t have to inject using IV. That means his parents have been able to administer the injections at home, and there’s been no difficulty doing this or missed doses. This drug wasn’t previously available in the UK, so it’s great to be the first Trust to be able to offer this.”
The patient’s family said: “Marstacimab has enabled my severely-disabled child to gain access for treatment which was otherwise challenging. It truly was a game-changer for us. To be able to administer a treatment that is more pleasant for our son is something we are grateful for.”
Dr Batac attended a conference in Washington DC last year to present on this complex patient case. She had to do a verbal and poster presentation in front of a packed room.
She added: “Everyone was very engaged with the discussion and it was attended by world experts. They were all interested to hear about the case.”
We were highlighted in the launch of Marstacimab in the UK to share our experience, as we started using the drug before it was officially launched nationally. Dr Batac has also been invited to speak in February to an international conference in Istanbul about our experience with Marstacimab.