New Treatment Option for Prophylaxis in Severe Haemophilia A

December 4th, 2019

Emicizumab (brand name: Hemlibra) is a bi-specific antibody which binds to both activated FIX and FX and allows the coagulation cascade to continue, and the blood to clot, in the absence of FVIII. Hemlibra mimics the effect of FVIII and can be used as an option for prophylaxis for people with FVIII deficiency. Hemlibra has been used in Ireland for the past year for people with FVIII inhibitors. It has now been licenced for prophylaxis in people with FVIII deficiency. There is existing clinical experience with Hemlibra in FVIII deficiency in Ireland as a number of people with haemophilia here participated in the clinical trial for Hemlibra in FVIII deficiency.

The Haemophilia Product Selection Monitoring Advisory Board (HPSMAB), in which the Society participates, recently completed a negotiation which has resulted in access to Hemlibra (Emicizumab) for people with severe factor VIII (FVIII) deficiency / Haemophilia A as an option for prophylaxis.

This means that every individual with severe FVIII deficiency will have the option to switch to Hemlibra or remain on extended half life FVIII (current treatment, which is Elocta). This will be an individual decision to be made following a consultation each person with haemophilia or parent will have with their comprehensive care centre.

What is Hemlibra used for?

Hemlibra is only used as prophylaxis (prevention of bleeds). Hemlibra cannot be used to treat acute bleeding and Elocta will still be used for acute bleeds for people on Hemlibra.  Elocta will also still be used for most surgeries and procedures in people on Hemlibra but some minor surgeries might be done without giving Elocta in advance – this will be decided after discussion with the individual person on Hemlibra.

What is different about Hemlibra?

Hemlibra is an antibody treatment which has been engineered to replace the action of Factor VIII in the body. It is manufactured and is not derived from blood products. Hemlibra is given by subcutaneous injection (under the skin) and lasts a long time in the body so it only needs to be given either once a week, once a fortnight or once a month (the dose is different depending on how often it is given).

Is Hemlibra suitable for everyone?

At the moment, Hemlibra is licensed for prophylaxis in people of all ages (including children) with severe FVIII deficiency. Therefore, in general, people with moderate or mild Haemophilia would not be suitable for this treatment. In addition, there are some people with a history of thrombosis (excessive clotting) or inhibitors which were cleared by immune tolerance treatment in the past who may not be suitable to have Hemlibra. This will be discussed individually with each person.

Will I have to change to Hemlibra?

Elocta is the current Factor VIII concentrate in use in Ireland for prophylaxis and on demand FVIII replacement.  Elocta will continue to be the FVIII concentrate available for use in Ireland. People on prophylaxis can continue to use Elocta and do not have to change treatment. The benefits and possible risks of a change in prophylactic treatment to Hemlibra will be discussed with each person.

Are there any side effects with Hemlibra?

The most common side effect is a minor skin reaction at the site of the injection which might cause redness, minor swelling or itch. This usually settles down very quickly and does not need treatment. Rarely, people have experienced thrombosis (excessive clotting) or a condition known as Thrombotic Microangiopathy. People starting Hemlibra will be reviewed to ensure that they do not have specific risks for these conditions and will be monitored while they are taking the treatment.

How is Hemlibra delivered and stored?

Hemlibra will be delivered by TCP, in the same way that Elocta is delivered now. Hemlibra needs to be stored in a fridge (TCP can provide a fridge or will service any existing product fridges). Hemlibra vials can be stored out of the fridge for up to 7 days in total.

Will I still need to scan Hemlibra on the Homescan App?

As this is a new treatment, it is really important that the Haemophilia treatment team monitors people taking this treatment and that we know when people are having bleeds or any other complications It is very important that people using Hemlibra continue to scan their treatment on the Homescan App, just as they scan Elocta now. If you cannot scan you will be able to complete paper treatment records. All patients who switch to Hemlibra will have to record their treatment either by scanning or by paper form

Will I still need to keep some Elocta at home?

Not necessarily. The experience of people taking Hemlibra as part of clinical trials (including some participants from Ireland) has been that bleed events are rare with Hemlibra. Depending on how far you are from the nearest treatment centre or hospital, you may decide with your Haemophilia team that it is not necessary to keep Elocta at home. This will be discussed with each person individually.

How do I change treatment to Hemlibra if this is the right treatment for me?

Once you have had a discussion with your Haemophilia clinic team, and if Hemlibra is chosen as the best prophylaxis for you, you will be asked to continue to use Elocta until your supplies have been used up. The recommended dose of Hemlibra will be decided by your Consultant Haematologist in discussion with you. The first and second doses of Hemlibra will need to be administered under medical supervision at your local Comprehensive Care Centre (CCC) and you will be given information and training on self-treatment with Hemlibra.  You will have some bloods and other tests such as a tracing of the heart (ECG) taken before treatment.  The home treatment nurses in your CCC will arrange with you for TCP to deliver supplies of Hemlbra to you after you have received your first dose.

Will everyone be able to change to Hemlibra if they wish to and the treatment is right for them?

Yes, if a person is reviewed and it is decided with them that Hemlibra is the right treatment, a plan to change to Hemlibra will be made as outlined above. However, we are not able to change everyone all at once and we need to plan each change so that each patient is managed safely. Also, there are some people who cannot access prophylaxis at the moment (for example if they have poor veins) or who are bleeding despite FVIII prophylaxis. These are people with an urgent clinical need for an alternative prophylaxis and Hemlibra will be offered to them first.

What follow up is needed if I change to Hemlibra?

It is recommended that three months after starting Hemlibra, a clinic review and bloods are done to assess your response to treatment. After this, if there are no new issues, clinic reviews will be done every 6 months as they are done now for people on Elocta.  Hemlibra is a relatively new treatment and it is very important that we continue to monitor treatment carefully. An appointment will be arranged for you to attend your nearest CCC for this clinic follow up.

Where can I get additional information?

The patient information leaflet on Hemlibra produced by the European Medicines Agency is reproduced at the end of this article.

A patient education booklet is currently being finalised. We will make this available on the website as soon as it is completed.

Your comprehensive care centre will provide you with hard copies of information in addition to a specific information card on Hemlibra when you attend for an individual discussion on your treatment options.

For further information from your comprehensive care centre, contact:

National Coagulation Centre (NCC) Dublin – Phone: 01- 4162141 | email: ncc@stjames.ie

Children’s Health Ireland at Crumlin – Phone: 01- 4096647 or 01- 4096939

Cork University Hospital – Phone: 021- 4922278

What is the role of the HPSMAB and who is involved?

The HPSMAB includes Haemophilia Consultants, representatives from the Irish Haemophilia Society and representatives of St James’s Hospital which is the contract holder for treatments for Haemophilia.  The HPSMAB reviews all Haemophilia treatments in Ireland and recommends treatments to the contract holder (St James’s Hospital), according to European and Irish procurement law.

Information Meetings

Each person with FVIII deficiency or parent will have an opportunity to discuss their future treatment options with their comprehensive care team in the coming months.

In order to provide the opportunity for more information on a collective basis the Society have organised information meetings, in collaboration with the Comprehensive care centres in Dublin and Cork and the treatment centre in Galway.

Cork (New Venue):

Date: Wednesday, January 8th 2020

Time:  18.30

Location: Imperial Hotel, Cork City

Dublin:

Date: Monday, January 13th 2020

Time: 7pm

Location: Irish Haemophilia Society Office

Galway

Date: Wednesday, January 29th 2020

Time: 7pm

Location: Maldron Hotel, Sandy Road, Galway

If you plan to attend any of these meetings, RSVP with your chosen location to Aoife in the office on: 01-6579900 or aoife@haemophilia.ie

Download info below, just click the links:

Hemlibra – Mechanism of Action | Hemlibra – Package leaflet Information for the user

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