CSL Behring Haemophilia B Treatment User Guide
- June 22, 2024
- CSL Behring
Table of Contents
CSL Behring Haemophilia B Treatment
Product Information
Specifications
- Manufacturer: CSL Behring Ltd
- Product: Haemophilia B Medication
- Key Component: Factor IX
- Usage: Treatment for individuals with haemophilia B
Product Usage Instructions
Patient and Caregiver Guide
This guide is essential for understanding the treatment and care required
for individuals with haemophilia B. Your doctor will provide you with a
Patient Card containing important safety information.
Importance of the Patient Card
Keep your Patient Card with you at all times. It contains crucial details
related to your medication and treatment that healthcare professionals need to
know.
Understanding Haemophilia B
Haemophilia B is a genetic condition where individuals lack sufficient
factor IX, a protein necessary for blood clotting. This leads to a higher risk
of bleeding episodes.
Monitoring Liver Health
Your doctor will monitor your liver enzyme levels regularly to ensure the
effectiveness of the therapy. Monitoring frequency may vary over time.
Managing Abnormal Clotting
Individuals with haemophilia B may experience abnormal clotting after
treatment. Be aware of symptoms indicating blood clots and seek immediate
medical attention if needed.
Development of Factor IX Inhibitors
If bleeds are not controlled, factor IX inhibitors may be checked by your
doctor to assess treatment efficacy.
FAQ
- What should I do if I suspect an adverse event?
If you experience any adverse events, report them to CSL Behring UK Ltd at 01444 447 405.
This educational material is provided by CSL Behring Ltd and is mandatory as a
condition of the Marketing Authorisation in order to further minimise
important selected risks.
This medicine is subject to additional monitoring. This will allow quick
identification of new safety information. You can help by reporting any side
effects you may experience.
Read this patient/caregiver guide carefully before you receive HEMGENIX®
because it contains important information for you.
HEMGENIX® RMP Patient/Caregiver Guide
Version 1.0 – GBR-HGX-0020
Date of preparation: October 2023
In addition to this guide, your doctor will give you a Patient Card. Read it
carefully and follow the instructions on it.
Keep your Patient Card with you at all times and present it to any doctor or other healthcare professional you consult. The card contains important information for your safety related to the follow-up of this medication that you and healthcare professionals need to know before taking care of you.
What is HEMGENIX® and what is it used for?
HEMGENIX® is a gene therapy product that contains the active substance
etranacogene dezaparvovec.
People with haemophilia B are born with an altered form of a gene needed to
make factor IX, an essential protein required for blood to clot and stop any
bleeding. People with haemophilia B have insufficient levels of factor IX and
are prone to internal or external bleeding episodes.
HEMGENIX® is used for the treatment of severe and moderately severe
haemophilia B (congenital factor IX deficiency) in adults who do not have
current or past antibodies against factor IX protein, called factor IX
inhibitors.
How does HEMGENIX® work?
The active substance in HEMGENIX® is based on a virus that does not cause
disease in humans. This virus (also known as a vector) has been modified so
that it cannot spread in the body but can deliver a copy of the factor IX gene
into the liver cells. This allows the liver to produce the factor IX protein
and raise the levels of working factor IX in the blood. This helps the blood
to clot more normally and prevents or reduces bleeding episodes.
What you need to know before you are given HEMGENIX®
It is important that you fully understand the benefits and risks of HEMGENIX® treatment, what is known and not yet known about the long-term effects of this therapy, related to both safety and efficacy. Important information about HEMGENIX® treatment is provided in the sections that follow. Read it carefully and ask your doctor or nurse if you have further questions.
Liver health
BEFORE TREATMENT WITH HEMGENIX®
To decide if this therapy is suitable for you, your doctor will check your
liver health before you start treatment with HEMGENIX® using:
- Blood tests to check the level of liver enzymes in your blood
- Liver ultrasound
- Elastography tests to check for scarring or thickening of your liver
AFTER TREATMENT WITH HEMGENIX®
After treatment with HEMGENIX®, your doctor will continue to check your health
using blood tests. It is important that you discuss the schedule for these
blood tests with your doctor so that they can be carried out as necessary.
HEMGENIX® will trigger a response within your immune system that could lead to
increased levels of certain liver enzymes (transaminases) in your blood. This
is called transaminitis.
Your doctor will regularly monitor your liver enzyme levels to ensure that the therapy is working as it should:
Both before and after treatment, it is recommended that you avoid taking medication that can cause liver damage. Tell your doctor or nurse if you are taking, have recently taken, or might take any other medicines. If you are taking medications that are known to damage the liver (hepatotoxic medication), your doctor may decide that you may need to stop this medication to be able to receive HEMGENIX®.
Abnormal clotting of blood (thromboembolic events)
As you have haemophilia B, you usually have a reduced risk for developing a
blood clot (e.g., pulmonary thromboembolism or deep venous thrombosis [DVT]),
due to the inborn deficiency in your blood clotting system.
Restoring factor IX activity may bring your risk of blood clots up to a level
similar to that experienced by the general non-haemophiliac population.
If you have pre-existing risk factors for abnormal clotting, such as a history
of heart disease, arteriosclerosis, hypertension, diabetes, or advanced age,
the potential risk of having a blood clot may be higher.
Be aware of signs of abnormal clotting:
Risk of cancer potentially associated with HEMGENIX®
HEMGENIX® will insert into liver cells, and it may insert into the DNA of
other body cells. The clinical relevance of this is unknown, but it may
contribute to a risk of cancer. In clinical studies, no cancers due to
HEMGENIX® were identified. You should therefore discuss this with your doctor.
If you are a patient with pre-existing risk factors for liver cancer, your
doctor will regularly (e.g., annually) monitor your long-term liver health for
at least 5 years after HEMGENIX® administration and perform the following
tests:
- Annual liver ultrasound
- Annual blood test to check for increases in alpha-fetoprotein
Some risk factors for liver cancer are:
- Liver fibrosis (scarring and thickening of the liver)
- History of hepatitis B or hepatitis C
- Fatty liver (nonalcoholic fatty liver disease, or NAFLD)
- Excessive use of alcohol
In the event of cancer, your doctor may perform further tests to check if HEMGENIX® has inserted into the cell DNA.
Transmission of HEMGENIX®
The active substance in HEMGENIX® may temporarily be excreted through your
blood, semen, breast milk, or body waste – a process called shedding.
To ensure that people without haemophilia B are not exposed to HEMGENIX® DNA
through the shedding process in your body and/or semen, you must not donate
blood; semen; or organs, tissues and cells for transplantation after treatment
with HEMGENIX®.
After a male patient has been treated with HEMGENIX®, the patient and any
female partner must avoid pregnancy for 12 months. You should use effective
contraception (e.g., barrier contraception such as a condom or diaphragm).
This is to minimise any risk of the factor IX gene from a father’s HEMGENIX®
treatment being transmitted to a child with unknown consequences. For the same
reason, male patients must not donate semen. Discuss with your doctor which
methods of contraception are suitable.
HEMGENIX® should not be used in women who are pregnant, and is not recommended
in women who may become pregnant. HEMGENIX® should not be used during breast
feeding.
Development of factor IX inhibitors
Neutralising antibodies against factor IX proteins, called factor IX
inhibitors, may stop HEMGENIX® from working properly.
If your bleeds are not controlled or your bleeds return, your doctor may check
your blood for these factor IX inhibitors.
Pre-existing immunity against the vector
Some people may have natural pre-existing “immunity” (antibodies) against
adeno-associated virus (AAV) vectors used for gene therapy – this may stop the
factor IX gene from being delivered effectively.
High pre-existing immunity against the vector may reduce the efficacy of
HEMGENIX®.
Therefore, you are expected to be assessed for neutralising anti-AAV5
antibodies before treatment with HEMGENIX®.
Response to treatment
There is a possibility that not all patients will benefit from treatment with
HEMGENIX®.
Patients who do not respond to HEMGENIX® may still experience the long-term
risks as outlined in sections 3a-g.
HEMGENIX® will not be re-administered if you have not responded to treatment
or if you have lost the response.
Long-term follow-up after administration of HEMGENIX®
The long-term efficacy and safety of HEMGENIX® are still unknown.
Therefore, after treatment with HEMGENIX®, you will be expected to enrol in a
follow-up study to help assess the long-term safety of the treatment for 15
years, how well it continues to work, and any side effects that may be linked
to the treatment.
You are encouraged to talk to your doctor for additional information about the
follow-up study before treatment with HEMGENIX®.
What is the Patient Card for?
The HEMGENIX® Patient Card contains important safety information that you and
any healthcare professional may need to know after treatment with HEMGENIX®.
As explained in this guide, certain recommendations must be followed for safe
use of HEMGENIX®, and it is therefore essential to share this information with
all healthcare professionals you may need to consult. This is the role of the
Patient Card.
- Your doctor should give you a HEMGENIX® Patient Card on the day of HEMGENIX® administration
- Carry the card with you at all times; you can keep it in your wallet or purse
- Show the Patient Card to any doctor or nurse whenever you have a medical appointment
- If you need to go to Accident and Emergency, show the card as soon as you arrive
- Tell your caregiver or anyone close to you about your treatment and show them the Patient Card, because they may notice side effects that you are not aware of
This card also reminds you of important information you need to know about significant risks of HEMGENIX®.
What should I do if I suspect an adverse event?
If you get any side effects, talk to your doctor, pharmacist or nurse. This
includes any possible side effects not listed in the patient information
leaflet or patient/caregiver guide. You can also report side effects directly
to the MHRA via the Yellow Card Scheme. Reporting forms and information can be
found at www.mhra.gov.uk/yellowcard or
search for MHRA Yellow Card in the Google Play or Apple App Store.
Alternatively, call 0800 731 6789 for free, Monday to Friday between 9am and
5pm (messages can be left outside these hours). When reporting please provide
as much information as possible. By reporting side effects, you can help
provide more information on the safety of this medicine.
Adverse events should also be reported to CSL Behring UK Ltd on 01444 447 405.
Additional information
This guide and other documents developed as part of the HEMGENIX® risk
management plan can be downloaded at
www.medicines.org.uk. The eMC website is
managed and owned by Datapharm Communications Limited. CSL Behring UK Ltd
publishes risk management materials on this independent website.
For more information, please refer to the HEMGENIX® Patient Information
Leaflet, which is also available at
www.medicines.org.uk.
©2023 CSL Behring UK Ltd
4 Milton Road, Haywards Heath, West Sussex RH16 1AH
HEMGENIX® RMP Patient/Caregiver Guide Version 1.0 – GBR-HGX-0020
Date of preparation: October 2023