STRIMVELIS 1-10 MILLION CELLS/ML Infuzja wyciek
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Treść ulotki
Strimvelis 1-10 x 10 cells/mL dispersion for infusion
Cell fraction enriched with autologous CD34 cells containing genetically modified CD34 cells
with a retroviral vector containing the cDNA sequence encoding for human adenosine deaminase (ADA)
Medicinal product subject to additional monitoring. This will allow the rapid identification of new
safety information. You can contribute by reporting any adverse effects experienced by your child
while taking this medicine. See the end of section 4 for information on
how to report adverse effects.
child because it contains important information for your child.
- If you want more information or advice, ask your child's doctor or nurse.
- If your child experiences any of the side effects, including effects not listed in this leaflet, ask your child's doctor or nurse. See section 4.
- Your child's doctor will give you a patient alert card containing important safety information about your child's treatment with Strimvelis. Read it carefully and follow the instructions.
- Always carry the patient alert card with you and show it to the doctor or nurse whenever your child sees them or if your child goes to hospital.
- 1. What Strimvelis is and what it is used for
- 2. What you need to know before Strimvelis is given to your child
- 3. How Strimvelis is given
- 4. Possible side effects
- 5. How to store Strimvelis
- 6. Contents of the pack and other information
1. What is Strimvelis and what is it used for
Strimvelis is a type of medicine called gene therapy. It is produced specifically for each patient.
Strimvelis is used in children to treat a serious condition called ADA-SCID
(adenosine deaminase deficiency severe combined immunodeficiency). It is used when your child
cannot receive a bone marrow transplant from a family donor because the compatibility is not
sufficient.
ADA-SCID occurs due to a defective gene in the blood cells of your child's immune system. As a result, the cells do not produce enough of an enzyme called adenosine
deaminase(ADA) and your child's immune system does not function properly to defend the
body against infections.
To produce Strimvelis, bone marrow stem cells from your child are modified in the laboratory
to insert a gene that produces ADA. When these modified stem cells are returned to your child, they can divide to produce different types of blood cells, including cells involved in
your child's immune system.
2. What you need to know before Strimvelis is given to you (or to your child)
Strimvelis is not suitable for some people
Strimvelis must not be given if your child:
- is allergicto any of the ingredients of this medicine (listed in section 6).
- has or has had a type of cancercalled leukaemiaor myelodysplasia
- tests positive for HIV or some other infections(your doctor will advise you about this)
- has already been treated with gene therapy
Warnings and precautions
Information about cell-based medicines, such as Strimvelis, must be kept for 30 years
at the hospital. The information that will be kept about your child will be their first name and surname and the
batch number of Strimvelis received.
Strimvelis is prepared specifically from the patient’s own cells. It must never be given to anyone
else.
Inserting a new gene into DNA can cause blood cancers. There has been one case of
blood cancer, called leukaemia, in a patient several years after treatment with Strimvelis. It is therefore
important to monitor your child for symptoms of leukaemia. These include fever, shortness of breath,
paleness, night sweats, fatigue, swollen lymph nodes, frequent infections, a tendency to
bleed and/or bruise easily, or red or purple spots under the skin. If your child develops any
of these symptoms you should contact your doctor immediately.
Before treatment with Strimvelis, your child will be given other medicines (see
sections 3 and 4 for more information about these medicines, including possible side effects).
If your child tests positive for hepatitis C, they may still be treated under certain
conditions. Your doctor will discuss this with you if necessary.
Central venous catheters are thin, flexible tubes that are inserted by a doctor into a large
vein to access your child’s bloodstream. The risks of these lines are infections and the
formation of blood clots. Your doctor and nurses will monitor your child for any
complications of the central venous catheter.
Treatment with Strimvelis has not been successful in some patients. These patients have received alternative
treatment options.
There is a small risk of infection following treatment. Your child’s doctors and nurses will monitor them during the infusion for signs of infection and provide care if needed.
Some patients may develop autoimmunity, i.e. trigger an immune response against their own
cells or tissues (see section 4). Your child’s doctor will discuss this with you if necessary.
After treatment, your child should never donate blood, organs or tissues in the future. This is because
Strimvelis is a gene therapy product.
When treatment with Strimvelis cannot be completed
In some cases, it may not be possible to proceed with the planned treatment with Strimvelis for the
following reasons:
- a problem with taking cells from your child’s bone marrow to produce the medicine
- not enough cells of the correct type in the tissue taken from your child’s body to produce the medicine
- the medicine does not meet all quality controls
- a delay in getting the medicine to the hospital where your child’s treatment is taking place.
Before receiving Strimvelis, your child will be given chemotherapy to remove their
existing bone marrow. If Strimvelis cannot be given after chemotherapy, or if the modified stem cells do not engraft (take) in your child’s body, your doctor will give
your child replacement stem cells, using the reserve sample that had been taken and stored
before the start of treatment (see also section 3, How Strimvelis is given).
You may need other treatments
Strimvelis undergoes several checks before it is used. Because it is given shortly after
being made, the final results of some of these checks will not be available before the
medicine is given. If the checks show anything that could affect your child, your doctor will treat them appropriately.
Other medicines and Strimvelis
Tell your doctorif your child is taking, has recently taken or might take any
other medicines.
Your child must not be given vaccines called live vaccines for 6 weeks before they
are given the medicine for conditioning to prepare them for treatment with Strimvelis, nor
after treatment while your child’s immune system is recovering.
Strimvelis contains sodium
This medicine contains 42 to 137 mg of sodium (the main component of table salt) per dose.
This is equivalent to 2-7% of the recommended maximum daily intake with the diet of an adult.
3. How Strimvelis is administered
Strimvelis is administered via infusion ( infusion) into a vein ( intravenously). It must be
administered exclusively in a specialized hospital and by a doctor who has experience in treating
patients with ADA-SCID and in the use of this type of medicine.
Before Strimvelis is prepared, the doctor will perform checks to ensure that your child does not have
certain infections (see section 2).
Two samples are taken
The doctor will take two samples of bone marrow cells before the scheduled treatment:
the reserve sample, collected at least 3 weeks before treatment with Strimvelis. This will be
stored to be administered as replacement cells if Strimvelis cannot be
administered or if it does not work (see "When treatment with Strimvelis cannot be
completed"in section 2)
the treatment sample, collected 4 or 5 days before treatment with Strimvelis. This will be
used to prepare Strimvelis, by inserting a new gene inside the cells.
Before and during treatment with Strimvelis
| g When | What is done | Why |
| A At least 3 weeks before treatment | Collection of the reserve sample of stem cells | to be stored as reserve (see above) |
| Approximately 4 or 5 days before treatment | Collection of the stem cell sample for treatment | to prepare Strimvelis (see above) |
| 3 days and 2 days before treatment | A medicine called busulfan is administered 4 times a day for 2 days (8 doses in total) | to prepare the bone marrow for treatment with Strimvelis and eliminate existing stem cells |
| Approximately 15-30 minutes before the treatment | An antihistamine medicine may be administered | or to make it less likely that you will have infusion reactions |
| Strimvelis is administered... | c a via infusion into a vein. The time required is approximately 20 minutes | |
4. Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Side effects related to Strimvelis are all caused by the fact that the immune system becomes
overactive and attacks the body's own tissues. Some side effects may be related to the
busulfan medicine used to prepare your child's bone marrow for treatment; these are
marked with an asterisk (*) in the following list.
Very common:may affectmore than 1 in 10 people:
- runny or blocked nose ( allergic rhinitis)
- shortness of breath, difficulty breathing ( asthma)
- skin inflammation and itching ( atopic dermatitis, eczema)
- high temperature ( pyrexia)
- underactive thyroid ( hypothyroidism)
- high blood pressure ( hypertension)*
- decreased number of red or white blood cells ( anemia, neutropenia)*
- increased liver enzymes (which indicates stress to the liver)*
- blood test results positive for antinuclear antibodies and anti-smooth muscle antibodies(which may suggest possible autoimmunity)
Common:may affectup to 1 in 10 people:
- red or purple spots on the skin, bleeding under the skin ( autoimmune thrombocytopenia)
- inflammation of the thyroid ( autoimmune thyroiditis)
- weakness and pain in the feet and hands caused by nerve damage ( Guillain-Barré syndrome)
- inflammation of the liver ( autoimmune hepatitis)
- reduction in the number of blood cells ( autoimmune hemolytic anemia, autoimmune aplastic anemia)
- blood test results positive for antineutrophil cytoplasmic antibodies(which may lead to autoimmune inflammation and swelling of blood vessels and possibly increased infection level)
- a type of blood cancer called leukemia
If you have any doubts about the symptoms or side effects, or if any symptom worries you, contact
your child's doctor or nurse.
Reporting of side effects
If your child gets any side effect, talk to your doctor or nurse. You can also report side effects directly through
the national reporting system listed in Annex V.
By reporting side effects you can help provide more information on the safety of
this medicine.
5. How to store Strimvelis
The following information is intended for healthcare professionals only.
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date and time (exp.) stated on the container label and on
that of the infusion bag.
Store at 15-30 ° C.
This medicine contains genetically modified human cells. Unused medicine and waste
materials must be disposed of in accordance with local guidelines on the handling of materials of
human origin. Since this medicine will be administered by a qualified healthcare professional, they are
responsible for the correct disposal of the product. This will help protect the environment.
6. Contents of the pack and other information
What Strimvelis contains
- The active substance is an enriched cellular fraction of autologous (patient's own) CD34 cells containing CD34 cells genetically modified with a retroviral vector containing the cDNA sequence encoding for human adenosine deaminase (ADA). The concentration is 1-10 x 10 CD34 cells /mL.
- The other component is sodium chloride (see paragraph 2, “ Strimvelis contains sodium”)
Description of the appearance of Strimvelis and contents of the pack
Strimvelis is a cell dispersion for infusion from turbid to clear, from colorless to pink, supplied in one or
more infusion bags. The infusion bags are inside a closed container.
Marketing Authorisation Holder
Fondazione Telethon ETS
Via Varese 16/B
00185 Roma
Italy
Manufacturer
AGC Biologics S.p.A.
Via Olgettina 58
20132 Milano
Italy
Other sources of information
More detailed information on this medicine is available on the website of the European Medicines
Agency: http://www.ema.europa.eu. Additionally, links to other websites on rare diseases and related
therapeutic treatments are provided.
European on the website of the European Medicines Agency.
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The following information is intended exclusively for healthcare professionals:
Precautions to be taken before handling or administering the medicine
Healthcare professionals handling Strimvelis must take appropriate precautions (wearing gloves,
protective clothing and eye protection) to avoid potential transmission of infectious diseases.
Strimvelis is transported directly to the healthcare facility where the infusion will be administered. The/e
infusion bag/bags is/are placed in a closed outer container. The bags must be
stored in the outer container until the time of use.
Strimvelis is for autologous use only. The patient's identity must match the unique patient identification
number on the/e infusion bag/bags and/or the outer container before infusion.
Gently shake the infusion bag to redisperse any cell aggregates, administer
using a transfusion administration set with a filter to remove any residual cell aggregates.
Precautions to be taken for disposal of the medicine
Local guidelines on the handling of human-derived materials must be followed for the
unused medicine and derived waste. All materials that have been in contact with Strimvelis (solid and liquid
waste) must be handled and disposed of as potentially infectious waste in accordance with
local guidelines on the handling of human-derived materials.
Accidental exposure
Avoid accidental exposure to Strimvelis. In case of accidental exposure, local guidelines on the handling of
human-derived materials must be followed, which may include
washing contaminated skin and removing contaminated clothing. Work surfaces and
materials that have potentially been in contact with Strimvelis must be decontaminated with
appropriate disinfectants.
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- Postać farmaceutycznaInfusion leak, 1-10 MILLION CELLS/ML
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- Producent
- Te treści mają charakter wyłącznie informacyjny i nie zastępują konsultacji lekarskiej.
- Zamienniki STRIMVELISPostać farmaceutyczna: Granules, 800 MGSubstancja czynna: pidotimodProducent: POLICHEM S.R.L.Wymaga receptyPostać farmaceutyczna: Powder and solvent for intravesical suspension, 50 mlSubstancja czynna: BCG vaccineBez receptyPostać farmaceutyczna: Powder and solvent for intravenous infusion solution, 1 MGSubstancja czynna: tasonerminProducent: BELPHARMA S.A.Wymaga recepty
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STRIMVELIS jest produkowany przez FONDAZIONE TELETHON ETS. Nazwy handlowe i opakowania mogą się różnić w zależności od dystrybutora.
Lekarze, tacy jak Lekarze rodzinni, Psychiatrzy, Dermatolodzy, Kardiolodzy, Endokrynolodzy, Gastroenterolodzy, Pulmonolodzy, Nefrolodzy, Reumatolodzy, Hematolodzy, Zakaźnicy, Alergolodzy, Geriatrzy, Pediatrzy, Onkolodzy, mogą ocenić, czy stosowanie STRIMVELIS jest odpowiednie w Twoim przypadku, w zależności od sytuacji klinicznej i lokalnych przepisów. Możesz umówić konsultację online, aby omówić objawy i możliwe dalsze kroki.
Polska posiada dobrze rozwiniętą infrastrukturę ochrony zdrowia w dużych miastach, takich jak Warszawa, Kraków, Wrocław i Gdańsk. Apteki są łatwo dostępne i działają zgodnie z obowiązującymi przepisami, zapewniając dostęp do leków na receptę.
Możesz kupić STRIMVELIS w Warszawie, Krakowie, Wrocławiu lub Gdańsku w każdej aptece, posiadając ważną receptę.
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Inne leki zawierające tę samą substancję czynną () to m.in. AXIL, BKG MEDAK, BEROMUN. Mogą one różnić się nazwą handlową lub postacią, ale zawierają ten sam składnik terapeutyczny. Przed zmianą lub rozpoczęciem nowego leku skonsultuj się z lekarzem.
