What is Zynteglo?
Zynteglo is a single-time gene therapy for treating beta-thalassemia (also called beta-thalassemia major or also known as Cooley's anemia) in patients who require frequent transfusions.Beta-thalassemia is a result of an alteration to the gene that makes beta-globin, which results in the body producing the beta-globin gene in a reduced or nonexistent way.
Zynteglo is specifically designed for each patient, utilizing the patient's blood stem cells. It also includes functional copies of the beta-globin gene in your cell. This could enable you to make enough hemoglobin to cease receiving regular transfusions.
Important information
Zynteglo could cause some of the following side effects that day:
- Heart rate increases
- Abdominal pain
Zynteglo could cause adverse side effects as a result of treatment for a period of up to 6 months:
- Platelets are low, which could reduce the capacity for blood clotting and cause bleeding.
- An insufficient amount of white blood cell count may make you more susceptible to the spread of.
- The pain in the legs or arms
Get in touch with your healthcare provider immediately if you notice unusual or unexpected bleeding. It could include any of the following indications or signs:
- A severe headache
- Abnormal bruising
- Nose bleed
- Urine, blood in the stool, or in your vomit
- The coughing up of blood
- Atypical back or stomach pain
You might experience adverse reactions that are related to other medications you take as part of your treatment plan. Consult your physician about these possible side effects. Your health care provider could give you additional medications to help treat the side effects.
It is essential to monitor your blood every year for at least 15 years for any changes in your blood. There is a risk of developing blood cancer as a result of this procedure; however, there are no reported cases in clinical research. If you're diagnosed with cancer, you should have your doctor contact Bluebird Bio by calling 1-833-999-6378.
How to take Zynteglo?
Your doctor will prescribe you additional medicines, such as chemotherapy medication, as part of the treatment you receive with Zynteglo. It is important to speak with your doctor about the potential risks and benefits of any medication used during your therapy.
Following treatment and radiation therapy, it might not be possible to get pregnant or have a child. It is recommended to discuss possibilities for preserving fertility with your doctor prior to starting the treatment.
Step 1. Zynteglo was created specifically to meet your needs by the stem cells of your blood. The healthcare professional will collect the stem cells of your blood in the process of mobilization and anaphoresis. The procedure takes about a week, and it may require a repeat procedure.
"Back-up" stem cells (or "rescue cells'') are also stored and retrieved at the hospital. This is to protect patients in the event of any issue during the treatment process. If this occurs, the stem cells that you have regenerated will be returned to you. If you get backup cells, there will be no advantage from Zynteglo.
Step 2. Blood stem cells are sent to a factory that uses them to create Zynteglo. It can take between 70 and 90 days after the cells are taken to create as well as test Zynteglo before being sent to your physician. However, the duration of time could vary.
Step 3. In the days before you get Zynteglo, the healthcare professional will provide you with chemotherapy for a couple of days in order to increase the size of your bone marrow. It is possible to be admitted to a hospital to undergo this procedure and stay in the hospital until the time of the Zynteglo infusion.
Step 4. Zynteglo can be given through an intravenous injection (into the vein). It is possible to receive several bags of Zynteglo. Each bag of infusion takes 30 minutes or less.After receiving the infusion, remain within the hospital for three weeks to ensure your healthcare team is able to closely observe your recovery. Your doctor will decide the date you can return home.
What should be avoided?
- Don't donate organs, blood, tissue, or cells.
Side effects of Zynteglo
The most frequently reported side effects during the course of treatment are:
- Heart rate increases
- Abdominal pain
The most commonly reported adverse effects that can occur after treatment lasting up to 6 months are:
- Platelets are low, which can reduce the capacity of blood to clot and cause bleeding.
- Low level of blood white cells, which can increase your risk of infection.
- Legs or arms hurt.
Zynteglo does not cause HIV infection. Treatment with Zynteglo could result in false-positive human immunodeficiency virus (HIV) results when using certain tests sold by commercial companies. If you require an HIV test, speak to your doctor about the most appropriate test for you.
This is not the only list of possible adverse consequences. Contact your doctor to get medical advice on adverse effects. You may report any adverse reactions to the FDA at 1-800-FDA-1088.
Interaction with other drugs
Don't take a drug that is used to treat HIV infection or hydroxyurea during the one month before mobilization or for the duration expected to get rid of the medication, up to the point that all cycles of apheresis have been completed.
It is recommended to stop taking iron chelators, such as deferoxamine (Desferal), deferasirox (Exjade, Jadenu), or deferiprone (Ferriprox), 7 days prior to the start of chemotherapy treatment.
