Zynteglo
Zynteglo (betibeglogene autotemcel) is a new injectable medication used to treat people with a blood condition called beta-thalassemia. This treatment is unique because it's the first cell-based gene therapy approved for this condition. While Zynteglo (betibeglogene autotemcel) only needs to be infused one time, the preparation, treatment, and recovery process can take months to complete. You'll also need to go through many blood tests for monitoring.
What is Zynteglo (betibeglogene autotemcel)?
What is Zynteglo (betibeglogene autotemcel) used for?
Beta-thalassemia in adults and children who need regular RBC transfusions
How Zynteglo (betibeglogene autotemcel) works
If you have beta-thalassemia, there are mutations (changes) in your gene that cause you to have less hemoglobin (a protein) and fewer red blood cells (RBCs) in your blood. As a result, your RBCs aren't able to carry enough oxygen to different parts of your body. If your condition is very serious, you'll need to get blood transfusions regularly.
Zynteglo (betibeglogene autotemcel) is a cell-based gene therapy. Your Zynteglo (betibeglogene autotemcel) treatment is designed specifically for you. Your provider will remove cells from your bone marrow and change their genetic material so that your cells are able to make healthy levels of hemoglobin. Then, these cells will be infused back into your body to help you make the hemoglobin you need. When you're treated with Zynteglo (betibeglogene autotemcel), it's likely that you will require fewer blood transfusions, or need none at all.
Drug Facts
What are the side effects of Zynteglo (betibeglogene autotemcel)?
Common Side Effects
- Mouth or gut inflammation (swelling/irritation) (95%)
- Fever during low white blood cell count (51%)
- Fever (49%)
- Vomiting (49%)
- Hair loss (44%)
- Nose bleed (42%)
- Stomach pain (39%)
- Muscle and bone pain (37%)
- Cough (34%)
Other Side Effects
- Headache
- Rash
- Diarrhea
- Nausea
- Constipation
- Low appetite
- Skin color changes
Serious Side Effects
- Serious allergic reaction: trouble breathing, rash, swelling of lips, tongue, or throat
- Severe bleeding: stomach cramps, red or tarry stool, blood in the urine, any bleeding that's difficult to stop, tiredness, weakness
- Serious infection: fever, cough, sore throat, tiredness, chills, body aches
Source: FDA
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Bleeding—bloody or black, tar-like stools, vomiting blood or brown material that looks like coffee grounds, red or dark brown urine, small red or purple spots on skin, unusual bruising or bleeding
- Infection—fever, chills, cough, or sore throat
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Zynteglo (betibeglogene autotemcel)
Pros
First cell-based gene therapy for beta-thalassemia
89% of people in clinical trials no longer needed blood transfusion for at least 12 months after treatment
Can be used in children and adults
Given as a single dose for one-time treatment
Not many interactions with other medications
Cons
Only available as an IV injection
Treatment process can take months to complete
Requires monitoring with frequent blood tests after treatment
Many side effects, including mouth and gut pain and swelling, fever, and vomiting
Unknown if safe during pregnancy or breastfeeding
Pharmacist tips for Zynteglo (betibeglogene autotemcel)
Make sure to complete all of your lab work before Zynteglo (betibeglogene autotemcel), including blood cell counts and screenings for different blood viral infections, so your provider can make sure it's safe for you to take.
Don't take human immunodeficiency virus (HIV) antiretroviral medications or hydroxyurea (Hydrea) for at least 1 month before your stem cells are collected, and until they're fully collected. These medications can interfere with the way your stem cells are changed in the lab to make Zynteglo (betibeglogene autotemcel).
Stop taking iron chelators (e.g., deferasirox (Exjade)) 7 days before starting the stem cell collection process. Avoid iron chelators for 6 months after receiving Zynteglo (betibeglogene autotemcel). Iron chelators are medications used to remove extra iron from your blood. Some iron chelators can interact with other medications used during Zynteglo (betibeglogene autotemcel) treatment and also make it difficult for your stem cells to recover after treatment.
After receiving Zynteglo (betibeglogene autotemcel), don't donate blood, organs, tissues, or cells at any point in the future.
You'll need to get frequent blood work done after receiving Zynteglo (betibeglogene autotemcel) treatment. This is because this treatment can make it difficult for your platelets and certain blood cells to recover, so you can be at risk for bleeds and infections. Please ask your provider how often you need to get blood work.
It isn't known if live vaccines (e.g., MMR (measles, mumps, and rubella vaccine), Varivax (Varicella vaccine) for chickenpox) are safe or work well after receiving Zynteglo (betibeglogene autotemcel). Speak to your provider before getting any vaccines.
While Zynteglo (betibeglogene autotemcel) hasn't been studied during pregnancy, the other medications you'll take before getting Zynteglo (betibeglogene autotemcel) is known to be harmful during pregnancy. Your provider might ask you to use a birth control method at the start of the stem cell collection process and for 6 months after getting Zynteglo (betibeglogene autotemcel).
If you're pregnant or breastfeeding, discuss the risks and benefits of Zynteglo (betibeglogene autotemcel) with your provider. There isn't enough information to know if it's safe for you or your baby while pregnant or breastfeeding, but other medications used during the treatment process can be harmful.
Frequently asked questions about Zynteglo (betibeglogene autotemcel)
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What are the risks and warnings for Zynteglo (betibeglogene autotemcel)?
Zynteglo (betibeglogene autotemcel) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Bleeding (problem with platelets)
After you receive Zynteglo (betibeglogene autotemcel) treatment, it can take a while for your platelet count rise to a healthy level. If you have low platelet levels, you're at a higher risk of bleeding because it's more difficult for your blood to clot. Your provider will monitor your platelet count and talk with you about your bleeding risk. They'll also let you know when you're no longer at risk for bleeds. If you've signs of bleeding, such as blood in your urine or stool, coughing up blood, severe headache, or severe unexplained bruising, get medical help right away.
Infection
You'll experience a low white blood cell (WBC) count after treatment with Zynteglo (betibeglogene autotemcel). Sometimes, your WBC count can remain low even more than a month after receiving Zynteglo (betibeglogene autotemcel). When you have a low number of WBCs, this put you at higher risk for infection. Your provider will monitor your WBC count after treatment until they return to normal. In some cases, you might receive rescue treatment with extra stem cells. Call your provider right away if you develop signs of infection, such as fever, sore throat, or cough.
Possible risk for blood cancer
While no cases have been seen in clinical studies, there's a small possibility that you might develop blood cancer after treatment with Zynteglo (betibeglogene autotemcel). For your safety, your provider will monitor your blood counts to check for cancer at 6 months, 12 months, and every year after that for at least 15 years after treatment. Speak with your provider if you have concerns. Let your provider know right away if you have symptoms of blood cancer, such as chills, frequent infections, swollen lymph nodes, unexplained weight loss.
Serious allergic reaction
Risk factors: Allergy to dimethyl sulfoxide (DMSO)
Though rare, you might experience a serious allergic reaction with Zynteglo (betibeglogene autotemcel), including life-threatening reactions like anaphylaxis. Get emergency help right away if you experience trouble breathing, rash, or swelling in your lips, tongue, or throat.
False positive for HIV
Zynteglo (betibeglogene autotemcel) can cause a false positive test result for HIV. This doesn't mean you have HIV. If you need to have an HIV test, speak with your provider about the appropriate test to use to make sure you get an accurate result.
Zynteglo (betibeglogene autotemcel) dosage forms
Typical dosing for Zynteglo (betibeglogene autotemcel)
Your provider will calculate your dose based on things like your body weight and the number of stem cells. Zynteglo (betibeglogene autotemcel) is given as a one-time infusion through your veins (IV).
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