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
Side effects of Zynteglo (betibeglogene autotemcel)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
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%)
Less Common Side Effects
- Headache
- Rash
- Diarrhea
- Nausea
- Constipation
- Low appetite
- Skin color changes
Zynteglo (betibeglogene autotemcel) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- 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
The following Zynteglo (betibeglogene autotemcel) 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.
Common questions about Zynteglo (betibeglogene autotemcel)
Thalassemia is a blood condition that's passed down from parents to children through genes. People with thalassemia don't make enough of an important protein in RBCs called hemoglobin. Hemoglobin carries oxygen to different parts of your body and helps RBCs function normally. Beta-thalassemia is one type of thalassemia. If you have this condition, you can experience symptoms such as pale skin, weakness, and dizziness. If your condition is serious, you'll need to get blood transfusions regularly.
Even though Zynteglo (betibeglogene autotemcel) is given as a one-time infusion, you'll start preparing for treatment months in advance. 1) Because your Zynteglo (betibeglogene autotemcel) is made specifically for you with your cells, your provider first collects your stem cells. This process can take a week and sometimes you might have to go through it more than once. To be safe, your provider also collects back-up stem cells in case there's a problem with the initial treatment. 2) Next, your stem cells are sent to a lab, where they're used to make Zynteglo (betibeglogene autotemcel) that's specific for you. This process takes about 3 months. 3) Once your Zynteglo (betibeglogene autotemcel) is ready, your provider will prepare you for the infusion at the hospital. First, you'll receive medication to make room in your bone marrow for the new cells. Then, you'll receive Zynteglo (betibeglogene autotemcel) as an infusion through your veins. 4) Afterwards, you'll recover in the hospital for about 3-6 weeks.
The length of your infusion will depend on how many infusion bags you're receiving. Each bag will be infused through your veins in less than 30 minutes. You might receive more than one bag to complete your treatment. Ask your provider how long your infusion will take.
Yes, Zynteglo (betibeglogene autotemcel) has been studied in children 4 years and older and has been proven to be safe and work well. If your child is under 4 years old, discuss alternative options with their provider.
While there isn't enough information to know if Zynteglo (betibeglogene autotemcel) affects your ability to have children, other medications used during the treatment process have risks of infertility (the inability to have children). If this concerns you, speak to your provider about the option to preserve your semen or eggs before treatment.
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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
Typical dosage 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).