Description
Etuvetidigene autotemcel injection is used to treat Wiskott-Aldrich syndrome (WAS) in patients with mutation in the WAS gene and without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant (HSCT).
This medicine is to be given only by or under the immediate supervision of your doctor.
Before Using
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:
Allergies
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Pediatric
Appropriate studies have not been performed on the relationship of age to the effects of etuvetidigene autotemcel injection in children younger than 6 months of age. Safety and efficacy have not been established.
Geriatric
No information is available on the relationship of age to the effects of etuvetidigene autotemcel injection in geriatric patients.
Breastfeeding
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Drug Interactions
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Other Interactions
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.
Other Medical Problems
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Allergy to dimethyl sulfoxide (DMSO), history of—Use with caution. May increase the risk of an allergic reaction.
- Infection or
- Liver disease—Use with caution. May make these conditions worse.
- Previous treatment with hematopoietic stem cell transplant (HSCT) within 6 months or
- Previous treatment with hematopoietic stem cell gene therapy—Should not be used in patients with these conditions.
Proper Use
A doctor or other trained health professional will give you this medicine in a hospital. It is given through an IV catheter that is placed in a vein. This medicine must be given slowly, so your IV will need to stay in place for at least 30 minutes.
You may also receive other medicines (eg, cancer medicine, allergy medicine) before receiving this medicine.
This medicine is made specifically for each patient, using the patient's own blood cells. Your doctor will collect your blood through a procedure called mobilization and apheresis. Talk with your doctor if you have any concerns about this.
Do not use medicine to treat HIV for at least 1 month before this infusion until at least 7 days after receiving this medicine.
Precautions
It is very important that your doctor check your or your child's progress at regular visits to make sure that this medicine is working properly. Blood tests are needed to check for unwanted effects.
Tell your doctor if you are pregnant. If you can get pregnant, you must have a negative pregnancy test before this infusion. Male patients with female partners should use an effective form of birth control before this infusion and for at least 6 months after the last dose.
This medicine may cause infusion-related reactions. Check with your doctor right away if you have back pain, chest tightness, chills, fever, flushing, headache, nausea and vomiting, trouble breathing, or weakness.
This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with your doctor right away if you have chest tightness, cough, difficulty swallowing, fast heartbeat, hives, itching, skin rash, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.
Etuvetidigene autotemcel will lower the number of some types of blood cells in your body. Because of this, you may bleed or get infections more easily. To help with these problems, avoid being near people who are sick or have infections. Wash your hands often. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Brush and floss your teeth gently. Be careful when using sharp objects, including razors and fingernail clippers. Call your doctor right away if you have fever, chills, or any signs or symptoms of an infection.
Check with your doctor right away if you have cough or hoarseness, fever or chills, lower back or side pain, or painful or difficult urination. These may be symptoms of infection.
Check with your doctor right away if you or your child have pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, unusual tiredness or weakness, or yellow eyes or skin. These could be symptoms of a serious liver problem (eg, hepatic veno-occlusive disease).
This medicine may increase your risk for cancer after treatment with this medicine. Talk to your doctor if you have concern about this.
While you are being treated with etuvetidigene autotemcel, do not have any immunizations (vaccinations) without your doctor's approval. Live virus vaccinations should not be given until recovery following treatment with this medicine.
This medicine is made from donated human blood. Some human blood products have transmitted certain viruses to people who have received them, although the risk is low. Human donors and donated blood are both tested for viruses to keep the transmission risk low. Talk with your doctor if you have concerns about this risk.
Do not donate blood, organs, tissues, or cells at any time in the future.
Before you have any medical tests (eg, HIV test), tell the medical doctor in charge that you are taking this medicine. The results of some tests may be affected by this medicine.
Side Effects
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor or nurse immediately if any of the following side effects occur:
More common
- Black, tarry stools
- bladder pain
- bleeding gums
- bloody or cloudy urine
- chest pain or tightness
- chills
- cough
- diarrhea
- difficult, burning, or painful urination
- difficulty swallowing
- fast heartbeat
- fever
- frequent urge to urinate
- headache
- head injury
- loss of appetite
- lower back or side pain
- nausea
- pale skin
- pinpoint red spots on the skin
- redness or pain at the catheter site
- skin itching, rash, or redness
- sneezing
- sore throat
- stomach pain
- stuffy or runny nose
- swelling of the face, throat, or tongue
- trouble breathing
- ulcers, sores, or white spots in the mouth
- unusual bleeding or bruising
- unusual tiredness or weakness
- weight gain
- yellow eyes or skin
Incidence not known
- Dizziness
- puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:
More common
- Change in hearing
- constipation
- earache
- ear drainage
- white patches in mouth or on tongue
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
DRG-80008579
