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Overview: Kresladi is a gene therapy that is given as a one-time infusion into a blood vessel. It is used to treat certain children with leukocyte adhesion deficiency-I who have a faulty gene. Kresladi is made from the person’s own stem cells. The faulty cells are “corrected” and then infused back into the body, which helps the body make new blood cells and certain proteins properly. Common side effects may include mouth sores, infections, blood disorders, skin rash, nausea, vomiting, and fever. Tell your health care provider right away if you think you have an infection or have flu-like symptoms, muscle aches, or painful, warm, or discolored skin areas. Keep all appointments with your health care provider before and after receiving Kresladi.

Uses

What is Kresladi used for?

Kresladi (marnetegragene autotemcel) is used to treat certain children with leukocyte adhesion deficiency-I (LAD-I). LAD-I is a rare inherited condition that leads to a weakened immune system. People with LAD-1 can have frequent, serious infections.

Learn more about Kresladi for LAD-1.

Kresladi may also be used for other conditions as determined by your health care provider.

How does Kresladi work (mechanism of action)?

Kresladi is a gene therapy. It is for certain children with leukocyte adhesion deficiency-I (LAD-I) who have a faulty gene. Kresladi uses the person’s own stem cells that are collected from their blood. These cells are changed to include working copies of the faulty gene. The “corrected” cells are then infused back into the body, which helps the body make new blood cells and certain proteins properly.

How is Kresladi supplied (dosage forms)?

Kresladi is available in an injectable form that is given by a health care provider.

How should I store Kresladi?

Kresladi is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Kresladi?

The most common side effects of Kresladi are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Mouth or lip sores
  • Infections (see below)
  • Blood disorders (see below)
  • Skin changes, such as sores, bumps, redness, or discoloration
  • Nausea and vomiting
  • Skin rash or inflammation 
  • Fever

There may be other side effects of Kresladi that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Kresladi?

While less common, the most serious side effects of Kresladi are described below, along with what to do if they happen.

Severe Allergic Reactions. Kresladi may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Infections. You may have a higher risk of infections, including serious infections, after receiving Kresladi. Your health care provider may give you certain medicines to prevent infections. Call your health care provider right away if you have an infection or any of the following symptoms.

  • Burning or stinging when you pee 
  • Coughing, or coughing up blood
  • Fever, sweats, chills, sore throat, or muscle aches
  • Shortness of breath
  • Diarrhea or stomach pain
  • Weight loss
  • Painful, warm, swollen, or discolored skin

Liver Problem. Some people who received Kresladi have had a liver problem called veno-occlusive disease. This happens when small blood vessels in the liver get blocked. Keep all appointments for blood work to check the health of your liver. Call your health care provider right away if you have any of the following symptoms.

  • Stomach or belly pain or swelling
  • Sudden weight gain
  • Nausea or vomiting
  • Weakness or unusual tiredness
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Blood Disorders. Some people receiving Kresladi have had blood disorders, such as low neutrophil levels (neutropenia) and low platelet levels (thrombocytopenia). Keep all appointments with your health care provider. Get help right away if you have any of the following symptoms of blood disorders.

  • Fever
  • Shortness of breath
  • Pale or yellowish skin
  • Easy bruising or bleeding
  • Frequent infection
  • Unusual weakness or tiredness
  • Dizziness, lightheadedness, or feeling like you are about to pass out
  • Headache
  • Fast or abnormal heartbeat

Cancer. Kresladi may increase the risk of you having a new cancer or may cause a previously treated cancer to come back. Your health care provider will likely monitor you for cancer at least every year for at least 15 years after getting Kresladi. Tell your health care provider about any new symptoms that you may get. 

Warnings & Precautions

Who should not use Kresladi?

Allergies to Ingredients. People who are allergic to any of the following should not use Kresladi.

  • Kresladi 
  • Marnetegragene autotemcel
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Kresladi.

What should I know about Kresladi before using it?

Kresladi is usually given by a health care provider in a hospital or clinic. It is given as a one-time infusion into a vein (IV).

You will need to take certain steps before receiving Kresladi. For example, you will need to have stem cells taken from your blood (apheresis). You will also need to receive certain medicines before receiving Kresladi. Talk to your health care provider about the steps you need to take.

It is very important to keep all appointments with your health care provider before and after receiving Kresladi. These appointments help your health care provider make sure the treatment is safe and working well for you.

There is a small risk that the “corrected” stem cells in Kresladi may not grow properly in your body. Your health care provider will likely keep a back-up supply of your stem cells before you receive treatment in case they are needed later. If you have any questions, ask your health care provider.

Tell all of your health care providers that you have received Kresladi. Kresladi may affect the results of certain lab tests, including HIV tests. 

You should not donate blood, organs, cells, or tissues at any time after receiving Kresladi. Talk with your health care provider if you have concerns. If you need a blood transfusion after receiving Kresladi, the blood may need to be treated with a special process first to make it safer for you.

The treatments used to prepare your body for Kresladi may make it hard for you to get pregnant or get your partner pregnant in the future. If you have concerns, talk to your health care provider.

What should I tell my health care provider before using Kresladi?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Kresladi is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

Vaccines. Talk with your health care provider about any vaccines that you may need. You should not receive certain vaccines for some time before or after receiving Kresladi. Tell your health care provider about any vaccines you have recently received or have scheduled.

Other Medicines and Supplements. Kresladi may interact with other medicines and supplements. Before receiving Kresladi, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. Kresladi should not be given during pregnancy. It is not known if or how Kresladi could affect pregnancy or harm a fetus. If you can get pregnant, you should have a test to make sure you are not pregnant before you take the steps needed to receive Kresladi. Tell your health care provider if you are pregnant or plan to become pregnant.

Breastfeeding. It is not known if Kresladi passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should receive Kresladi while breastfeeding.

How can I get more information about Kresladi?

For more information about Kresladi, you can visit the drugmaker’s website at www.kresladi.com or call them at 800-982-2410.

Interactions

Does Kresladi interact with foods or drinks?

There are no known interactions between Kresladi and foods or drinks.

It is unknown if drinking alcohol will affect Kresladi.

Does Kresladi interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are taking any medicine for HIV or AIDS (an antiretroviral medicine) before  Kresladi.

This may not be a complete list of medicines that can interact with Kresladi. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Kresladi?

If you or someone else has used too much Kresladi, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Kresladi?

Kresladi is usually given by a health care provider in a hospital or clinic. If you miss your appointment to receive Kresladi, call to make another appointment as soon as possible.