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Kresladi (marnetegragene autotemcel) is a prescription gene therapy approved by the U.S. Food and Drug Administration (FDA) to treat children with severe leukocyte adhesion deficiency-I (LAD-I). LAD-I is a rare immune system condition that makes it harder for the body to fight infections.
Kresladi is a type of gene therapy. It is made using a child’s own blood-forming stem cells.
LAD-I stands for leukocyte adhesion deficiency-I. It is a rare primary immunodeficiency condition. In severe LAD-I, some white blood cells do not work the way they should.
These white blood cells have trouble leaving the blood and getting to areas of infection or inflammation. This can make it harder for the body to fight infections.
Kresladi works by adding working copies of the ITGB2 gene to the child’s own blood-forming stem cells. This helps the body make white blood cells with working CD18 protein. CD18 works together with another protein called CD11a.
Together, CD18 and CD11a help white blood cells move out of blood vessels and reach areas of infection or inflammation.
Doctors prescribe Kresladi when children have severe LAD-I caused by changes in both copies of the ITGB2 gene, one inherited from each parent. It is for children who do not have a matched sibling donor for a stem cell transplant.
Kresladi is given as a one-time intravenous (IV) infusion. It is given 24 to 48 hours after conditioning treatment is finished. Conditioning treatment prepares the body for Kresladi.
Kresladi is given at a qualified treatment center.
The recommended minimum dose of Kresladi for LAD-I is 2.8 × 10^6 CD34+ cells per kilogram of body weight. CD34+ cells are blood-forming stem cells. The dose is based on the child’s weight before the first cell collection.
Up to two product lots may be used to reach the minimum dose.
This information is based on the prescribing information, but your healthcare provider may tailor your treatment plan. Always follow their guidance.
In a clinical study of nine children treated with Kresladi for severe leukocyte adhesion deficiency-I, the most common side effects occurred in at least 30 percent of children. These include:
Kresladi can cause serious side effects that may require immediate medical attention. These include:
Get medical help right away if you think your child is having a serious reaction.
Rocket Pharmaceuticals, the manufacturer of Kresladi, does not offer a traditional copay card program because Kresladi is a one-time gene therapy administered at specialized treatment centers. Instead, patient support programs are available to help individuals navigate insurance coverage and access to treatment.
These support programs may offer assistance with insurance approvals and prior authorizations. Financial counselors at treatment centers may also help eligible individuals identify financial assistance options, including hospital-based financial support programs, grants, or other funding resources.
To learn more about available support services, speak with your healthcare provider or call Rocket Pharmaceuticals at 646-440-9100.
Before Kresladi, your doctor will do an HIV blood test. The test must be negative before your cells can be used to make Kresladi.
If you can become pregnant, your doctor will do a blood pregnancy test. This test must be negative before mobilization. Mobilization is the step that helps move blood-forming stem cells into the blood so they can be collected.
The pregnancy test will be checked again before conditioning and before Kresladi is given.
Vaccines are not recommended during the six weeks before conditioning and until blood counts recover after Kresladi. When possible, childhood vaccines should be given before conditioning.
Kresladi contains dimethyl sulfoxide, also called DMSO. DMSO can cause allergic reactions.
Antiretroviral medicines may interfere with making Kresladi. People should not take antiretroviral medicines for at least one month before mobilization, or for the time needed for the medicine to leave the body, and until all apheresis cycles are finished. Apheresis is the process used to collect blood-forming stem cells.
Before Kresladi, doctors will collect your blood-forming stem cells. These cells are used to make Kresladi. Doctors will also collect and store back-up stem cells in case they are needed.
Kresladi should be avoided in people with active bloodstream infections or other serious, untreated infections.
After Kresladi, some HIV tests may give a false-positive result. This can happen with PCR-based HIV tests. Tell your healthcare team that you received Kresladi before having an HIV test.
People treated with Kresladi should not donate blood, organs, tissues, or cells for transplantation at any time in the future.
Breastfeeding should be stopped during conditioning. Talk with your doctor if you are pregnant, planning to become pregnant, or breastfeeding.
These answers are fact-checked by our editorial staff.
How effective is Kresladi for LAD-I?
Kresladi was studied in nine children with severe leukocyte adhesion deficiency-I, also called LAD-I.
The study measured two markers on white blood cells: CD18 and CD11a. These markers help show whether white blood cells may be able to work better.
CD18 increased in all seven children who could be evaluated for CD18 after treatment. At 12 months, the median CD18 level was 54 percent. At 24 months, it was 50 percent.
CD11a increased in all nine children after treatment. At 12 months, the median CD11a level was 45 percent. At 24 months, it was 39 percent.
How long does Kresladi take to work for LAD-I?
The study does not say exactly when Kresladi starts to work.
In the study, doctors checked CD18 and CD11a levels at 12 months and 24 months after treatment. The increases in these markers lasted through at least month 42 after treatment.
This means the study showed long-term changes in these white blood cell markers, but it did not give an exact earlier time when treatment effects begin.
What precautions and monitoring are needed with Kresladi for LAD-I?
Doctors will monitor people before and after Kresladi.
They will watch for signs and symptoms of infection. They will also check liver tests during the first month after Kresladi to watch for veno-occlusive disease. This is a condition that can affect small blood vessels in the liver.
Doctors will check neutrophil counts until neutrophil engraftment happens. Neutrophil engraftment means the body has enough neutrophils, a type of white blood cell.
Doctors will also check platelet counts and watch for bleeding until platelets recover. Platelets help blood clot.
Long term, doctors will monitor for blood cancers, also called hematologic malignancies. This includes a complete blood count with a differential at least once a year for at least 15 years after Kresladi. Doctors may also do other testing if needed.
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