What is stem cell transplant?

Stem Cell Transplant

What is a stem cell transplant in children?

A stem cell transplant replaces damaged stem cells with healthy ones. Stem cells are special cells made in the bone marrow. They become red blood cells, white blood cells, platelets, and more stem cells. The procedure is sometimes called a bone marrow transplant.

A stem cell transplant may be needed if your child has a disease such as leukemia, lymphoma, or some kinds of blood or bone marrow diseases.

Having the transplant is a serious decision that you and your child's treatment team will make. The procedure is expensive and risky. It's a long and often painful process. Success depends on the type and stage of your child's disease and your child's age and general health. A transplant doesn't work for every child who gets one. But when it works, it can increase the chances of remission or cure of the disease.

Why is a stem cell transplant for sickle cell disease done?

Stem cell transplants offer a potential cure for a child's sickle cell disease. They are usually considered only for children younger than 16 who have:

  • Severe sickle cell disease complications, including repeat strokes, episodes of acute chest syndrome, and painful events.
  • An available donor (someone who has closely matching bone marrow).

The risks of stem cell transplant become greater as a person gets older and/or develops damage to major organs. For these reasons, a bone marrow transplant is not a treatment option for most adults who have sickle cell disease. But research on bone marrow transplants in adults is ongoing.

How is a stem cell transplant done in children?

A stem cell transplant is done in several stages. Parts of the treatment may be done in a hospital. Others may be done in an outpatient center. How long your child's treatment takes will depend on your child's disease, how your child responds to the treatment, and any problems your child may have from the treatment. The doctor can tell you more about this.

Testing

Your child will first have tests to make sure that your child's health is good enough for the chemotherapy (chemo) and radiation that are part of the procedure. This may take a few days to several weeks.

Deciding on treatment

You and the doctor will look at your child's test results and discuss the physical and emotional challenges your child will face. Based on these things, you might go ahead with the transplant. Or you might choose some other treatment instead.

There are three types of stem cell transplants:

  • The stem cells come from your child's own body (autologous). Your child's blood is sent through a machine that separates stem cells from the blood. The cells are stored until your child needs them for transplant.
  • The stem cells come from a donor (allogeneic). The donor may take medicine to help make more stem cells. Then the stem cells are taken from the donor's blood or bone marrow and given to your child.
  • The stem cells come from umbilical cord blood. The umbilical cord is usually thrown away after birth. But the blood inside the cord can be saved, or banked, to be used later if needed. The cord blood can come from one or more donors. Or it can come from your child if you banked the cord blood when your child was born.

Conditioning

Your child will have chemo and radiation to kill any remaining cancer or bone marrow cells and to prepare your child's body for the new stem cells. This can take 1 to 2 weeks. Your child may have side effects, such as mouth sores, nausea, hair loss, and poor appetite. The side effects may last several weeks to months. But the doctor can give your child medicine to help ease them.

Transplant

After the damaged blood cells are destroyed, the healthy stem cells are put into your child's bloodstream. This is called infusion. It usually takes several hours. The infused stem cells travel through the blood to your child's bone marrow. They will start to make healthy blood cells in 1 to 4 weeks.

Your child may spend up to 4 weeks or longer in the hospital after the transplant.

The chemo and radiation destroy your child's white blood cells. Without them, your child's body can't fight infection. The doctor may give your child medicine to help protect your child from getting an infection while the body is making new white blood cells. Caregivers, family, and other visitors may need to take extra steps during this time to protect your child from infection.

Who is a good candidate for a stem cell transplant?

Your doctor will consider your health and your age. People who are good candidates for a stem cell transplant usually are younger than 70, do not have other diseases such as heart disease or diabetes, and have a normal kidney and liver. Your doctor will also consider how much your disease has grown and how aggressive your cancer is. People with aggressive cancer that has spread to many areas of the body usually aren't good candidates. Your doctor may also consider if you have cancer that has come back, such as relapsed non-Hodgkin lymphoma or leukemia.

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