What is stem cell transplant?

Stem cell transplant is a possible cure for sickle cell disease. It's usually considered only for children younger than 16 who have severe complications from the disease.

Stem cells can be found in bone marrow. Bone marrow is the substance in the center of your bones that produces red blood cells. A person with sickle cell disease has bone marrow that produces red blood cells with defective hemoglobin S. But if that bone marrow is replaced with healthy bone marrow, a person's body may start to produce normal hemoglobin.

Stem cell transplants require bone marrow from another person (donor). This is called an allogeneic stem cell transplant.

Before the transplant, bone marrow stem cells are taken from someone who has closely matching bone marrow. This is most often a healthy brother or sister. The child who has sickle cell disease is then treated with drugs that destroy the child's bone marrow cells. After that, the donated bone marrow stem cells are injected into a vein.

After the process is complete, the donor's bone marrow starts to replace the recipient's bone marrow. These new cells restore the immune system and make normal red blood cells.

Until recently this treatment was mostly done for children. That's because the risk for serious problems increases with a person's age. But new methods of doing stem cell transplant are making it possible to successfully treat adults.

Activity

• Rest when you feel tired. Getting enough sleep will help you recover.
• Try to walk each day. Start by walking a little more than you did the day before. Bit by bit, increase the amount you walk. Walking boosts blood flow and helps prevent pneumonia and constipation.
• Avoid strenuous activities, such as bicycle riding, jogging, weight lifting, or aerobic exercise, until your doctor says it is okay.

Diet

• You can eat your normal diet. If your stomach is upset, try bland, low-fat foods like plain rice, broiled chicken, toast, and yogurt.

Medicines

• Your doctor will tell you if and when you can restart your medicines. You will also get instructions about taking any new medicines.
• If you stopped taking aspirin or some other blood thinner, your doctor will tell you when to start taking it again.
• Be safe with medicines. Take pain medicines exactly as directed.
  • If the doctor gave you a prescription medicine for pain, take it as prescribed.
  • If you are not taking a prescription pain medicine, ask your doctor if you can take an over-the-counter medicine.
• If you think your pain medicine is making you sick to your stomach:
  • Take your medicine after meals (unless your doctor has told you not to).
  • Ask your doctor for a different pain medicine.
• If your doctor prescribed antibiotics, take them as directed. Do not stop taking them just because you feel better. You need to take the full course of antibiotics.

If it works, a stem cell transplant can cure sickle cell disease. This treatment has been successful in about 85 out of 100 children who had transplants. But the risk of dying after a transplant is about 5%.

Procedures can be stressful. This information will help you understand what you can expect. And it will help you safely prepare for your procedure.

Preparing for the procedure

• Understand exactly what procedure is planned, along with the risks, benefits, and other options.
• If you take a medicine that prevents blood clots, your doctor may tell you to stop taking it before your procedure. Or your doctor may tell you to keep taking it. (These medicines include aspirin and other blood thinners.) Make sure that you understand exactly what your doctor wants you to do.
• Tell your doctor ALL the medicines, vitamins, supplements, and herbal remedies you take. Some may increase the risk of problems during your procedure. Your doctor will tell you if you should stop taking any of them before the procedure and how soon to do it.
• Make sure your doctor and the hospital have a copy of your advance directive. If you don’t have one, you may want to prepare one. It lets others know your health care wishes. It’s a good thing to have before any type of surgery or procedure.

• Any stem cell transplant is risky because complications, such as severe infections and immune system problems, can occur.
  • If the recipient's natural defense (immune) system isn't weakened enough by the medicines given before the transplant, it can attack the new stem cells and cause the transplant to fail.
  • If the donor's stem cells don't match the recipient's closely enough, the donor's new immune system cells may attack certain organs in the recipient (graft-versus-host disease).
  • Other complications can include seizures and bleeding in the brain.
• Life-threatening problems can happen. (About 5 out of 100 stem cell transplant recipients die.)
• A person may be unable to have children (infertile) after a stem cell transplant. But newer methods of doing this procedure are reducing this risk.

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.

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.

You may spend up to 4 weeks or longer in the hospital after the transplant. How long you stay depends on whether you got your own stem cells or donor stem cells. It also depends on your health and whether you have problems during or after the transplant.

You'll need constant care for a while after you are home. For the next 6 to 12 months, you'll see your doctor and have your blood tested often. You may get blood transfusions until you can make enough blood cells of your own. If the stem cells came from a donor, your doctor will check for signs that your body is rejecting the cells. Your doctor will want to see you if you have any sign of an infection.

Your doctor will let you know when it's safe for you to go public places where you could be exposed to germs from other people.

Your immune system will need time to get back to normal. It may take several years.

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

Your child will need constant care after going home. Your child may need to stay near the transplant center for the first 3 months. For the next year, your child will see the doctor and get blood tests often. During this time, the doctor may restrict your child from some activities, such as going to school or being in public places, until concerns about infection go away.

Your child may get blood transfusions until your child's body can make enough blood cells on its own. If the stem cells came from a donor, the doctor will check for signs that your child's body is rejecting the cells or that the new cells are attacking other cells in the body (graft-versus-host disease). The doctor will want to see your child if there's any sign of an infection.

Your child's immune system will need time to get back to normal. It may take several years.

• Follow the instructions exactly about when to stop eating and drinking. If you don't, your procedure may be canceled. If your doctor told you to take your medicines on the day of the procedure, take them with only a sip of water.
• Take a bath or shower before you come in for your procedure. Do not apply lotions, perfumes, deodorants, or nail polish.
• Take off all jewelry and piercings. And take out contact lenses, if you wear them.

At the hospital or surgery center

• Bring a picture ID.
• The doctor will put a tube called a catheter into a vein in your chest. The stem cells flow through the catheter into your blood and bone marrow. The stem cells will start to make new cells in 1 to 3 weeks. During this time:
  • You may be kept away from other patients. This is to help prevent infection. You may also get antibiotics to prevent or treat infection.
  • Your blood will be tested often to check the levels of red blood cells, white blood cells, and platelets.
  • You may need several transfusions of blood cells and platelets until your body starts to make its own.
  • You may need to limit visitors and avoid having flowers in your room.
  • You may need to avoid eating certain foods, such as salads.
• The infusion may take several hours.

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.

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.