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.
Stem cell transplant is the replacement of damaged bone marrow cells with healthy cells (stem cells). Stem cells are immature cells produced in the bone marrow that make more stem cells, red blood cells, white blood cells, and platelets.
Stem cells used for transplants can be taken from bone marrow, from the bloodstream, or from umbilical cord blood.
Stem cell transplant is used:
Stem cell transplants that use stem cells donated by someone else are called allogeneic transplants. But when a person's own stem cells are used, it is called an autologous transplant.
The success of a stem cell transplant depends on the person's age and general health condition and whether the donated cells match the body cells. Serious complications that can occur after a stem cell transplant include rejection of the new stem cells, destruction of other cells in the person's body by the new stem cells, or severe, often life-threatening, infection.
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.
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.
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:
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.
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.
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:
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.
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.
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