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Bone Marrow Transplant for Sickle Cell Disease

Sickle cell disease is a group of blood disorders that affect hemoglobin in red blood cells. People with sickle cell disease have red blood cells that are hard, sticky, and shaped like a banana (sickle-shaped).

In sickle cell disease, the sickle-shaped blood cells clump together and block tiny blood vessels and slow the flow of blood and delivery of oxygen to the body’s organs. The disease can cause many health problems, such as pain, fatigue, breathing problems, infections, stroke, and organ damage.  

Illustration of normal red blood cells and sickle cells

Sickle-shaped red blood cells can clump together and block the flow of blood.

Treatments for sickle cell disease include a medicine called hydroxyurea and blood transfusions to help manage pain episodes and other health problems. The only potential cures currently available for sickle cell disease are bone marrow transplant (stem cell transplant) and gene therapy. 

What is bone marrow transplant (BMT)?

A bone marrow transplant is a procedure to replace abnormal bone marrow cells with new healthy blood-forming cells. The new cells are taken from a healthy donor, usually a related family member. These new cells will eventually make healthy red blood cells that do not sickle. 

A bone marrow transplant can cure sickle cell disease. But it is a major procedure that takes time and comes with risks. 

Benefits of bone marrow transplant

If the transplant is successful, a bone marrow transplant can cure sickle cell disease.  

Possible benefits of bone marrow transplant for sickle cell disease include: 

  • No more blood transfusions needed 
  • No longer needing medicines used to treat sickle cell disease  
  • Fewer hospital visits over time 
  • Less pain 
  • More energy and better overall health 
  • No further organ damage due to sickle cell disease 

Your care team will discuss your treatment options with you. Treatment options will be based on factors such as your child’s age, severity of sickle cell disease, response to other treatments, and health status.  

Your child’s options will also depend on whether they have a fully matched donor for a bone marrow transplant. Sometimes, a half-matched donor can also be used for a bone marrow transplant. Biological parents are always half a match to their children, and some siblings can also be a half-match. Your physician can discuss all the possible options with you.  

If a suitable donor is not available, gene therapy may be a treatment option. Talk with your care team to see what treatment options are best for your child. 

Bone marrow transplant is a long process and involves several steps. Your family should plan for at least 4-6 months of frequent clinic visits and time in the hospital. Your care team will explain each of the steps and let you know what to expect

Using donor stem cells for transplant

Finding a matched donor  

Several people within your family will have blood tests to learn if their cells could match your child’s. The medical term for this blood testing is HLA typing. 

The care team will test your child, both parents, and all full brothers and sisters. A full brother or sister has the same mother and father. People with sickle cell trait can serve as donors. 

After testing, it takes about 2 weeks to get the results. The chance of a patient with sickle cell disease having an HLA-matched brother or sister who does not have sickle cell disease is less than 20% (2 out of 10). If a full sibling is not a match, the team may discuss other donors for transplant.  

Types of bone marrow donors 

There are 3 main types of bone marrow donors: 

  • Matched related donor: A brother or sister who has the same HLA type and the same mother and father. Full siblings can sometimes be a full match. But they can’t be a donor if they have sickle cell disease. 
  • Matched unrelated donor: A volunteer who has the same HLA type as the patient. Usually, these donors are found through national organizations that match donors and patients who have the same type of HLA. 
  • Haploidentical donor: A half-matched family member, usually a mother, father, or sibling. Biological parents are always a half-match.

Donor health screening 

The donor has pre-transplant evaluation testing including a physical exam, blood work, and other tests. This helps to make sure that their body is healthy enough to donate the stem cells for transplant. 

Collecting donor stem cells for transplant

There are 2 ways to collect stem cells for transplant: bone marrow harvest and apheresis.

Harvesting:

If the cells for transplant are taken from the bone marrow, the collection procedure is called a bone marrow harvest. Bone marrow harvest is usually done in an operating room under general anesthesia. A care provider will insert a hollow needle through the skin into the back of the hip bone and into the bone marrow. The marrow is drawn out with the syringe, then filtered and collected. The needle is inserted several times until enough marrow is collected. Harvesting the marrow usually takes about an hour. 

Bone marrow harvesting illustration

Stem cells for transplant may be collected from the bone marrow.

Apheresis:

If the cells for transplant are taken from blood in a vein, the collection procedure is called apheresis. For several days before apheresis, the donor will get a medicine called granulocyte-colony stimulating factor (G-CSF). G-CSF increases the number of stem cells that circulate in the blood. In apheresis, blood is removed from a vein using a thin, flexible tube (catheter). The blood goes through a machine that removes the stem cells. The remaining blood is then returned to the donor. Apheresis typically takes 4–6 hours. 

A patient in a hospital bed undergoing apheresis

Stem cells for transplant may be collected from blood in a vein.

What to expect during a bone marrow transplant 

  1. 1. Screening and planning

    Illustration of child undergoing screening test

    Your child will have several tests before the bone marrow transplant process begins. Your care team will help you plan and know what to expect.

    Your child will need several tests and scans before the transplant process begins. The care team can start planning for this testing after they identify a donor. 

    Tests may include imaging tests, blood tests, and tests to measure heart, lung, kidney, and brain function. Testing before the transplant takes about 2-3 weeks. You can expect to spend several hours at your care center each day for these tests. 

    When the tests are complete, you will meet with members of your care team to review the transplant information and make sure you understand it. Be sure to ask any questions that you have. Then, you will sign a consent form that allows your child to undergo the transplant. You will also review plans for your child’s hospital stay with a nurse or coordinator. Your child will stay in the hospital for at least 5–6 weeks, so planning that stay is important. 

    Some of the treatments used in the transplant process can affect your child’s fertility, or their ability to get pregnant or father a child in the future. Your care team will discuss options to preserve your child’s fertility before the transplant.  

    Your child will have a procedure to place a central line, such as a PICC line or tunneled central line. The central line will allow your child to receive chemotherapy and other medicines and have blood tests with fewer needle sticks.  If your child has a line already, the care team may replace it with a different central line. 

  2. 2. Conditioning treatment (chemotherapy)

    Female child receiving chemotherapy

    Your child will get chemotherapy medicines to help prepare their body for the stem cells. This is called conditioning, the preparative regimen, or “prep.”

    To prepare for the transplant, your child will get chemotherapy medicines. This is called conditioning, the preparative regimen, or “prep.”  

    Conditioning helps your child’s body get rid of the old stem cells to make room for the donor cells that will grow and make healthy blood cells. It also weakens the immune system so that your child’s body does not reject the donor cells. Conditioning will take about 1-2 weeks. 

    Your care team will give you information about the chemotherapy, so you and your child know what to expect. Your child must complete their chemotherapy before they can receive the donor cells. 

    Your child will be admitted to the hospital for the conditioning treatment. They will remain in the hospital for at least 4-6 weeks until after the donor cell infusion (transplant) and engraftment when the body starts making healthy red blood cells. 

  3. 3. Transplant

    3. Transplant

    The donor cells are given to your child through their central line. This is called an infusion. The infusion takes about 1 hour.

    Several nurses and other staff will be in the room to monitor your child for signs of a reaction to the infused cells. A parent or family caregiver may stay with your child during the infusion.  

    The day of the transplant is sometimes called “Day 0.”  

    Stem cell infusion

    After conditioning is complete, your child will get the stem cells through an infusion.

  4. 4. Engraftment

    4. Engraftment

    The next step is waiting for the donor cells to grow and multiply in your child’s body. This is called engraftment. Engraftment takes several weeks. Your child’s body will start making healthy cells. In time, your child’s new bone marrow cells will make red blood cells that are healthy and round. 

    Your child will have regular blood tests to see if the new cells are working correctly. 

    Your child will remain in the hospital until engraftment occurs. Then, your child will be discharged to home or housing. 

    Engraftment

    If the transplant is successful, the stem cells will grow and multiply. Then, your child’s body will start making healthy red blood cells.

After the transplant

Patients are closely monitored for the first 100 days after transplant. Your child will stay in local or long-term housing for several months after hospital discharge. They may need to stay longer if there are problems. 

During this time, your child will have regular outpatient clinic visits and lab tests several times a week to check their progress. After the transplant, your child will have fewer sickle cells and more healthy red blood cells in their blood.

Your child will have routine appointments as part of long-term follow-up care. Your child will have fewer checkups as they get older if they are doing well. Follow-up care continues for several years.

Possible risks of bone marrow transplant

Side effects of chemotherapy 

Most patients who receive chemotherapy have some degree of side effects. These may include:  

Your care team will take steps to help manage side effects, including giving medicines to help control nausea, vomiting, and pain. Red light therapy may be used to prevent and treat mouth sores (oral mucositis). Your child may also need a nasogastric (NG) tube to give them food (nutrition).  

Because of the increased risk of infection, your child will stay in their room on the transplant unit for their inpatient stay. All caregivers and visitors must follow strict guidelines for infection prevention.  

Low blood counts 

The bone marrow transplant process may cause your child to have low blood counts. Blood transfusions may be needed to restore platelets and red blood cells.  

Graft failure 

There is a small chance that the new bone marrow from a matched family member will not work. The chance of this happening is higher with half-matched or matched unrelated donors. If this happens, your child will not be able to make white blood cells, red blood cells, or platelets, and the transplant would need to be done again.  

Sometimes, the recipient’s own cells can grow back, which means that sickle cell disease could return.  

Graft rejection 

Graft rejection is a type of graft failure. Although uncommon, graft rejection happens when your child’s immune system recognizes the donor cells as being different and destroys them.  

Patients who experience graft rejection can become quite ill. To prevent graft rejection, your child will get chemotherapy with or without radiation to destroy their immune system before the transplant happens. If your child experiences graft rejection, another transplant will be needed. 

Graft-versus-host disease (GVHD) 

Graft versus host disease is when the immune cells of the donor (graft cells) sense that the cells of your child (host cells) are different and attack them. It can occur early or late in the process, and symptoms can range from mild to severe.   

GVHD symptoms include: 

  • Rash 
  • Nausea and vomiting 
  • Diarrhea 
  • Yellow skin and eyes 
  • Scaly skin 
  • Darkening of skin 
  • Hardening of skin texture 
  • Skin scarring/restriction of joints 
  • Dryness and sores in the mouth and esophagus 
  • Dry eyes and redness in the eyes 
  • Dryness of the vagina and other skin surfaces 
  • Drying and scarring of lungs 
  • Liver injury or liver failure 

Your child will get immunosuppressant medicines to help prevent GVHD. It is very important that patients take all medicines exactly as prescribed. 

Veno-occlusive disease (VOD) 

Veno-occlusive disease (VOD) occurs when blood clots form in blood vessels in and around the liver. Bone marrow transplant, certain chemotherapy medicines, and frequent blood transfusions can increase the risk of VOD. VOD can cause increased bilirubin levels, an enlarged liver, water buildup in the body, or weight gain. If not treated, VOD can lead to organ failure and even death.  

VOD is a serious condition. If your child develops VOD, they may need to be treated in the intensive care unit (ICU). VOD treatment often includes limiting fluids and treatment with the medicine defibrotide.  

To help prevent VOD, your child may get the medicine ursodiol before hospital admission. Your care team will also monitor your child’s weight and fluid balance while they are in the hospital.  

Social and emotional concerns 

A bone marrow transplant is hard for both the patient and their family. Your child’s daily life will be different for a while since they will be away from home, school, friends, and family.  

The family plays a big role in supporting your child during this time. On average, your child will stay in the hospital for 4–6 weeks. If you live far from the care center, you will need to stay nearby for at least 100 days or longer, depending on how well your child is doing after the transplant. Regular check-ins with a psychologist, social worker, and other care team members will help in navigating the transplant process.  

Infertility  

Some people who have bone marrow transplant may not be able to have children on their own. Chemotherapy can affect the ability to have children later in life. If your child is old enough, the care team may be able to collect their sperm or eggs before treatment so that they can be frozen and stored to use later if they choose. In some cases, ovarian or testicular tissue can be taken out and stored for future use.   

Talk to your care team about your child’s risk of infertility and fertility preservation options that may be available. 

Learn more about male and female fertility.

Bone marrow transplant is a potential cure for sickle cell disease. But it only treats your child’s blood. Your child can still pass the gene for sickle cell disease on to their children. 

Talk with your care team to find out if bone marrow transplant might be an option for your child.

Questions to ask your care team

  • Is my child a good candidate for bone marrow (stem cell) transplant?  
  • Does a bone marrow transplant cure sickle cell disease?  
  • What other treatments could help manage my child’s sickle cell disease?  
  • What are the potential benefits of bone marrow transplant compared to other treatments?  
  • What are the short-term and long-term risks or side effects?  
  • How long does the whole transplant process take?  
  • What type of school or work support might be helpful? 
  • What kind of follow-up care and monitoring will my child need?  

Key points about bone marrow transplant for sickle cell disease

  • A bone marrow (stem cell) transplant replaces a patient’s blood-forming stem cells with healthy stem cells.  
  • A bone marrow transplant may cure your child’s sickle cell disease. 
  • Your child will be in the hospital for 4–6 weeks or longer. A family caregiver must stay with your child.  
  • It takes 2–4 weeks for new cells to start growing. This is called engraftment.  
  • It will take up to 1 year for your child’s immune system to return to normal.  
  • Your care team can help you know what to expect during the bone marrow transplant process. 
  • Your care team can help determine if bone marrow transplant is a good treatment option for your child and let you know what to expect. 

Related Factsheets

—
Reviewed: February 2026

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