Bone Marrow Transplants for Sickle Cell Disease

A bone marrow transplant is the only way to cure sickle cell disease, but it’s not a simple process. If you’re thinking about a transplant for yourself or a child, here are some things to know.

How Does It Work?

A bone marrow transplant replaces the cells in your body that make red blood cells, called hematopoietic stem cells, with new ones. That means your body will stop making the sickle-shaped cells that cause the disease.

In the procedure, a doctor takes healthy stem cells from the bone marrow of a donor and injects them into your body, usually through an IV tube into one of your veins. Once inside, the cells go to your bone marrow and start creating healthy blood cells.

While that sounds simple, a bone marrow transplant is a long process. Once you have a donor, you’ll spend several weeks in the hospital and have several more months of follow-up care. The process begins before the actual transplant procedure:

  • For 1 to 2 weeks before the transplant, you’ll stay in the hospital, and doctors will give you chemotherapy. These powerful medicines destroy the cells that make abnormal blood cells. They also make your immune system weak, so it won’t reject and attack the new stem cells. You might get radiation therapy as well.
  • Then, doctors will inject the donor’s cells into your body. The cells should replace the old bone marrow and start making new, healthy blood cells. Your care team will run tests for about a month to make sure the new cells start to work.
  • Once doctors can tell that the transplant has worked, you can leave the hospital. It may take 6-12 months or longer before your blood cells and immune system get back to normal. Your doctor will watch your health closely during this time.

Finding a Donor

People with severe sickle cell disease -- who have had a lot of complications or episodes of pain -- are the most likely candidates for a bone marrow transplant. Doctors will have to make sure you or your child is healthy enough to have this procedure. An interview with a psychologist or social worker can also help doctors know if you’re mentally ready for the process.

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Doctors need to find a donor whose bone marrow matches yours. This can be one of the biggest challenges in the process.

Blood tests will tell doctors whether a brother, sister, or parent has matching bone marrow. Between 20% and 30% of children who need a transplant will have a sibling whose bone marrow matches theirs.

You can also look for a donor in a national registry of people who have volunteered to be tested. If you saved your child’s umbilical cord blood after she was born, doctors may also be able to take stem cells from it.

What Are the Risks?

Like any other major operation, a bone marrow transplant comes with a chance of complications and setbacks. The risks include:

  • Rejection, which happens when the body turns against the new cells. That’s known as graft-versus-host disease (GVHD). It happens in about one in 10 cases. You can take medicines to treat or prevent it. But if the drugs don’t work, GVHD can damage your organs or cause death.
  • Infection, since treatment before the transplant makes the body’s immune system weaker. Medicines can help prevent bacteria or viruses from setting in while you or your child is going through the process.
  • Chemotherapy before the transplant may cause nutritional problems if it makes you lose your appetite or causes diarrhea or vomiting.
  • Damage to blood vessels in the liver, called veno-occlusive disease. Severe damage happens in about 1 out of 20 people.
  • Infertility. Most people won’t be able to have children after they’ve had a bone marrow transplant, usually because of the drugs you take before the procedure.

What if It Doesn’t Work?

In about nine out of 10 cases, a transplant will result in new, healthy blood cells and no more sickle cell disease.

But if a transplant fails, doctors will have to repeat the procedure to try to cure you. Or they’ll have to inject your own stem cells back into your body -- which means sickle cell disease will return.

WebMD Medical Reference Reviewed by Melinda Ratini, DO, MS on March 06, 2018

Sources

SOURCES:

CDC: “Sickle Cell Disease - data and statistics.”

Journal of Blood Medicine: “Hematopoietic stem cell transplantation in sickle cell disease: patient selection and special considerations.”

Children’s National Health System: “Stem Cell Transplant -- A Step-by-Step Timeline,” “Blood and Marrow Transplant for Sickle Cell Disease.”

St. Jude Children’s Research Hospitals: “Bone Marrow (Stem Cell) Transplant for Sickle Cell Disease.”

National Heart, Lung, and Blood Institute: “Blood and Bone Marrow Transplant.”

National Marrow Donor Program: “Sickle Cell Disease.”

Pediatric Blood Cancer: “Allogeneic hematopoietic stem cell transplantation in children with sickle cell disease.”

News release, National Institutes of Health: “Stem Cell Transplant Reverses Sickle Cell Disease in Adults.”

Therapeutic Advances in Hematology: “Hematopoietic stem-cell transplantation for sickle cell disease: current evidence and opinions.”

Current Opinion in Oncology: “Blood and marrow transplantation for sickle cell disease: Overcoming barriers to success.”

American Cancer Society: “What’s It Like to Get a Stem Cell Transplant?”

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