BMT for Sickle Cell Disease

At the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, 73 children have been cured of sickle cell disease through blood and marrow transplant (BMT). As one of the leading pediatric BMT programs in the U.S., we strive to improve and enhance the quality of life for children and young adults with sickle cell disease by offering BMT as a cure.

How does a BMT cure sickle cell disease?

Bone marrow is the soft, spongy tissue found in bones that makes red blood cells. Hemoglobin is a protein in red blood cells that helps move oxygen through the body.

Blood stem cells that come from a healthy donor make red blood cells with normal hemoglobin while a child with sickle cell disease makes hemoglobin-S. Hemoglobin S can cause red blood cells to be crescent-shaped allowing them to get stuck in blood vessels and block the flow of blood. This leads to sickle cell complications, such as pain crisis, acute chest syndrome and stroke.

When a child with sickle cell disease undergoes a BMT, he receives healthy bone marrow that produces normal red blood cells.

BMT process

HLA typing and finding a donor

  • The blood test will be done on your child and his brothers and sisters who have the same parents. These are called full siblings.
  • HLA matching improves your child’s chances of a successful transplant.

The best donor is an HLA-matched sibling who does not have sickle cell disease. Your child’s full siblings each have a 25 percent chance of matching. If your child does not have a matched sibling, the chance of complications is higher.

  • The donor does not have to be the same blood type or gender as the patient, just the same HLA type.
  • A donor can have sickle cell trait.
  • Donor blood stem cells may come from stored cord blood, bone marrow or circulating blood.

Preparative regimen (treatment plan)

Your child will take medicine to prepare his body for the healthy, new blood stem cells. The medicines:

  • Help his body accept healthy cells during a transplant
  • Can be given to your child in different ways, such as by intravenous (I.V.) line or mouth
  • May cause side effects like: nausea, vomiting, hair loss, mouth sores, poor appetite, diarrhea and low blood counts

The transplant

The transplant will be given to your child through a central venous line (CVL). It can take five minutes or several hours.

After the transplant

After receiving a BMT, your child will:

  • Stay in the hospital for several weeks
  • Be watched closely by his doctor
  • Take many different medicines
  • Follow special rules to prevent infection
  • Stay home from school for a while
  • Need vaccinations or shots to prevent infection
  • Need a full-time caregiver
  • May still pass on the hemoglobin-S gene

BMTs are usually given to patients with severe sickle cell disease because they are at the highest risk for disability or death. For a patient who does not have a healthy matched-sibling donor, the risk of BMT complications is higher. Therefore, fewer of these children are candidates for BMTs.

Your child may qualify for a BMT if he has:

  • Several episodes of acute chest syndrome
  • Stroke
  • Severe and/or frequent pain

What are the risks?

The risks of BMT include:

  • Rejection of the transplant if your child’s body does not accept the new cells
  • Infection
  • Infertility
  • Stroke or seizure
  • Graft-versus-host disease (GVHD) (when the donor’s cells attack your child’s body)
  • Death

A BMT is hard on patients and their families because it is such a long process. Our team of professionals will work to support your family.

Can research help my child?

Every advancement in curing sickle cell disease is the result of research. At the Aflac Cancer and Blood Disorders Center, we are committed to excellence and innovation in pediatric BMT and sickle cell research. We work with other research centers around the country and have clinical trials for children who are getting BMTs for sickle cell disease.

Find a clinical trial

Learn more about our research efforts