Sickle Cell Disease

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A BMT is not a surgery. It is a medical treatment that takes place over several months. It replaces blood stem cells that are damaged, missing or not working with healthy ones.

What is bone marrow?

Bone marrow is the soft, spongy tissue found in bones. It makes blood cells.

  • White blood cells fight infection
  • Red blood cells carry oxygen.
  • Platelets help stop bleeding
  • Blood stem cells make other cells

How does a BMT cure sickle cell disease?

Sickle cell disease is cured when blood stem cells from the healthy donor make red blood cells with normal hemoglobin instead of cells with the hemoglobin-S that can cause sickling.

     
 
BMTforSickleCell

    A BMT is the only cure for children with sickle cell disease. At the Aflac Cancer Center, more than 40 children have been cured of sickle cell disease using BMT. Worldwide, more than 200 sickle cell patients have received matched sibling BMT.

    More than 10 years ago, a BMT cured Tendoh of his sickle cell disease. Meet Tendoh.

 
     

What is the process?

1. Finding a donor

The best donor is a HLA-matched sibling (brother or sister) who does not have sickle cell disease. HLA stands for human leukocyte antigens, which are proteins found on certain cells in the body. HLA is also called transplant tissue typing.

  • Only one in four people have an HLA-matched sibling.
  • If there is not a matched sibling, the chance of complications is higher.
  • The donor does not have to be the same blood type as your child, just the same HLA type.
  • The donor can have sickle cell trait and still donate.
  • Donor blood stem cells may come from stored cord blood, bone marrow or circulating blood.

2. Preparative regimen (treatment plan)

Chemotherapy and other medicines that weaken your child’s immune system are given to prepare his body for the new healthy blood stem cells. The medicines:

  • Destroy your child’s bone marrow cells so they will not reject the new healthy cells after transplant.
  • Cause side effects, such as nausea, vomiting, loss of hair, mouth sores, poor appetite, diarrhea and low blood counts.

3. The BMT

Receiving a BMT is like getting a blood transfusion through the central venous line (CVL). It can take between five minutes and a few hours.

4. After the transplant

Your child:

  • Could stay in the hospital for four to six weeks.
  • May take five to 10 different medicines for many months after leaving the hospital.
  • Will make many visits to the BMT clinic.
  • Will have to stay out of school and follow special infection rules for a time.
  • Will have to get his vaccinations once again since he has a new immune system.

What are the risks?

A BMT is currently the only cure for sickle cell disease. But there are risks:

  • Rejection of the transplant
  • Infection
  • Infertility
  • Neurologic complications, such as stroke or seizure
  • Graft-versus-host disease (GvHD), which is when the donor’s cells attack your child’s body
  • Death

A BMT is also demanding on patients and their families because it is such a long process.

Is my child eligible for a BMT?

Because there are serious risks, BMTs are currently only offered to younger patients with severe sickle cell disease who are at the highest risk for disability or death. The most common reasons for a BMT are:

  • Several episodes of acute chest syndrome
  • Stroke
  • Frequent pain crises

Many severely affected patients do not have a healthy matched-sibling donor. The risks of BMT are higher for these patients, so fewer get transplants from an alternate donor.

Can research help my child?

Research has improved treatment for sickle cell disease. We work with other research centers around the country. We have clinical trials for children who are getting BMTs for sickle cell disease. Ask your child’s team about research.