Bone Marrow Transplant for Sickle Cell Disease

Treatment Overview:

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 abnormal sickle shape.   However, if that bone marrow is replaced with healthy bone marrow, a person's body may start to produce normal hemoglobin. Bone marrow transplants require bone marrow from another person (donor) and carry a high degree of risk. They can be used for children with severe sickle cell disease for children or young adults who have only minimal organ damage due to the disease.

Before the transplant, bone marrow stem cells are taken from someone who has closely matching bone marrow, usually a healthy brother or sister. If a child does not have a sibling, we can try and identify a match through the national bone marrow donor program (NMDP).  The patient who has sickle cell disease is then treated with chemotherapy (drugs that destroy his or her bone marrow cells) to make room for new bone marrow. After that, the donated bone marrow stem cells are injected into a vein.

After the process is complete, the donor's bone marrow begins to replace the recipient's bone marrow. These new cells restore the body's immune system and can now produce normal red blood cells.

How Long Does the Transplant Take?

Patients undergoing bone marrow transplant usually remain in the hospital for 4-8 weeks.  These patients remain in specialized room in the bone marrow transplant unit designed to prevent infection.  They must remain in this unit throughout their hospitalization.  For children, we allow a parent to stay with them in their room.  It gives the person time for their immune system to start working again.  

During recovery, doctors watch closely for signs that the immune system is rejecting the new bone marrow and for signs of infection. If a problem occurs, recovery can take longer or the transplant may fail to work.

Why Is It Done?

Bone marrow transplants offer a potential cure for a child's sickle cell disease. They are usually considered only for persons who have:

• Severe sickle cell disease complications, including repeat strokes, episodes of acute chest syndrome or life-limiting painful events.
• Children identified early with signs and symptoms of severe sickle cell disease (such as splenic sequestration, dactylitis, osteomyelitis)
• An available donor (someone who has closely matching bone marrow).
• No significant damage to major organs.

How Well It Works

If successful, a bone marrow transplant can cure sickle cell disease. Although only used in select cases, it is successful in nearly 85% of transplant recipients. Over 90% of patients survive, despite transplant complications and/or failure (when the new bone marrow does not grow). About 5% percent of bone marrow transplant recipients die following the procedure.

Risks

• Any bone marrow transplant is risky because complications, such as severe infections and immune system problems, can occur. 
•  If the recipient's natural defense (immune) system isn't weakened enough by the medicines given before the transplant, it can attack the new stem cells and cause the transplant to fail.
• If the donor's stem cells don't match the recipient's closely enough, the donor's new immune system cells may attack certain organs in the recipient (graft-versus-host disease).
• Other complications can include seizures although we give medicines to prevent these
• Rare complications can cause bleeding in the brain.
•  In some cases, this disease can cause long-term sickness or death (5% of bone marrow transplant recipients die).1 
• A person may be unable to have children (infertile) after a bone marrow transplant.

What To Think About

• Transplanted bone marrow doesn't always work properly. This can cause life-threatening problems.
• Bone marrow transplant is still an experimental treatment for people with sickle cell disease although this number is increasing.
• Very few hospitals offer this procedure.
• The risks of bone marrow 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.
• About 1 out of 10 people with sickle cell disease have a matching donor

TULANE OFFERS BMT FOR SICKLE CELL DISEASE:

Patients being seen at the Sickle Cell Center for Southern Louisiana may have the option to undergo bone marrow transplant.  If interested, please contact the center at 504-988-5413 and ask for Dr. Julie Kanter.  This procedure is reserved for patients with severe disease.  If you believe you or your child could benefit from this treatment, please inquire.  In addition, we will have available research protocols open for bone marrow transplant in the coming months.