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.
Bone marrow transplants offer a potential cure for a child's sickle cell disease. They are usually considered only for persons who have:
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.
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.
Tulane University, New Orleans, LA 70118 504-865-5000 firstname.lastname@example.org