At Children's National Health System, we offer patients living with sickle cell disease bone marrow transplantation (BMT) as an option for a cure. Past methods of sickle cell disease treatment include: regular health checkups and immunizations, the use of pain relieving medicines and adequate oral or intravenous fluids for patients during pain crises, and blood transfusions and hydroxyurea for children who develop more serious complications. These treatments improve the outcome of children with sickle cell disease, but do not cure them.
BMT is the only treatment that can cure sickle cell disease, but only 20-30 percent of patients who need one have a matching sibling to donate bone marrow for them. BMT has been used to cure a variety of malignant (blood cancers) and immune system diseases for 30 years. For the past 20 years, BMT has been used to treat and cure children with sickle cell disease. Our Sickle Cell team is closely integrated with our BMT team. We have the ability to guide parents through this difficult decision because of the expertise in both the sickle cell program and the BMT division.
How BMT works:
Bone marrow is the organ in which all circulating blood cells, including red blood cells are produced. A child with sickle cell disease produces Hemoglobin S or sickle hemoglobin, which distorts the shape of red blood cells into crescent-shaped, fragile cells that clog small blood vessels, leading to the complications associated with sickle cell disease, like pain crisis, acute chest syndrome, and stroke. Children's National's Division of Blood and Marrow Transplantation is a nationally recognized leader in pediatric blood and marrow treatment advances, known for its cutting-edge treatment protocols developed by Children's physicians. Since 1988, Children's specialists have performed more than 500 BMTs.
What to expect:
Before the BMT procedure, a child with sickle cell disease receives a high dose of chemotherapy to destroy the patient's bone marrow to make room for new bone marrow to grow. Then, the patient receives an infusion of healthy bone marrow containing blood-forming stem cells. The new bone marrow produces healthy red blood cells and resolves the symptoms of sickle cell disease.
The multidisciplinary team of physicians, nurses, pharmacists, social workers, chaplains, and physical therapists help guide families through the process of BMT. The entire BMT procedure requires hospitalization for about 5-6 weeks. Because the procedure is associated with very serious risks, parents can expect to answer a series of questions before enrolling their child to undergo BMT to cure him or her of sickle cell disease.
Finding new treatments:
At Children's National Health System we continue to pioneer curative therapy for sickle cell disease. We collaborate with other major centers focused on sickle cell disease and are involved in national, multicenter clinical research initiatives designed to improve outcomes and expand the use of transplant for sickle cell patients. Areas of investigation include using reduced doses of chemotherapy ("reduced intensity conditioning") to minimize short-term and long-term side effects, and use of matched, unrelated donor bone marrow transplant and cord blood transplant to increase the availability of donor options for those who do not have a matched sibling donor.