Bone Marrow Transplant Gives Hope To Adult Sickle Cell Patients

Sickle cell disease in adults might be treatable with a bone marrow transplant, researchers reportJuly 1 in JAMA. The researchers and others say the findings show age need not be a barrier and that the technique may change practice for some adult patients when standard treatment fails. “We’re very pleased,” said Dr. John Tisdale, the study’s senior author and a senior investigator at the National Institutes of Health. “This is what we hoped for.” The treatment is a modified version of bone marrow transplants that have worked in kids. Donors are a brother or sister whose stem cell-rich bone marrow is a good match for the patient. Hundreds of children with sickle cell disease had previously been treated and most cured with the procedure, which requires that their defective bone marrow be largely wiped out by radiation and chemotherapy and then replaced by a closely matched marrow transplant, but, until recently, the harsh regimen was considered too risky for adults because they often have accumulated organ damage from sickle cell disease. In a test of 30 adult patients, 29 have now survived for an average of 3.4 years after undergoing a mild course of radiation and chemo before getting a marrow transplant. The replacement cells act as starters that grow into normal blood without sickle-shaped deformities, which cause pain, anemia, blood flow problems and a need for blood transfusions. The mild course of radiation and chemo partially wiped out defective marrow, leaving the patients with a mixed batch of marrow cells. The procedure failed to fully replace the abnormal cells in four of the patients, and a fifth died of bleeding in the brain 10 months after transplant. But one year after transplant, the other 25 patients had fully functioning blood cells, and 15 have since stopped taking immune-suppressing medication, according to the study coauthors at the National Institutes of Health and Johns Hopkins University