Relatively mild treatment works for most patients in small study
Sickle cell disease in adults might be treatable with a bone marrow transplant, researchers report July 1 in JAMA.
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. 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.