Dataset from Unrelated Donor Reduced Intensity Bone Marrow Transplant for Children With Severe Sickle Cell Disease (BMT CTN #0601)

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease
that can cause organ damage, stroke, and intense pain episodes. A blood stem cell transplant
is a treatment option for someone with a severe form of the disease. Prior to undergoing a
transplant, people typically receive a conditioning regimen of high doses of chemotherapy and
other medications to prepare the body to accept the transplant. A conditioning regimen that
uses lower doses of chemotherapy and medications may be safer for transplant recipients. This
study will evaluate the safety and effectiveness of blood stem cell transplants, using bone
marrow from unrelated donors, in children with severe SCD who receive a reduced intensity
conditioning regimen prior to the transplant.