Gentler stem cell transplant using a mismatched family donor for blood disorders

This trial is testing a new way to do bone marrow transplants for children with serious blood disorders like sickle cell disease or bone marrow failure. Instead of using the strongest conditioning medicines, doctors will use a gentler preparation regimen—including hydroxyurea, alemtuzumab, fludarabine, thiotepa, and melphalan—followed by a transplant from a family member's bone marrow that doesn't have to be a perfect match. The main goal is to make sure the treatment is safe and that the new bone marrow cells successfully take root in the patient's body.

Many children with serious blood disorders could be helped by bone marrow transplants, but finding a perfectly matched donor is difficult, and the strongest conditioning medicines can be very hard on young patients. This trial is testing whether a gentler approach using a mismatched family member's bone marrow can work, giving more children access to this potentially life-changing treatment with fewer side effects.