Milder transplant shows promise for kids with severe blood and immune diseases

This study tests a milder chemotherapy and immune-suppressing regimen before a bone marrow or cord blood transplant in children and young adults (ages 6 months to 25 years) with non-malignant diseases like sickle cell disease, thalassemia, and certain immune deficiencies. The goal is to see if this gentler approach helps the donor cells take hold (engraft) with fewer side effects. While the treatment may control or even eliminate the underlying disease, patients may still need long-term medication to manage their condition or prevent complications.