Bone marrow failure syndrome

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This study tested the drug eltrombopag in 34 people with moderate aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. Eltrombopag is a pill that mimics a natural protein to stimulate blood cell production. Researchers measured whether it safely imp…

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…

This study tested a drug called eltrombopag in 118 people with bone marrow failure who had a stem cell transplant. The goal was to see if the drug could help their platelet levels recover faster after the transplant. Platelets are blood cells that help stop bleeding, and slow rec…