Transplantation using targeted busulfan for Diamond–Blackfan anemia.

The article presents a case study of a three year old with Diamond–Blackfan anemia (DBA) who underwent allogeneic hematopoietic cell transplantation (HCT). It is reported that the patient received reduced-intensity conditioning with targeted busulfan. It is further reported that the patient achieved successful engraftment, became transfusion-independent, and showed no severe adverse events during the 12-month follow-up.