Irradiation‐Free Reduced Intensity Conditioning Stem Cell Transplantation for Young Patients With Diamond‐Blackfan Anemia Syndrome Is Well‐Tolerated and Effective

Hematopoietic stem cell transplantation (HSCT) is a curative option for patients with Diamond‐Blackfan anemia (DBA) syndrome. Myeloablative regimens have generally been standard practice, but reduced intensity conditioning (RIC) regimens are increasingly used to minimize toxicities. We retrospectively analyzed patients with transfusion‐dependent DBA who underwent RIC HSCT with distal alemtuzumab, fludarabine, melphalan, and thiotepa at our institution. Median neutrophil engraftment was Day +15. Maximum acute graft‐versus‐host disease (GVHD) was grade II and chronic GVHD was NIH chronic moderate. All patients remain transfusion‐free with excellent donor chimerism at a median follow‐up of 52 months. HSCT with irradiation‐free RIC was well‐tolerated and effective for DBA.