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.