Pediatr Blood Cancer. 2025 Nov 19:e70007. doi: 10.1002/pbc.70007. Online ahead of print.
ABSTRACT
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.
PMID:41257311 | DOI:10.1002/pbc.70007
