The study explores a conditioning regimen using fludarabine, Campath, and low-dose cyclophosphamide (FCC) for allogeneic hematopoietic cell transplants in 15 children with idiopathic severe aplastic anemia (SAA), with added total body irradiation for unrelated donors.
After a median follow-up of 2.3 years, the treatment showed a 100% failure-free survival rate, along with low infection and toxicity rates and no cases of severe acute graft-versus-host disease (GVHD).
All patients exhibited full donor myeloid chimerism post-transplant and demonstrated immune tolerance with stable mixed donor T cell chimerism, indicating a successful