In a phase 1b trial in three patients with Fanconi anemia, an irradiation- and busulfan-free conditioning regimen containing an anti-CD117 antibody, combined with T cell- and B cell-depleted haploidentical hematopoietic stem cell transplantation, was safe and led to high levels of donor engraftment.
- Rajni Agarwal
- Alice Bertaina
- Agnieszka Czechowicz
