Bonfim C, Ribeiro L, Nichele S, Loth G, Bitencourt M, Koliski A, Kuwahara C, Fabro AL, Pereira NF, Pilonetto D, Thakar M, Kiem HP, Page K, Fuchs EJ, Eapen M, Pasquini R. Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia. Biol Blood Marrow Transplant. 2016 Nov 7. pii: S1083-8791(16)30484-0.
Bonfim et al, recently reported successful haplo-identical bone marrow transplantation with post-transplant cyclophosphamide in 30 patients with Fanconi anemia (FA). Twenty-six were transplanted upfront and the preparatory regimens included fludarabine 150mg/m2 + total body irradiation 200-300cGy +/- CY 10mg/kg without (n=12) or with rabbit ATG (r-ATG) 4-5mg/kg (n=14). Post-transplant cyclophosphamide was given at a dose of 25mg/kg/day (total dose: 50mg/kg) followed by cyclosporine and mycophenolate mofetil in all of the patients. Hemorrhagic cystitis has been reported to occur in 50% of patients while CMV reactivation occurred in 75%. Importantly, one-year overall survival for the study group was 73% (95% CI: 64-81%) and all surviving patients achieved full donor chimerism. In conclusion haploidentical donor transplantation with post-transplant cyclophosphamide was reported as an alternative for FA paients who had no matched related or unrelated donor.