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Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia. Biol Blood Marrow Transplant 2017 Feb;23(2):310-317



Pubmed ID




Scopus ID

2-s2.0-85007414127 (requires institutional sign-in at Scopus site)   45 Citations


We describe haploidentical bone marrow transplantation with post-transplant cyclophosphamide (PT-CY) for 30 patients with Fanconi anemia (FA). Twenty-six patients were transplanted upfront, and the preparatory regimens included fludarabine 150 mg/m2 + total body irradiation 200 to 300 cGy ± CY 10 mg/kg without (n = 12) or with rabbit antithymocyte globulin (r-ATG) 4 to 5 mg/kg (n = 14). Four patients were rescued after primary or secondary graft failure after related or unrelated donor transplantation with the above regimen with (n = 2) or without r-ATG (n = 2). PT-CY at 25 mg/kg/day (total dose, 50 mg/kg) followed by cyclosporine and mycophenolate mofetil was given to all patients. All patients engrafted in the subgroup of patients who did not receive r-ATG (n = 14), but their transplant course was complicated by high rates of acute and chronic graft-versus-host disease (GVHD), and only 8 patients are alive. In the subgroup that received r-ATG (n = 16), 14 patients had sustained engraftment, severe GVHD rates were lower, and 13 patients are alive. Hemorrhagic cystitis occurred in 50% of patients, whereas cytomegalovirus reactivation occurred in 75%. One-year overall survival for the entire cohort was 73% (95% CI, 64% to 81%), and all surviving patients achieved full donor chimerism. In conclusion, haploidentical donor transplantation with PT-CY is a suitable option for FA patients without a matched related or unrelated donor.

Author List

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


Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Bone Marrow Transplantation
Child, Preschool
Cytomegalovirus Infections
Epstein-Barr Virus Infections
Fanconi Anemia
Graft Survival
Graft vs Host Disease
Immunosuppressive Agents
Kaplan-Meier Estimate
Leukocyte Count
Primary Graft Dysfunction
Retrospective Studies
Salvage Therapy
Tissue Donors
Transplantation Conditioning
Virus Activation