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Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia.

  • Bonfim, Carmem1
  • Ribeiro, Lisandro2
  • Nichele, Samantha2
  • Loth, Gisele2
  • Bitencourt, Marco2
  • Koliski, Adriana2
  • Kuwahara, Cilmara2
  • Fabro, Ana Luiza2
  • Pereira, Noemi F2
  • Pilonetto, Daniela2
  • Thakar, Monica3
  • Kiem, Hans-Peter4
  • Page, Kristin5
  • Fuchs, Ephraim J6
  • Eapen, Mary7
  • Pasquini, Ricardo2
  • 1 Hospital de Clinicas, Federal University of Parana, Curitiba, Brazil. Electronic address: [email protected] , (Brazil)
  • 2 Hospital de Clinicas, Federal University of Parana, Curitiba, Brazil. , (Brazil)
  • 3 Department of Pediatric Hematology Oncology, Blood and Marrow Transplantation Program, Medical College of Wisconsin, Milwaukee, Wisconsin.
  • 4 Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, Washington.
  • 5 Department of Pediatrics, Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, North Carolina.
  • 6 Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, Maryland.
  • 7 Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin.
Published Article
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Publication Date
Feb 01, 2017
DOI: 10.1016/j.bbmt.2016.11.006
PMID: 27832981


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. Copyright © 2017 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

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