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

Carmem Bonfim, Lisandro Ribeiro, Samantha Nichele, Gisele Loth, Marco Bitencourt, Adriana Koliski, Cilmara Kuwahara, Ana Luiza Fabro, Noemi F. Pereira, Daniela Pilonetto, Monica Thakar, Hans Peter Kiem, Kristin Page, Ephraim J. Fuchs, Mary Eapen, Ricardo Pasquini

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish (US)
Pages (from-to)310-317
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Volume23
Issue number2
DOIs
StatePublished - Feb 1 2017

Fingerprint

Fanconi Anemia
Bone Marrow Transplantation
Cyclophosphamide
Transplants
Antilymphocyte Serum
Rabbits
Unrelated Donors
Graft vs Host Disease
Transplantation
Cystitis
Whole-Body Irradiation
Cytomegalovirus
Cyclosporine

Keywords

  • Fanconi anemia
  • Haploidentical
  • Inherited bone marrow failure syndromes

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia. / Bonfim, Carmem; Ribeiro, Lisandro; Nichele, Samantha; Loth, Gisele; Bitencourt, Marco; Koliski, Adriana; Kuwahara, Cilmara; Fabro, Ana Luiza; Pereira, Noemi F.; Pilonetto, Daniela; Thakar, Monica; Kiem, Hans Peter; Page, Kristin; Fuchs, Ephraim J.; Eapen, Mary; Pasquini, Ricardo.

In: Biology of Blood and Marrow Transplantation, Vol. 23, No. 2, 01.02.2017, p. 310-317.

Research output: Contribution to journalArticle

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 2017, 'Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia' Biology of Blood and Marrow Transplantation, vol 23, no. 2, pp. 310-317. DOI: 10.1016/j.bbmt.2016.11.006

Bonfim, Carmem; Ribeiro, Lisandro; Nichele, Samantha; Loth, Gisele; Bitencourt, Marco; Koliski, Adriana; Kuwahara, Cilmara; Fabro, Ana Luiza; Pereira, Noemi F.; Pilonetto, Daniela; Thakar, Monica; Kiem, Hans Peter; Page, Kristin; Fuchs, Ephraim J.; Eapen, Mary; Pasquini, Ricardo / Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia.

In: Biology of Blood and Marrow Transplantation, Vol. 23, No. 2, 01.02.2017, p. 310-317.

Research output: Contribution to journalArticle

@article{5d34e92ffb6a4b96ac917c5a79d1fc5b,
title = "Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia",
abstract = "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.",
keywords = "Fanconi anemia, Haploidentical, Inherited bone marrow failure syndromes",
author = "Carmem Bonfim and Lisandro Ribeiro and Samantha Nichele and Gisele Loth and Marco Bitencourt and Adriana Koliski and Cilmara Kuwahara and Fabro, {Ana Luiza} and Pereira, {Noemi F.} and Daniela Pilonetto and Monica Thakar and Kiem, {Hans Peter} and Kristin Page and Fuchs, {Ephraim J.} and Mary Eapen and Ricardo Pasquini",
year = "2017",
month = "2",
doi = "10.1016/j.bbmt.2016.11.006",
volume = "23",
pages = "310--317",
journal = "Biology of Blood and Marrow Transplantation",
issn = "1083-8791",
publisher = "Elsevier Inc.",
number = "2",

}

TY - JOUR

T1 - Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia

AU - Bonfim,Carmem

AU - Ribeiro,Lisandro

AU - Nichele,Samantha

AU - Loth,Gisele

AU - Bitencourt,Marco

AU - Koliski,Adriana

AU - Kuwahara,Cilmara

AU - Fabro,Ana Luiza

AU - Pereira,Noemi F.

AU - Pilonetto,Daniela

AU - Thakar,Monica

AU - Kiem,Hans Peter

AU - Page,Kristin

AU - Fuchs,Ephraim J.

AU - Eapen,Mary

AU - Pasquini,Ricardo

PY - 2017/2/1

Y1 - 2017/2/1

N2 - 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.

AB - 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.

KW - Fanconi anemia

KW - Haploidentical

KW - Inherited bone marrow failure syndromes

UR - http://www.scopus.com/inward/record.url?scp=85007414127&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85007414127&partnerID=8YFLogxK

U2 - 10.1016/j.bbmt.2016.11.006

DO - 10.1016/j.bbmt.2016.11.006

M3 - Article

VL - 23

SP - 310

EP - 317

JO - Biology of Blood and Marrow Transplantation

T2 - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

IS - 2

ER -