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.
| Language | English (US) |
|---|---|
| Pages | 310-317 |
| Number of pages | 8 |
| Journal | Biology of Blood and Marrow Transplantation |
| Volume | 23 |
| Issue number | 2 |
| DOIs | |
| State | Published - Feb 1 2017 |
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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 journal › Article
}
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 -