Stem cell transplantation for the treatment of Fanconi anaemia using a fludarabine-based cytoreductive regimen and T-cell-depleted related HLA-mismatched peripheral blood stem cell grafts

Citation
F. Boulad et al., Stem cell transplantation for the treatment of Fanconi anaemia using a fludarabine-based cytoreductive regimen and T-cell-depleted related HLA-mismatched peripheral blood stem cell grafts, BR J HAEM, 111(4), 2000, pp. 1153-1157
Citations number
18
Language
INGLESE
art.tipo
Article
Categorie Soggetti
Hematology,"Cardiovascular & Hematology Research
Journal title
BRITISH JOURNAL OF HAEMATOLOGY
ISSN journal
0007-1048 → ACNP
Volume
111
Issue
4
Year of publication
2000
Pages
1153 - 1157
Database
ISI
SICI code
0007-1048(200012)111:4<1153:SCTFTT>2.0.ZU;2-9
Abstract
We have employed a new cytoreductive regimen to transplant two patients wit h Fanconi anaemia (FA), using T cell-depleted two HLA-allele disparate rela ted peripheral blood stem cell transplants (PBSCTs). Patient 1, a 5-year-ol d male with FA and aplastic anaemia, initially received an BLA two-antigen mismatched unrelated cord blood transplant acid failed to engraft, lie rece ived fludarabine (Flu) and cyclophosphamide (CY), followed by a CD34(+) E-r osette(-) (CD34(+)E(-)), T cell-depleted, granulocyte colony-stimulating fa ctor (G-CSF)-mobilized PBSCT from his HLA B-DRB1. mismatched father, Be rec eived anti-thymocyte globulin (ATG), steroids, FK506 and G-CSF after transp lant for rejection and graft-versus-host disease (GVHD) prophylaxis, The pa tient is now 23 months after SCT with no evidence of GVHD and with full hae matopoietic and immune reconstitution. Patient 2, a 10-year-old boy with FA and myelodysplastic syndrome, received single-dose total body irradiation (SDTBI), Flu and Cy followed by a CD34(+)E(-), T-cell-depleted, G-CSF-mobil ized PBSCT from his HLA B-DRB1 mismatched sister. He also received ATG, ste roids, FK506 and G-CSF after transplant. The patient is now 12 months after SCT in complete remission with no evidence of GVHD. Absolute neutrophil co unts (ANC) of >1x10(9)/l were achieved on day 11 and day 10 post transplant respectively. Both patients are fully engrafted, In summary, we report two successful T-cell-depleted stem cell transplants from mismatched related d onors for the treatment of Fanconi anaemia, using a fludarabine-based cytor eduction. Both patients experienced minimal toxicity rapid engraftment and no GVHD.