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 Journal Article
| Authors: | Boulad, F.; Gillio, A.; Small, T. N.; George, D.; Prasad, V.; Torok-Castanza, J.; Regan, A. D.; Collins, N.; Auerbach, A. D.; Kernan, N. A.; O'Reilly, R. J. |
| Article Title: | 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 |
| Abstract: | We have employed a new cytoreductive regimen to transplant two patients with Fanconi anaemia (FA), using T cell-depleted two HLA-allele disparate related peripheral blood stem cell transplants (PBSCTs). Patient 1, a 5-year-old male with FA and aplastic anaemia, initially received an HLA two-antigen mismatched unrelated cord blood transplant and failed to engraft. He received fludarabine (Flu) and cyclophosphamide (Cy), followed by a CD34 + E-rosette - (CD34 +E -), T cell-depleted, granulocyte colony-stimulating factor (G-CSF)-mobilized PBSCT from his HLA B-DRB1 mismatched father. He received anti-thymocyte globulin (ATG), steroids, FK506 and G-CSF after transplant for rejection and graft-versus-host disease (GVHD) prophylaxis. The patient is now 23 months after SCT with no evidence of GVHD and with full haematopoietic 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-mobilized PBSCT from his HLA B-DRB1 mismatched sister. He also received ATG, steroids, FK506 and G-CSF after transplant. The patient is now 12 months after SCT in complete remission with no evidence of GVHD. Absolute neutrophil counts (ANC) of > 1 × 10 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 donors for the treatment of Fanconi anaemia, using a fludarabine-based cytoreduction. Both patients experienced minimal toxicity, rapid engraftment and no GVHD. |
| Keywords: | child; child, preschool; fludarabine; clinical feature; case report; hypertension; follow up; follow-up studies; t-lymphocytes; cd34 antigen; mucosa inflammation; cyclophosphamide; steroid; stem cell transplantation; hematopoietic stem cell transplantation; hla matching; myelodysplastic syndrome; whole body radiation; peripheral blood stem cell; diabetes mellitus; graft versus host reaction; remission; methylprednisolone; graft rejection; tacrolimus; granulocyte colony stimulating factor; hemorrhagic cystitis; immunosuppressive agents; lymphocyte depletion; vidarabine; thymocyte antibody; aplastic anemia; fanconi anemia; immunoglobulins, intravenous; t-cell depletion; fanconi anaemia; granulocyte colony stimulating factor, recombinant; humans; human; male; priority journal; article |
| Journal Title: | British Journal of Haematology |
| Volume: | 111 |
| Issue: | 4 |
| ISSN: | 0007-1048 |
| Publisher: | John Wiley & Sons |
| Date Published: | 2000-12-01 |
| Start Page: | 1153 |
| End Page: | 1157 |
| Language: | English |
| DOI: | 10.1111/j.1365-2141.2000.02443.x |
| PUBMED: | 11167755 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Export Date: 18 November 2015 -- Source: Scopus |
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