Boulad, Farid;
Gillio, Alfred;
Small, Trudy N.;
George, Diane;
Prasad, Vinod;
Torok‐Castanza, Joanne;
Regan, Anne D.;
Collins, Nancy;
Auerbach, Arleen D.;
Kernan, Nancy A.;
O'reilly, Richard J.
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
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Media type:
E-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
Contributor:
Boulad, Farid;
Gillio, Alfred;
Small, Trudy N.;
George, Diane;
Prasad, Vinod;
Torok‐Castanza, Joanne;
Regan, Anne D.;
Collins, Nancy;
Auerbach, Arleen D.;
Kernan, Nancy A.;
O'reilly, Richard J.
Description:
<jats:p>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<jats:sup>+</jats:sup> E‐rosette<jats:sup>−</jats:sup> (CD34<jats:sup>+</jats:sup>E<jats:sup>−</jats:sup>), 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<jats:sup>+</jats:sup>E<jats:sup>−</jats:sup>, 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<jats:sup>9</jats:sup>/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.</jats:p>