Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia: An update of the Cincinnati Children's experience

Azadeh Farzin, Stella M. Davies, Franklin O. Smith, Alexandra Filipovich, Matthew Hansen, Arleen D. Auerbach, Richard E. Harris

Research output: Contribution to journalArticle

Abstract

Our results for 18 patients undergoing matched sibling donor stem cell transplant for Fanconi anaemia at Cincinnati Children's Hospital Medical Center were published in 1994. The present report updates our results in 35 consecutive patients. Thirty patients transplanted for marrow aplasia received cyclophosphamide 5 mg/kg for 4 d and 400 cGy thoraco-abdominal irradiation. Five patients with clones involving chromosome 7, myelodysplastic syndrome or leukaemia received a more aggressive regimen with total body irradiation. Horse antithymocyte globulin was administered in the pretransplant period to promote engraftment and in the post-transplant period for additional graft-versus-host disease (GVHD) prophylaxis. The median age at bone marrow transplantation was 7·6 years. Median day of engraftment was day +12 (range 9-49), eight patients developed acute GVHD and four chronic GVHD, one limited and three extensive. Twenty-nine of 35 patients (89% actuarial survival at 10 years) had survived with a median follow up of 10·2 years; two children had developed secondary malignancy. All surviving patients had normal blood counts with full donor engraftment. These data indicate excellent long-term outcomes and serve as a reference for newer radiation-free preparative regimes that may reduce the risk of late secondary malignancy.

Original languageEnglish (US)
Pages (from-to)633-640
Number of pages8
JournalBritish Journal of Haematology
Volume136
Issue number4
DOIs
StatePublished - Feb 2007
Externally publishedYes

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Fanconi Anemia
Hematopoietic Stem Cell Transplantation
Siblings
Tissue Donors
Graft vs Host Disease
Transplants
Chromosomes, Human, Pair 7
Antilymphocyte Serum
Whole-Body Irradiation
Myelodysplastic Syndromes
Bone Marrow Transplantation
Cyclophosphamide
Horses
Neoplasms
Leukemia
Stem Cells
Clone Cells
Bone Marrow
Radiation
Survival

Keywords

  • Chimaerism
  • Engraftment
  • Fanconi anaemia
  • Graft-versus-host disease
  • Sibling donor transplant

ASJC Scopus subject areas

  • Hematology

Cite this

Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia : An update of the Cincinnati Children's experience. / Farzin, Azadeh; Davies, Stella M.; Smith, Franklin O.; Filipovich, Alexandra; Hansen, Matthew; Auerbach, Arleen D.; Harris, Richard E.

In: British Journal of Haematology, Vol. 136, No. 4, 02.2007, p. 633-640.

Research output: Contribution to journalArticle

Farzin, Azadeh ; Davies, Stella M. ; Smith, Franklin O. ; Filipovich, Alexandra ; Hansen, Matthew ; Auerbach, Arleen D. ; Harris, Richard E. / Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia : An update of the Cincinnati Children's experience. In: British Journal of Haematology. 2007 ; Vol. 136, No. 4. pp. 633-640.
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