Favourable long-term outcome after matched sibling transplantation for fanconi-anemia (FA) and in vivo T-cell depletion

K. Huck, H. Hanenberg, W. Nürnberger, D. Dilloo, S. Burdach, U. Göbel, H. J. Laws

Research output: Contribution to journalArticle

8 Scopus citations

Abstract

Hematopoietic stem cell transplantation is the only permanent treatment for the hematological manifestations in Fanconi anemia (FA). As FA patients have a dramatically increased intrinsic propensity to develop malignancies later in life and the genotoxic stress afflicted during conditioning advances the manifestation age especially of squamous cell carcinomas, choosing an optimally suited treatment regimen appears critical for long-term, tumor-free survival after stem cell transplantation. Here, we report our experiences in 6 consecutive FA patients transplanted with HLA-matched sibling donors where we combined an established pre-transplantation treatment consisting of thoraco-abdominal irradiation (TAI), cyclophosphamide (CYC) and cyclosporine A graft-versus-host prophylaxis with antibody-mediated in vivo T-cell depletion strategies after infusion of the graft. This approach has ensured sustained engraftment with long-term survival and an excellent post transplant performance status without any evidence of secondary malignancies in all six patients after a median follow-up of more than 10 years.

Original languageEnglish (US)
Pages (from-to)147-152
Number of pages6
JournalKlinische Padiatrie
Volume220
Issue number3
DOIs
StatePublished - May 1 2008

Keywords

  • CD25 monoclonal antibody
  • Fanconi anemia
  • GvHD
  • Secondary malignancies
  • Stem cell transplantation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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    Huck, K., Hanenberg, H., Nürnberger, W., Dilloo, D., Burdach, S., Göbel, U., & Laws, H. J. (2008). Favourable long-term outcome after matched sibling transplantation for fanconi-anemia (FA) and in vivo T-cell depletion. Klinische Padiatrie, 220(3), 147-152. https://doi.org/10.1055/s-2008-1065326