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Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita.

Abstract
Since the results of conventional hematopoietic stem-cell transplantation (HSCT) for patients with dyskeratosis congenita (DC) are poor owing to the high incidence of transplant-related complications, we explored the use of a low-intensity HSCT regimen. We report two children with DC with severe cytopenia, who underwent successful HSCT from a matched unrelated donor after conditioning with fludarabine, cyclophosphamide, and antithymocyte globulin. Graft-versus-host-disease (GVHD) prophylaxis consisted of corticosteroids and cyclosporin A. The regimen was well tolerated, no significant transplant-related complications were observed, and engraftment was rapid and complete. At 15 and 16 months after HSCT, the children were fully engrafted, in excellent clinical condition, full-donor chimerism, and no signs of GVHD. We conclude that a low-intensity regimen is sufficient to induce durable engraftment using matched unrelated donor HSCT in DC patients, with minimal 1-year transplant-related toxicity. Longer follow-up will determine whether this regimen also reduces long-term toxicity.
AuthorsY Dror, M H Freedman, M Leaker, J Verbeek, C A Armstrong, F E Saunders, J J Doyle
JournalBone marrow transplantation (Bone Marrow Transplant) Vol. 31 Issue 10 Pg. 847-50 (May 2003) ISSN: 0268-3369 [Print] England
PMID12748659 (Publication Type: Case Reports, Journal Article, Review)
Chemical References
  • Adrenal Cortex Hormones
  • HLA Antigens
  • Immunosuppressive Agents
  • Cyclosporine
Topics
  • Adrenal Cortex Hormones (therapeutic use)
  • Adult
  • Child, Preschool
  • Cyclosporine (therapeutic use)
  • Dyskeratosis Congenita (therapy)
  • Female
  • Graft vs Host Disease (immunology, prevention & control)
  • HLA Antigens (immunology)
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents (therapeutic use)
  • Male
  • Stem Cell Transplantation (methods)

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