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.
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Authors | Y Dror, M H Freedman, M Leaker, J Verbeek, C A Armstrong, F E Saunders, J J Doyle |
Journal | Bone marrow transplantation
(Bone Marrow Transplant)
Vol. 31
Issue 10
Pg. 847-50
(May 2003)
ISSN: 0268-3369 [Print] England |
PMID | 12748659
(Publication Type: Case Reports, Journal Article, Review)
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Chemical References |
- Adrenal Cortex Hormones
- HLA Antigens
- Immunosuppressive Agents
- Cyclosporine
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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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