Reduced-intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita

Nobuhiro Nishio, Yoshiyuki Takahashi, Haruhiko Ohashi, Sayoko Doisaki, Hideki Muramatsu, Asahito Hama, Akira Shimada, Hiroshi Yagasaki, Seiji Kojima

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

DC is an inherited bone marrow failure syndrome mainly characterized by nail dystrophy, abnormal skin pigmentation, and oral leukoplakia. Bone marrow failure is the most common cause of death in patients with DC. Because previous results of HSCT with a myeloablative regimen were disappointing, we used a reduced-intensity conditioning regimen for two patients with classic DC, and one patient with cryptic DC who harbored the TERT mutation. Graft sources included two mismatched-related bone marrow (BM) donors and one unrelated BM donor. Successful engraftment was achieved with few regimen-related toxicities in all patients. They were alive 10, 66, and 72 months after transplantation, respectively. Long-term follow-up is crucial to determine the late effects of our conditioning regimen.

Original languageEnglish
Pages (from-to)161-166
Number of pages6
JournalPediatric Transplantation
Volume15
Issue number2
DOIs
Publication statusPublished - Mar 1 2011

Keywords

  • dyskeratosis congenita
  • non-myeloablative hematopoietic stem cell transplantation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

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