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Reduced‐intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita
Author(s) -
Nishio Nobuhiro,
Takahashi Yoshiyuki,
Ohashi Haruhiko,
Doisaki Sayoko,
Muramatsu Hideki,
Hama Asahito,
Shimada Akira,
Yagasaki Hiroshi,
Kojima Seiji
Publication year - 2011
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/j.1399-3046.2010.01431.x
Subject(s) - dyskeratosis congenita , medicine , transplantation , regimen , hematopoietic stem cell transplantation , stem cell , conditioning regimen , surgery , gastroenterology , biology , genetics , dna , telomere
Nishio N, Takahashi Y, Ohashi H, Doisaki S, Muramatsu H, Hama A, Shimada A, Yagasaki H, Kojima S. Reduced‐intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita.
Pediatr Transplantation 2011: 15:161–166. © 2010 John Wiley & Sons A/S. 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.