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

被引：34
作者：
Nishio, Nobuhiro
Takahashi, Yoshiyuki
Ohashi, Haruhiko [2 ]
Doisaki, Sayoko
Muramatsu, Hideki
Hama, Asahito
Shimada, Akira
Yagasaki, Hiroshi [3 ]
Kojima, Seiji [1 ]
机构：
[1] Nagoya Univ, Grad Sch Med, Dept Pediat, Shouwa Ku, Nagoya, Aichi 4668550, Japan
[2] Natl Hosp Org, Nagoya Med Ctr, Clin Res Ctr Blood Dis, Nagoya, Aichi, Japan
[3] Nihon Univ, Sch Med, Dept Pediat, Tokyo, Japan
来源：
PEDIATRIC TRANSPLANTATION | 2011年 / 15卷 / 02期
关键词：
dyskeratosis congenita; non-myeloablative hematopoietic stem cell transplantation; BONE-MARROW-TRANSPLANTATION; APLASTIC-ANEMIA; FAILURE; CYCLOPHOSPHAMIDE; COMPLICATIONS; FLUDARABINE; DISEASE; GRAFT;
D O I：
10.1111/j.1399-3046.2010.01431.x
中图分类号：
R72 [儿科学];
学科分类号：
100202 ;
摘要：
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.
引用
收藏
页码：161 / 166
页数：6
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