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 language | English |
|---|---|
| Pages (from-to) | 161-166 |
| Number of pages | 6 |
| Journal | Pediatric Transplantation |
| Volume | 15 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - Mar 2011 |
| Externally published | Yes |
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Keywords
- dyskeratosis congenita
- non-myeloablative hematopoietic stem cell transplantation
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Transplantation
Cite this
Reduced-intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita. / Nishio, Nobuhiro; Takahashi, Yoshiyuki; Ohashi, Haruhiko; Doisaki, Sayoko; Muramatsu, Hideki; Hama, Asahito; Shimada, Akira; Yagasaki, Hiroshi; Kojima, Seiji.
In: Pediatric Transplantation, Vol. 15, No. 2, 03.2011, p. 161-166.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Reduced-intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita
AU - Nishio, Nobuhiro
AU - Takahashi, Yoshiyuki
AU - Ohashi, Haruhiko
AU - Doisaki, Sayoko
AU - Muramatsu, Hideki
AU - Hama, Asahito
AU - Shimada, Akira
AU - Yagasaki, Hiroshi
AU - Kojima, Seiji
PY - 2011/3
Y1 - 2011/3
N2 - 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.
AB - 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.
KW - dyskeratosis congenita
KW - non-myeloablative hematopoietic stem cell transplantation
UR - http://www.scopus.com/inward/record.url?scp=79951633240&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=79951633240&partnerID=8YFLogxK
U2 - 10.1111/j.1399-3046.2010.01431.x
DO - 10.1111/j.1399-3046.2010.01431.x
M3 - Article
C2 - 21176016
AN - SCOPUS:79951633240
VL - 15
SP - 161
EP - 166
JO - Pediatric Transplantation
JF - Pediatric Transplantation
SN - 1397-3142
IS - 2
ER -