PMID- 17663681
OWN - NLM
STAT- MEDLINE
DCOM- 20071003
LR  - 20130207
IS  - 1397-3142 (Print)
IS  - 1397-3142 (Linking)
VI  - 11
IP  - 6
DP  - 2007 Sep
TI  - Hematopoietic stem cell transplantation for Diamond-Blackfan anemia: a report 
      from the Aplastic Anemia Committee of the Japanese Society of Pediatric 
      Hematology.
PG  - 601-7
AB  - Transfusion-dependent Diamond-Blackfan anemia (DBA) patients opt for allogeneic 
      hematopoietic stem cell transplantation (HSCT) as curative therapy. Clinical 
      outcomes of 19 transplanted Japanese patients were analyzed. Prior to HSCT, 10 
      patients (53%) suffered hemosiderosis with organ dysfunction, and all eight with 
      short stature (42%) had adverse effects of prednisolone. Median age at the time 
      of HSCT was 56 months. Transplantation sources were 13 bone marrow [six human 
      leukocyte antigen (HLA)-matched siblings, and six HLA-matched and one 
      HLA-mismatched unrelated donors], five cord blood (two HLA-matched siblings and 
      three HLA-mismatched unrelated donors), and one peripheral blood from 
      haploidentical mother. All 13 patients with bone marrow transplantation (BMT) and 
      two with sibling cord blood transplantation (CBT) had successful engraftment. Of 
      three patients who underwent unrelated CBT, one died after engraftment, and the 
      other two had graft failure but succeeded in a second BMT from an HLA-disparate 
      father and unrelated donor, respectively. One died shortly after haploidentical 
      PBSCT. The five-yr failure-free survival rate after BMT was higher than CBT 
      (100%: 40%, p=0.002). Platelet recovery was slower in seven unrelated BMT than in 
      six sibling BMT (p=0.030). No other factors were associated with engraftment and 
      survival. These results suggest that allogeneic BMT, but not unrelated CBT, is an 
      effective HSCT for refractory DBA.
FAU - Mugishima, H
AU  - Mugishima H
AD  - Department of Advanced Medicine, Division of Cell Regeneration and 
      Transplantation, Nihon University School of Medicine, Tokyo, Japan. 
      mugishim@med.nihon-u.ac.jp
FAU - Ohga, S
AU  - Ohga S
FAU - Ohara, A
AU  - Ohara A
FAU - Kojima, S
AU  - Kojima S
FAU - Fujisawa, K
AU  - Fujisawa K
FAU - Tsukimoto, I
AU  - Tsukimoto I
CN  - Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - Denmark
TA  - Pediatr Transplant
JT  - Pediatric transplantation
JID - 9802574
SB  - IM
MH  - Anemia, Diamond-Blackfan/*surgery
MH  - Female
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects
MH  - Humans
MH  - Infant
MH  - Infant, Newborn
MH  - Male
MH  - Postoperative Complications/epidemiology/etiology
MH  - Retrospective Studies
EDAT- 2007/08/01 09:00
MHDA- 2007/10/04 09:00
CRDT- 2007/08/01 09:00
PHST- 2007/08/01 09:00 [pubmed]
PHST- 2007/10/04 09:00 [medline]
PHST- 2007/08/01 09:00 [entrez]
AID - PTR616 [pii]
AID - 10.1111/j.1399-3046.2006.00616.x [doi]
PST - ppublish
SO  - Pediatr Transplant. 2007 Sep;11(6):601-7. doi: 10.1111/j.1399-3046.2006.00616.x.