PMID- 17293882
OWN - NLM
STAT- MEDLINE
DCOM- 20070516
LR  - 20071203
IS  - 0268-3369 (Print)
IS  - 0268-3369 (Linking)
VI  - 39
IP  - 7
DP  - 2007 Apr
TI  - Hematopoietic cell transplantation for Chediak-Higashi syndrome.
PG  - 411-5
AB  - We reviewed outcomes after allogeneic hematopoietic cell transplantation (HCT) in 
      35 children with Chediak-Higashi syndrome (CHS). Twenty-two patients had a 
      history of the life-threatening accelerated phase of CHS before HCT and 11 were 
      in accelerated phase at transplantation. Thirteen patients received their 
      allograft from an human leukocyte antigen (HLA)-matched sibling, 10 from an 
      alternative related donor and 12 from an unrelated donor. Eleven recipients of 
      HLA-matched sibling donor, three recipients of alternative related donor and 
      eight recipients of unrelated donor HCT are alive. With a median follow-up of 6.5 
      years, the 5-year probability of overall survival is 62%. Mortality was highest 
      in those with accelerated phase disease at transplantation and after alternative 
      related donor HCT. Only four of 11 patients with active disease at 
      transplantation are alive. Seven recipients of alternative related donor HCT had 
      active disease at transplantation and this may have influenced the poor outcome 
      in this group. Although numbers are limited, HCT appears to be effective therapy 
      for correcting and preventing hematologic and immunologic complications of CHS, 
      and an unrelated donor may be a suitable alternative for patients without an 
      HLA-matched sibling. Early referral and transplantation in remission after 
      accelerated phase disease may improve disease-free survival.
FAU - Eapen, M
AU  - Eapen M
AD  - Department of Medicine, Statistical Center, Center for International Blood and 
      Marrow Transplant Registry, Medical College of Wisconsin, Milwaukee, WI, USA.
FAU - DeLaat, C A
AU  - DeLaat CA
FAU - Baker, K S
AU  - Baker KS
FAU - Cairo, M S
AU  - Cairo MS
FAU - Cowan, M J
AU  - Cowan MJ
FAU - Kurtzberg, J
AU  - Kurtzberg J
FAU - Steward, C G
AU  - Steward CG
FAU - Veys, P A
AU  - Veys PA
FAU - Filipovich, A H
AU  - Filipovich AH
LA  - eng
GR  - U24-CA76518-08/CA/NCI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
DEP - 20070212
PL  - England
TA  - Bone Marrow Transplant
JT  - Bone marrow transplantation
JID - 8702459
RN  - 0 (HLA Antigens)
SB  - IM
MH  - Adolescent
MH  - Chediak-Higashi Syndrome/*therapy
MH  - Child
MH  - Child, Preschool
MH  - Disease-Free Survival
MH  - Female
MH  - Follow-Up Studies
MH  - Graft vs Host Disease
MH  - HLA Antigens/biosynthesis
MH  - Hematopoietic Stem Cell Transplantation/*methods
MH  - Hematopoietic Stem Cells/cytology
MH  - Humans
MH  - Male
MH  - Retrospective Studies
MH  - Transplantation, Homologous
MH  - Treatment Outcome
EDAT- 2007/02/13 09:00
MHDA- 2007/05/17 09:00
CRDT- 2007/02/13 09:00
PHST- 2007/02/13 09:00 [pubmed]
PHST- 2007/05/17 09:00 [medline]
PHST- 2007/02/13 09:00 [entrez]
AID - 1705600 [pii]
AID - 10.1038/sj.bmt.1705600 [doi]
PST - ppublish
SO  - Bone Marrow Transplant. 2007 Apr;39(7):411-5. doi: 10.1038/sj.bmt.1705600. Epub 
      2007 Feb 12.