PMID- 22435112
OWN - NLM
STAT- MEDLINE
DCOM- 20120406
LR  - 20191027
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 58
IP  - 4
DP  - 2012 Apr
TI  - Bone marrow transplant options and preferences in a sickle cell anemia cohort on 
      chronic transfusions.
PG  - 611-5
AB  - BACKGROUND: Bone marrow transplantation (BMT) using human leukocyte antigen 
      (HLA)-matched sibling donors can be curative for children with sickle cell anemia 
      (SCA). However, minimal data exist regarding availability of HLA-identical 
      matched siblings for transplant-eligible children, and family interest in 
      pursuing transplantation. METHODS: We retrospectively analyzed a pediatric SCA 
      cohort receiving chronic transfusions between July 2004 and January 2011. Data 
      were analyzed regarding the number of full siblings and half-siblings, 
      availability, and family interest in HLA testing the full siblings, and interest 
      in proceeding with HLAmatched transplantation. RESULTS: Among 113 patients, 46 
      (41%) had at least 1 full sibling and 40 (35%) had an unaffected full sibling who 
      could serve as a BMT donor. The families of 23 of these patients (58%) agreed to 
      HLA-type sibling, 8 of whom (35%) were matched. Transfusion indications for 
      families agreeing to HLA typing included stroke (46%) abnormal TCD (29%), acute 
      chest syndrome (21%), and other CNS reasons (4%). Common reasons to decline HLA 
      typing or transplantation included fear of the process, toxicities of the 
      procedure, and comfort with current quality of life on transfusions. Only 8 of 
      113 (7%) were eligible for matched BMT, and only 3 (3%) underwent HLA-matched 
      transplantation. Two unmatched children received haploidentical transplantation. 
      CONCLUSIONS: Most families of children with SCA on chronic transfusions choose to 
      proceed with HLA typing. However, when a matched sibling was identified, most 
      families declined to proceed with matched-sibling transplantation. Discussing BMT 
      as a treatment option, offering HLA typing and identifying barriers may improve 
      acceptance of this treatment modality.
FAU - Hansbury, Eileen N
AU  - Hansbury EN
AD  - Baylor College of Medicine, Houston, Texas, USA.
FAU - Schultz, William H
AU  - Schultz WH
FAU - Ware, Russell E
AU  - Ware RE
FAU - Aygun, Banu
AU  - Aygun B
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
SB  - IM
CIN - Pediatr Blood Cancer. 2012 Apr;58(4):485-6. PMID: 22183942
MH  - Adolescent
MH  - Anemia, Sickle Cell/*therapy
MH  - *Blood Transfusion
MH  - *Bone Marrow Transplantation
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - *Histocompatibility Testing
MH  - Humans
MH  - Infant
MH  - Male
MH  - Retrospective Studies
MH  - Transplantation, Homologous
EDAT- 2012/03/22 06:00
MHDA- 2012/04/07 06:00
CRDT- 2012/03/22 06:00
PHST- 2012/03/22 06:00 [entrez]
PHST- 2012/03/22 06:00 [pubmed]
PHST- 2012/04/07 06:00 [medline]
AID - 10.1002/pbc.23304 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2012 Apr;58(4):611-5. doi: 10.1002/pbc.23304.