PMID- 11238097
OWN - NLM
STAT- MEDLINE
DCOM- 20010426
LR  - 20211109
IS  - 0006-4971 (Print)
IS  - 0006-4971 (Linking)
VI  - 97
IP  - 6
DP  - 2001 Mar 15
TI  - Impact of donor type on outcome of bone marrow transplantation for 
      Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow 
      Transplant Registry and the National Marrow Donor Program.
PG  - 1598-603
AB  - Human leukocyte antigen (HLA)-identical sibling bone marrow transplantation is an 
      effective treatment for Wiskott-Aldrich syndrome. However, most children with 
      this disease lack such donors and many patients receive transplants from 
      alternative donors. This study compared outcomes of HLA-identical sibling, other 
      related donor, and unrelated donor transplantation for Wiskott-Aldrich syndrome. 
      The outcome of 170 transplantations for Wiskott-Aldrich syndrome, from 1968 to 
      1996, reported to the International Bone Marrow Transplant Registry and/or 
      National Marrow Donor Program were assessed. Fifty-five were from HLA-identical 
      sibling donors, 48 from other relatives, and 67 from unrelated donors. 
      Multivariate proportional hazards regression was used to compare outcome by donor 
      type and identify other prognostic factors. Most transplant recipients were 
      younger than 5 years (79%), had a pretransplantation performance score greater 
      than or equal to 90% (63%), received pretransplantation preparative regimens 
      without radiation (82%), and had non-T-cell-depleted grafts (77%). Eighty percent 
      received their transplant after 1986. The 5-year probability of survival (95% 
      confidence interval) for all subjects was 70% (63%-77%). Probabilities differed 
      by donor type: 87% (74%-93%) with HLA-identical sibling donors, 52% (37%-65%) 
      with other related donors, and 71% (58%-80%) with unrelated donors (P =.0006). 
      Multivariate analysis indicated significantly lower survival using related donors 
      other than HLA-identical siblings (P =.0004) or unrelated donors in boys older 
      than 5 years (P =.0001), compared to HLA-identical sibling transplants. Boys 
      receiving an unrelated donor transplant before age 5 had survivals similar to 
      those receiving HLA-identical sibling transplants. The best transplantation 
      outcomes in Wiskott-Aldrich syndrome are achieved with HLA-identical sibling 
      donors. Equivalent survivals are possible with unrelated donors in young 
      children.
FAU - Filipovich, A H
AU  - Filipovich AH
AD  - Statistical Center of the International Bone Marrow Transplant Registry, Health 
      Policy Institute, Medical College of Wisconsin, Milwaukee, WI 53226, USA.
FAU - Stone, J V
AU  - Stone JV
FAU - Tomany, S C
AU  - Tomany SC
FAU - Ireland, M
AU  - Ireland M
FAU - Kollman, C
AU  - Kollman C
FAU - Pelz, C J
AU  - Pelz CJ
FAU - Casper, J T
AU  - Casper JT
FAU - Cowan, M J
AU  - Cowan MJ
FAU - Edwards, J R
AU  - Edwards JR
FAU - Fasth, A
AU  - Fasth A
FAU - Gale, R P
AU  - Gale RP
FAU - Junker, A
AU  - Junker A
FAU - Kamani, N R
AU  - Kamani NR
FAU - Loechelt, B J
AU  - Loechelt BJ
FAU - Pietryga, D W
AU  - Pietryga DW
FAU - Ringden, O
AU  - Ringden O
FAU - Vowels, M
AU  - Vowels M
FAU - Hegland, J
AU  - Hegland J
FAU - Williams, A V
AU  - Williams AV
FAU - Klein, J P
AU  - Klein JP
FAU - Sobocinski, K A
AU  - Sobocinski KA
FAU - Rowlings, P A
AU  - Rowlings PA
FAU - Horowitz, M M
AU  - Horowitz MM
LA  - eng
GR  - P01-CA-40053/CA/NCI NIH HHS/United States
GR  - U24-76518/PHS HHS/United States
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
SB  - IM
MH  - Actuarial Analysis
MH  - Bone Marrow Transplantation/adverse effects/*immunology/mortality
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - Follow-Up Studies
MH  - *Histocompatibility
MH  - Humans
MH  - Infant
MH  - International Agencies
MH  - Karnofsky Performance Status
MH  - Male
MH  - Multivariate Analysis
MH  - Registries
MH  - Survival Rate
MH  - Tissue Donors
MH  - Transplantation, Homologous/adverse effects/immunology/mortality
MH  - Treatment Outcome
MH  - Wiskott-Aldrich Syndrome/complications/mortality/*therapy
EDAT- 2001/03/10 10:00
MHDA- 2001/05/01 10:01
CRDT- 2001/03/10 10:00
PHST- 2001/03/10 10:00 [pubmed]
PHST- 2001/05/01 10:01 [medline]
PHST- 2001/03/10 10:00 [entrez]
AID - S0006-4971(20)56174-0 [pii]
AID - 10.1182/blood.v97.6.1598 [doi]
PST - ppublish
SO  - Blood. 2001 Mar 15;97(6):1598-603. doi: 10.1182/blood.v97.6.1598.