Table 1.

Characteristics of 170 patients receiving allogeneic BMT for Wiskott-Aldrich syndrome

HLA-identical sibling donor
(n = 55)
Other related donor
(n = 48)
Unrelated donor
(n = 67)
P
Age at BMT, median (range), y 3  (< 1-16) 2  (< 1-17) 2  (< 1-32) < .05  
Age at BMT    .26  
 Younger than 1 y 13/55  (24%) 12/48  (25%) 9/67  (13%)  
 1-2 y 18/55  (33%) 26/48  (54%) 34/67  (51%)  
 3-4 y 10/55  (18%) 3/48  (6%) 9/67  (13%)  
 5-9 y 7/55  (13%) 3/48  (6%) 8/67  (12%)  
 10 y or older 7/55  (13%) 4/48  (8%) 7/67  (10%)  
Lansky/Karnofsky performance score before BMT ≥ 90% 26/45  (58%) 28/47  (60%) 43/63  (68%) .48 
Infection in the 2 wk before BMT 12/52  (23%) 16/48  (33%) 11/61  (18%) .18  
Donor-recipient HLA mismatch    < .001  
 0 antigen mismatch 55/55  (100%) 3/43  (7%) 43/62  (69%)  
 1 antigen mismatch NA 16/43  (37%) 18/62  (29%)  
 ≥ 2 antigen mismatch NA 24/43  (56%) 1/62  (2%)  
Male donor 23/51  (45%) 32/48  (67%) 35/64  (55%) .10  
Year of transplant    < .001  
 1968-1979 5/55  (9%) 2/48  (4%) 0/67  (0%)  
 1980-1984 8/55  (15%) 10/48  (21%) 0/67  (0%)  
 1985-1989 13/55  (24%) 13/48  (27%) 3/67  (4%)  
 1990-1993 14/55  (25%) 19/48  (40%) 35/67  (52%)  
 1994-1996 15/55  (27%) 4/48  (8%) 29/67  (43%)  
TBI for conditioning pretransplant 4/55  (7%) 11/45  (24%) 14/64  (22%) < .05  
GVHD prophylaxis    < .001  
 MTX + CsA 20/55  (36%) 14/48  (29%) 37/64  (58%)  
 MTX ± other 19/55  (35%) 2/48  (4%) 3/64  (5%)  
 CsA ± other 14/55  (25%) 3/48  (6%) 13/64  (20%)  
 T-cell depletion ± other 0/55  (0%) 26/48  (54%) 11/64  (17%)  
 Other (non–T-cell depleted) or none* 2/55  (4%) 3/48  (6%) 0/64  (0%)  
HLA-identical sibling donor
(n = 55)
Other related donor
(n = 48)
Unrelated donor
(n = 67)
P
Age at BMT, median (range), y 3  (< 1-16) 2  (< 1-17) 2  (< 1-32) < .05  
Age at BMT    .26  
 Younger than 1 y 13/55  (24%) 12/48  (25%) 9/67  (13%)  
 1-2 y 18/55  (33%) 26/48  (54%) 34/67  (51%)  
 3-4 y 10/55  (18%) 3/48  (6%) 9/67  (13%)  
 5-9 y 7/55  (13%) 3/48  (6%) 8/67  (12%)  
 10 y or older 7/55  (13%) 4/48  (8%) 7/67  (10%)  
Lansky/Karnofsky performance score before BMT ≥ 90% 26/45  (58%) 28/47  (60%) 43/63  (68%) .48 
Infection in the 2 wk before BMT 12/52  (23%) 16/48  (33%) 11/61  (18%) .18  
Donor-recipient HLA mismatch    < .001  
 0 antigen mismatch 55/55  (100%) 3/43  (7%) 43/62  (69%)  
 1 antigen mismatch NA 16/43  (37%) 18/62  (29%)  
 ≥ 2 antigen mismatch NA 24/43  (56%) 1/62  (2%)  
Male donor 23/51  (45%) 32/48  (67%) 35/64  (55%) .10  
Year of transplant    < .001  
 1968-1979 5/55  (9%) 2/48  (4%) 0/67  (0%)  
 1980-1984 8/55  (15%) 10/48  (21%) 0/67  (0%)  
 1985-1989 13/55  (24%) 13/48  (27%) 3/67  (4%)  
 1990-1993 14/55  (25%) 19/48  (40%) 35/67  (52%)  
 1994-1996 15/55  (27%) 4/48  (8%) 29/67  (43%)  
TBI for conditioning pretransplant 4/55  (7%) 11/45  (24%) 14/64  (22%) < .05  
GVHD prophylaxis    < .001  
 MTX + CsA 20/55  (36%) 14/48  (29%) 37/64  (58%)  
 MTX ± other 19/55  (35%) 2/48  (4%) 3/64  (5%)  
 CsA ± other 14/55  (25%) 3/48  (6%) 13/64  (20%)  
 T-cell depletion ± other 0/55  (0%) 26/48  (54%) 11/64  (17%)  
 Other (non–T-cell depleted) or none* 2/55  (4%) 3/48  (6%) 0/64  (0%)  

BMT indicates bone marrow transplant; HLA, human leukocyte antigen; TBI, total body irradiation; GVHD, graft-versus-host disease; MTX, methotrexate; CsA, cyclosporine A.

*

One patient with an HLA-identical sibling donor and 3 patients with other related donors received no GVHD prophylaxis.

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