Probability of survival (95% confidence interval) 5 years after allogeneic BMT for Wiskott-Aldrich syndrome (univariate analyses)
| Variable . | HLA-identical sibling . | Other relative . | Unrelated donor . | ||||||
|---|---|---|---|---|---|---|---|---|---|
| N . | Probability (95% CI) . | P . | N . | Probability (95% CI) . | P . | N . | Probability (95% CI) . | P . | |
| Donor | 55 | 87 (74-94)% | NA | 48 | 52 (37-65)% | NA | 67 | 71 (58-80)% | < .0006* |
| Age | .22 | .77 | < .0001 | ||||||
| No older than 5 years | 41 | 90 (75-96)% | 41 | 53 (37-67)% | 52 | 84 (70-92)% | |||
| Older than 5 years | 14 | 79 (47-93)% | 7 | 38 (6-72)% | 15 | 0 | |||
| Lansky/Karnofsky performance score | .20 | .92 | |||||||
| Below 90% | 19 | 84 (58-95)% | 19 | 41 (20-62)% | 20 | 70 (45-85)% | |||
| At least 90% | 26 | 96 (75-99)% | 28 | 61 (40-76)% | .20 | 43 | 71 (54-82)% | ||
| Infection pre-BMT | .16 | .13 | < .04 | ||||||
| No | 40 | 84 (68-93)% | 32 | 59 (41-74)% | 51 | 71 (56-82)% | |||
| Yes | 12 | 100% | 16 | 35 (13-59)% | 11 | 55 (23-80)% | |||
| Donor-recipient HLA match | NA | < .03 | .98 | ||||||
| 0-1 antigen mismatched related | 19† | 74 (48-88)% | — | ||||||
| At least 2 antigen mismatched related | 24 | 36 (18-55)% | — | ||||||
| 0 antigen mismatched unrelated | — | 44 | 69 (52-81)% | ||||||
| 1 antigen mismatched unrelated | — | 19‡ | 68 (43-84)% | ||||||
| Donor sex | .16 | .09 | .26 | ||||||
| Male | 23 | 95 (72-99)% | 32 | 59 (40-74)% | 35 | 65 (47-78)% | |||
| Female | 28 | 82 (62-92)% | 16 | 38 (15-60)% | 30 | 75 (55-87)% | |||
| Year of BMT | .81 | .30 | .87 | ||||||
| Before 1985 | 13 | 85 (51-96)% | 12 | 33 (10-59)% | 0 | — | |||
| 1985-1989 | 13 | 92 (57-99)% | 13 | 62 (31-82)% | 3 | 67 (5-95)% | |||
| 1990-1996 | 29 | 85 (65-94)% | 23 | 57 (34-74)% | 64 | 71 (58-81)% | |||
| Conditioning regimen | .37 | .46 | .17 | ||||||
| No TBI | 51 | 88 (75-94)% | 34 | 56 (38-71)% | 50 | 73 (58-83)% | |||
| TBI | 4 | 75 (13-96)% | 11 | 36 (11-63)% | 14 | 64 (34-83)% | |||
| GVHD prophylaxis | .29 | < .0002 | .56 | ||||||
| MTX | 19 | 94 (67-99)% | 2 | 0 | 3 | 33 (1-77)% | |||
| CsA | 14 | 93 (59-99)% | 3 | 100% | 13 | 68 (36-87)% | |||
| CsA + MTX | 20 | 80 (55-92)% | 14 | 79 (47-93)% | 37 | 69 (51-81)% | |||
| T-cell depletion | 0 | — | 26 | 34 (16-52)% | 11 | 82 (45-95)% | |||
| Variable . | HLA-identical sibling . | Other relative . | Unrelated donor . | ||||||
|---|---|---|---|---|---|---|---|---|---|
| N . | Probability (95% CI) . | P . | N . | Probability (95% CI) . | P . | N . | Probability (95% CI) . | P . | |
| Donor | 55 | 87 (74-94)% | NA | 48 | 52 (37-65)% | NA | 67 | 71 (58-80)% | < .0006* |
| Age | .22 | .77 | < .0001 | ||||||
| No older than 5 years | 41 | 90 (75-96)% | 41 | 53 (37-67)% | 52 | 84 (70-92)% | |||
| Older than 5 years | 14 | 79 (47-93)% | 7 | 38 (6-72)% | 15 | 0 | |||
| Lansky/Karnofsky performance score | .20 | .92 | |||||||
| Below 90% | 19 | 84 (58-95)% | 19 | 41 (20-62)% | 20 | 70 (45-85)% | |||
| At least 90% | 26 | 96 (75-99)% | 28 | 61 (40-76)% | .20 | 43 | 71 (54-82)% | ||
| Infection pre-BMT | .16 | .13 | < .04 | ||||||
| No | 40 | 84 (68-93)% | 32 | 59 (41-74)% | 51 | 71 (56-82)% | |||
| Yes | 12 | 100% | 16 | 35 (13-59)% | 11 | 55 (23-80)% | |||
| Donor-recipient HLA match | NA | < .03 | .98 | ||||||
| 0-1 antigen mismatched related | 19† | 74 (48-88)% | — | ||||||
| At least 2 antigen mismatched related | 24 | 36 (18-55)% | — | ||||||
| 0 antigen mismatched unrelated | — | 44 | 69 (52-81)% | ||||||
| 1 antigen mismatched unrelated | — | 19‡ | 68 (43-84)% | ||||||
| Donor sex | .16 | .09 | .26 | ||||||
| Male | 23 | 95 (72-99)% | 32 | 59 (40-74)% | 35 | 65 (47-78)% | |||
| Female | 28 | 82 (62-92)% | 16 | 38 (15-60)% | 30 | 75 (55-87)% | |||
| Year of BMT | .81 | .30 | .87 | ||||||
| Before 1985 | 13 | 85 (51-96)% | 12 | 33 (10-59)% | 0 | — | |||
| 1985-1989 | 13 | 92 (57-99)% | 13 | 62 (31-82)% | 3 | 67 (5-95)% | |||
| 1990-1996 | 29 | 85 (65-94)% | 23 | 57 (34-74)% | 64 | 71 (58-81)% | |||
| Conditioning regimen | .37 | .46 | .17 | ||||||
| No TBI | 51 | 88 (75-94)% | 34 | 56 (38-71)% | 50 | 73 (58-83)% | |||
| TBI | 4 | 75 (13-96)% | 11 | 36 (11-63)% | 14 | 64 (34-83)% | |||
| GVHD prophylaxis | .29 | < .0002 | .56 | ||||||
| MTX | 19 | 94 (67-99)% | 2 | 0 | 3 | 33 (1-77)% | |||
| CsA | 14 | 93 (59-99)% | 3 | 100% | 13 | 68 (36-87)% | |||
| CsA + MTX | 20 | 80 (55-92)% | 14 | 79 (47-93)% | 37 | 69 (51-81)% | |||
| T-cell depletion | 0 | — | 26 | 34 (16-52)% | 11 | 82 (45-95)% | |||
CI indicates confidence interval; BMT, bone marrow transplant; NA, not applicable; GVHD, graft-versus-host disease; TBI, total body irradiation; CsA, cyclosporine A; MTX, methotrexate.
Comparison of survival by donor type.
Three donors phenotypically identical (0 antigen mismatch).
One donor-recipient pair mismatched for 2 antigens.