Patient and transplant characteristic
| . | HLA-matched sibling* . | Alternative donor† . |
|---|---|---|
| Number | 65 | 128 |
| Age, median (range), y | 1 (<1-6) | 1 (<1-7) |
| Age, no. (%) | ||
| <6 mo | 29 (45) | 43 (34) |
| 6-12 mo | 18 (28) | 48 (38) |
| 13-24 mo | 12 (18) | 29 (23) |
| 25-60 mo | 6 (9) | 8 (6) |
| Sex, male (%) | 32 (49) | 75 (59) |
| Lansky performance score, no. (%) | ||
| 90-100 | 35 (54) | 52 (41) |
| 70-80 | 13 (20) | 39 (30) |
| ≤60 | 11 (17) | 25 (20) |
| Not reported | 6 (9) | 12 (9) |
| Visual impairment at diagnosis, no. (%) | ||
| Present | 37 (57) | 77 (60) |
| Absent | 12 (18) | 28 (22) |
| Not reported | 16 (25) | 23 (18) |
| Auditory impairment at diagnosis, no. (%) | ||
| Present | 11 (17) | 31 (24) |
| Absent | 38 (58) | 66 (52) |
| Not reported | 16 (25) | 31 (24) |
| Gross motor delay at diagnosis, no. (%) | ||
| Present | 12 (18) | 50 (39) |
| Absent | 34 (53) | 49 (38) |
| Not reported | 19 (29) | 29 (23) |
| Conditioning regimen, no. (%) | ||
| Myeloablative | ||
| TBI + busulfan + cyclophosphamide | — | 16 (12) |
| TBI + cyclophosphamide | — | 7 (5) |
| Busulfan + cyclophosphamide | 56 (86) | 83 (65) |
| Busulfan + melphalan | 8 (12) | 1 (1) |
| Busulfan + fludarabine | 9 (7) | |
| Melphalan + fludarabine | — | 3 (2) |
| Reduced intensity | ||
| TBI + chemotherapeutic agent | — | 3 (3) |
| TLI + cyclophosphamide | — | 1 (1) |
| Busulfan + fludarabine | 1 (2) | 3 (2) |
| Melphalan + fludarabine | — | 2 (2) |
| GVHD prophylaxis | ||
| Ex vivo T-cell depletion | — | 20 (16) |
| Cyclosporine-containing | 62 (95) | 100 (78) |
| Tacrolimus-containing | 3 (5) | 8 (6) |
| In vivo T-cell depletion, no. (%) | 22 (34) | 87 (68) |
| Transplant period, no. (%) | ||
| 1990-2004 | 56 (86) | 94 (73) |
| 2005-2011 | 9 (14) | 34 (27) |
| Follow up, median (range), mo | 67 (3-172) | 81 (3-223) |
| . | HLA-matched sibling* . | Alternative donor† . |
|---|---|---|
| Number | 65 | 128 |
| Age, median (range), y | 1 (<1-6) | 1 (<1-7) |
| Age, no. (%) | ||
| <6 mo | 29 (45) | 43 (34) |
| 6-12 mo | 18 (28) | 48 (38) |
| 13-24 mo | 12 (18) | 29 (23) |
| 25-60 mo | 6 (9) | 8 (6) |
| Sex, male (%) | 32 (49) | 75 (59) |
| Lansky performance score, no. (%) | ||
| 90-100 | 35 (54) | 52 (41) |
| 70-80 | 13 (20) | 39 (30) |
| ≤60 | 11 (17) | 25 (20) |
| Not reported | 6 (9) | 12 (9) |
| Visual impairment at diagnosis, no. (%) | ||
| Present | 37 (57) | 77 (60) |
| Absent | 12 (18) | 28 (22) |
| Not reported | 16 (25) | 23 (18) |
| Auditory impairment at diagnosis, no. (%) | ||
| Present | 11 (17) | 31 (24) |
| Absent | 38 (58) | 66 (52) |
| Not reported | 16 (25) | 31 (24) |
| Gross motor delay at diagnosis, no. (%) | ||
| Present | 12 (18) | 50 (39) |
| Absent | 34 (53) | 49 (38) |
| Not reported | 19 (29) | 29 (23) |
| Conditioning regimen, no. (%) | ||
| Myeloablative | ||
| TBI + busulfan + cyclophosphamide | — | 16 (12) |
| TBI + cyclophosphamide | — | 7 (5) |
| Busulfan + cyclophosphamide | 56 (86) | 83 (65) |
| Busulfan + melphalan | 8 (12) | 1 (1) |
| Busulfan + fludarabine | 9 (7) | |
| Melphalan + fludarabine | — | 3 (2) |
| Reduced intensity | ||
| TBI + chemotherapeutic agent | — | 3 (3) |
| TLI + cyclophosphamide | — | 1 (1) |
| Busulfan + fludarabine | 1 (2) | 3 (2) |
| Melphalan + fludarabine | — | 2 (2) |
| GVHD prophylaxis | ||
| Ex vivo T-cell depletion | — | 20 (16) |
| Cyclosporine-containing | 62 (95) | 100 (78) |
| Tacrolimus-containing | 3 (5) | 8 (6) |
| In vivo T-cell depletion, no. (%) | 22 (34) | 87 (68) |
| Transplant period, no. (%) | ||
| 1990-2004 | 56 (86) | 94 (73) |
| 2005-2011 | 9 (14) | 34 (27) |
| Follow up, median (range), mo | 67 (3-172) | 81 (3-223) |
TBI, total body irradiation; TLI, total lymphoid irradiation.
N = 62 received bone marrow grafts and N = 3, peripheral blood.
N = 25 mismatched relative (bone marrow, n = 20; peripheral blood, n = 4; cord blood, n = 1), N = 55 unrelated adult donor (bone marrow, n = 46; 8 were HLA-matched and 38 HLA-mismatched; peripheral blood, n = 9, 6 were HLA-matched and 3 HLA-mismatched), N = 48 umbilical cord blood (10 HLA-matched and 38, HLA-mismatched).