Table 1.
Patient, disease and transplant characteristics
| HLA match | ||||||
|---|---|---|---|---|---|---|
| Matched | 1 allele mismatch (7/8) |
2 allele mismatch (6/8) |
3 allele mismatch (5/8) |
≥4 allele mismatch (3/8 or 4/8) |
p-value | |
| Number | 234 | 266 | 303 | 261 | 135 | |
| Age, years | <0·001 | |||||
| <1 | 96 (41%) | 108 (41%) | 111 (37%) | 76 (29%) | 40 (30%) | |
| 1 – 5 | 100 (43%) | 117 (44%) | 131 (43%) | 112 (43%) | 57 (42%) | |
| 6 – 10 | 36 (15%) | 30 (11%) | 49 (16%) | 57 (22%) | 25 (19%) | |
| 11 – 16 | 2 (<1%) | 11 (4%) | 12 (4%) | 16 (6%) | 13 (10%) | |
| Sex | 0·548 | |||||
| Male | 143 (61%) | 154 (58%) | 188 (62%) | 167 (64%) | 77 (57%) | |
| Female | 91 (39%) | 112 (42%) | 115 (38%) | 94 (36%) | 58 (43%) | |
| CMV serostatus | 0·040 | |||||
| Negative | 124 (53%) | 150 (56%) | 162 (53%) | 111 (43%) | 71 (53%) | |
| Positive | 92 (39%) | 97 (36%) | 126 (42%) | 132 (51%) | 59 (44%) | |
| Not reported | 18 (8%) | 19 (7%) | 15 (5%) | 18 (7%) | 5 (4%) | |
| Disease | <0·001 | |||||
| SCID | 39 (17%) | 30 (11%) | 27 (9%) | 22 (8%) | 13 (10%) | |
| Non-SCID PIDD | 57 (24%) | 71 (27%) | 73 (24%) | 66 (25%) | 22 (16%) | |
| IEM | 92 (39%) | 110 (41%) | 136 (45%) | 94 (36%) | 52 (39%) | |
| Marrow failure | 17 (7%) | 20 (8%) | 29 (10%) | 30 (11%) | 18 (13%) | |
| Fanconi anemia | 8 (3%) | 16 (6%) | 26 (9%) | 28 (11%) | 22 (16%) | |
| Hemoglobinopathy | 21 (9%) | 19 (7%) | 12 (4%) | 21 (8%) | 8 (6%) | |
| Conditioning regimen | 0·889 | |||||
| Myeloablative | 153 (65%) | 169 (64%) | 202 (67%) | 164 (63%) | 88 (65%) | |
| Bu + Cy ± other | 131 | 140 | 163 | 140 | 74 | |
| Bu + flud ± other | 26 | 26 | 36 | 18 | 7 | |
| Mel + flud ± other | 3 | 8 | 9 | 3 | 5 | |
| TBI + Cy ± other | 3 | 2 | 3 | 5 | 3 | |
| Reduced intensity | 81 (35%) | 97 (36%) | 101 (33%) | 97 (37%) | 47 (35%) | |
| Bu + Cy ± other | 1 | 4 | 7 | 3 | 3 | |
| Bu + flud ± other | 6 | 5 | 2 | 4 | 1 | |
| Mel + flud ± other | 18 | 27 | 30 | 21 | 4 | |
| Cy + flud | 7 | 11 | 12 | 27 | 4 | |
| Cy ± other | 5 | 8 | 11 | 4 | – | |
| Flud + other | 10 | 10 | 11 | 11 | 7 | |
| TBI + Cy + flud | 5 | 13 | 13 | 18 | 18 | |
| TBI + other | 7 | 5 | 3 | 6 | 5 | |
| None | 11 | 6 | 3 | 2 | 3 | |
| In vivo T cell depletion | 0·013 | |||||
| None | 37 (16%) | 38 (14%) | 36 (12%) | 33 (13%) | 7 (5%) | |
| Yes | 181 (77%) | 215 (81%) | 255 (84%) | 218 (84%) | 127 (94%) | |
| Not reported | 16 (7%) | 13 (5%) | 12 (4%) | 10 (4%) | 1 (1%) | |
| GVHD prophylaxis | 0·623 | |||||
| Cyclosporine ± steroid | 106 (45%) | 120 (45%) | 140 (46%) | 128 (49%) | 72 (53%) | |
| Cyclosporine + MTX | 19 (8%) | 17 (6%) | 25 (8%) | 29 (11%) | 8 (6%) | |
| Cyclosporine + MMF | 83 (35%) | 90 (34%) | 100 (33%) | 70 (27%) | 33 (24%) | |
| Tacrolimus | 5 (2%) | 7 (3%) | 7 (2%) | 9 (3%) | 9 (7%) | |
| Tacrolimus + MTX | 10 (4%) | 15 (6%) | 10 (3%) | 11 (4%) | 5 (4%) | |
| Tacrolimus + MMF | 11 (5%) | 17 (6%) | 21 (7%) | 14 (5%) | 8 (6%) | |
| Pre cryopreserved TNC | 0·213 | |||||
| Median (range) | 10 (2 – 58) |
10 (2 – 61) |
11 (2 – 77) |
12 (2 – 74) |
11 (1 – 66) |
|
| < 5 × 107/kg | 23 (10%) | 21 (8%) | 19 (6%) | 22 (8%) | 15 (11%) | |
| 5 – 10 × 107/kg | 76 (32%) | 68 (26%) | 110 (36%) | 100 (38%) | 53 (39%) | |
| 11 – 15 × 107/kg | 36 (15%) | 48 (18%) | 44 (15%) | 40 (15%) | 21 (16%) | |
| 16 – 20 × 107/kg | 25 (11%) | 25 (9%) | 20 (7%) | 22 (8%) | 14 (10%) | |
| ≥ 21 × 107/kg | 27 (12%) | 41 (15%) | 36 (12%) | 33 (13%) | 16 (12%) | |
| Not reported | 47 (20%) | 63 (24%) | 74 (24%) | 44 (17%) | 16 (12%) | |
| Transplant period | 0·005 | |||||
| 2000 – 2005 | 62 (26%) | 65 (24%) | 87 (29%) | 100 (38%) | 45 (33%) | |
| 2006 – 2012 | 172 (74%) | 201 (76%) | 216 (71%) | 161 (62%) | 90 (67%) | |
| Follow up, median (inter quartile range), months | 60 (33–81) |
61 (36–84) |
59 (36–78) |
70 (48–105) |
65 (48–95) |
|
Number in parenthesis = column percent
Abbreviations:
Diseases: SCID = severe combined immunodeficiency; non-SCID PIDD = non- severe combined immunodeficiency primary immunodeficiency diseases; IEM: inborn errors of metabolism Conditioning regimen: Bu = busulfan; Cy = cyclophosphamide; flud = fludarabine; mel = melphalan; TBI = total body irradiation
GVHD = graft versus host disease; MTX = methotrexate; MMF = mycophenolate