Table 1.
Variable | Training cohort | Validation cohort | P |
---|---|---|---|
Patients, n | 612 | 612 | |
Age at transplantation, y * | .47 | ||
<3 y | 152 (25) | 163 (27) | |
≥3 y | 460 (75) | 449 (73) | |
Sex | .69 | ||
Male | 321 (52) | 328 (54) | |
Female | 291 (48) | 284 (46) | |
Hematopoietic comorbidity index score | .35 | ||
≤2 | 505 (83) | 507 (83) | |
≥3 | 107 (17) | 103 (17) | |
Not reported | 0 | 2 (< 1) | |
Performance score | .64 | ||
90-100 | 517 (84) | 527 (86) | |
≤80 | 90 (15) | 79 (13) | |
Not reported | 5 (1) | 6 (1) | |
Cytomegalovirus serostatus | .16 | ||
Negative | 239 (39) | 207 (34) | |
Positive | 365 (60) | 395 (65) | |
Not reported | 8 (1) | 10 (2) | |
Cytogenetic risk† | .80 | ||
Favorable | 53 (9) | 47 (8) | |
Intermediate | 377 (62) | 401 (66) | |
Poor | 167 (27) | 150 (25) | |
Not reported | 15 (2) | 14 (2) | |
Disease status | .44 | ||
1st complete remission MRD+ | 91 (15) | 84 (14) | |
1st complete remission MRD− | 205 (33) | 223 (36) | |
1st complete remission | 24 (4) | 24 (4) | |
2nd complete remission MRD+ | 42 (7) | 42 (7) | |
2nd complete remission MRD− | 135 (22) | 134 (22) | |
2nd complete remission | 15 (2) | 13 (2) | |
Not in remission | 96 (15) | 92 (15) | |
Donor | .06 | ||
HLA-matched sibling | 114 (19) | 143 (23) | |
HLA-mismatched relative | 54 (9) | 35 (6) | |
HLA-matched unrelated | 177 (29) | 167 (27) | |
1-locus mismatched unrelated | 66 (11) | 60 (10) | |
6-8/8 HLA-matched cord blood | 109 (18) | 129 (21) | |
≤5/8 HLA-matched cord blood | 92 (15) | 78 (13) | |
Conditioning regimen | .82 | ||
TBI/cyclophosphamide | 43 (7) | 49 (8) | |
TBI/cyclophosphamide/fludarabine | 76 (12) | 67 (11) | |
TBI/cyclophosphamide+other | 23 (4) | 23 (4) | |
TBI+other | 29 (5) | 24 (4) | |
Busulfan/cyclophosphamide | 261 (43) | 276 (45) | |
Busulfan/melphalan | 42 (7) | 33 (5) | |
Fludarabine/busulfan/thiotepa | 138 (23) | 140 (23) | |
GVHD prophylaxis | .89 | ||
Ex vivo T-cell depletion/CD34 selection | 36 (6) | 30 (5) | |
Posttransplantation CY, with or without other(s) | 28 (5) | 23 (4) | |
Calcineurin inhibitor+mycophenolate | 217 (35) | 210 (34) | |
Calcineurin inhibitor+methotrexate | 264 (43) | 285 (47) | |
Calcineurin inhibitor, with or without other | 50 (8) | 50 (8) | |
Other | 17 (2) | 14 (2) | |
Transplantation period | .91 | ||
2008-2012 | 221 (36) | 223 (36) | |
2013-2017 | 391 (64) | 389 (64) |
Data are the number of patients (percentage of subgroup), unless stated otherwise.
CY, cyclophosphamide.
Age distribution. Training cohort: n = 152 (25%) aged <3 y, n = 117 (19%) aged 3-6 y, n = 122 (20%) aged 7-11 y, and n = 221 (36%) aged 12-18 y. Validation cohort, n = 163 (27%) aged <3 y, n = 115 (19%) aged 3-6 y, n = 118 (19%) aged 7-11 y, and n = 216 (35%) aged 12-18 y.
Cytogenetic risk: favorable (inv(16), t(16;16), t(15;17), and t(8;21)) without complex abnormality; poor (−5/5q, −7/7q, FLT3/internal tandem duplication with high allelic ratio, t(6;9), 3q); intermediate (all others).