Patient characteristics of the overall cohort, the derivation cohort, and the validation cohort
| Characteristic . | Overall cohort . | Derivation cohort . | Validation cohort . | P . |
|---|---|---|---|---|
| N (%) | 616 (100) | 308 (50) | 308 (50) | |
| HCT time period | August 1999-May 2015 | August 1999-April 2009 | May 2009-May 2015 | |
| Method, n (%) | .001 | |||
| MFC | 260 (58) | 110 (36) | 150 (49) | |
| qPCR | 356 (42) | 198 (64) | 158 (51) | |
| MRD categories, n (%) | <.001 | |||
| Negative | 331 (54) | 174 (56) | 157 (51) | |
| Low | 107 (17) | 32 (10) | 75 (24) | |
| High | 82 (13) | 49 (16) | 33 (11) | |
| Very high | 96 (16) | 53 (17) | 43 (14) | |
| Age at HCT, y | .279 | |||
| Median | 9.6 | 9.2 | 9.8 | |
| 1st quartile-3rd quartile | 6.0-14 | 6.0-14 | 6.0-14 | |
| Range | 0.5-21.9 | 0.5-21.9 | 0.7-21.4 | |
| Age groups, n (%) | .612 | |||
| <2 y | 26 (4) | 13 (4) | 13 (4) | |
| 2 to 10 y | 308 (50) | 160 (52) | 148 (48) | |
| ≥10 y | 282 (46) | 135 (44) | 147 (48) | |
| Sex, n (%) | .803 | |||
| Female | 234 (38) | 115 (37) | 119 (39) | |
| Male | 382 (62) | 193 (63) | 189 (61) | |
| Remission status, n (%) | .164 | |||
| CR1 | 236 (38) | 112 (36) | 124 (40) | |
| CR2 | 339 (55) | 170 (55) | 169 (55) | |
| >CR2 | 41 (7) | 26 (8) | 15 (5) | |
| Subgroup CR2, n (%) | ||||
| Time to relapse | .001 | |||
| Very early | 72 (21) | 34 (20) | 38 (22) | |
| Early | 114 (34) | 53 (31) | 61 (36) | |
| Late | 129 (38) | 62 (36) | 67 (40) | |
| No data | 24 (7) | 21 (12) | 3 (2) | |
| Site to relapse | .007 | |||
| Isolated BM | 240 (71) | 132 (78) | 108 (64) | |
| Isolatedextramedullary | 45 (13) | 13 (8) | 32 (19) | |
| BM combined | 51 (15) | 24 (14) | 27 (16) | |
| No data | 3 (1) | 1 (1) | 2 (1) | |
| Immunophenotype, n (%) | .266 | |||
| BCP | 494 (80) | 253 (82) | 241 (78) | |
| T lineage | 122 (20) | 55 (18) | 67 (22) | |
| BCR/ABL rearrangement, n (%) | .501 | |||
| Negative | 445 (72) | 224 (73) | 221 (72) | |
| Positive | 54 (9) | 30 (10) | 24 (8) | |
| No data | 117 (19) | 54 (18) | 63 (20) | |
| MLL rearrangement, n (%) | .011 | |||
| Negative | 374 (61) | 201 (65) | 173 (56) | |
| Positive | 22 (4) | 14 (5) | 8 (3) | |
| No data | 220 (36) | 93 (30) | 127 (41) | |
| Donor, n (%) | <.001 | |||
| MSD | 191 (31) | 98 (32) | 93 (30) | |
| MUD | 259 (42) | 142 (46) | 117 (38) | |
| MMD | 56 (9) | 36 (12) | 20 (6) | |
| Cord | 110 (18) | 32 (10) | 78 (25) | |
| Stem cell source, n (%) | <.001 | |||
| BM | 385 (62.5) | 200 (65) | 185 (60) | |
| PB | 104 (16.9) | 67 (22) | 37 (12) | |
| CB | 127 (20.6) | 41 (13) | 86 (28) | |
| Conditioning, n (%) | .044 | |||
| TBI-based | 521 (84.6) | 251 (81) | 270 (88) | |
| Non–TBI-based | 95 (15.4) | 57 (19) | 38 (12) |
| Characteristic . | Overall cohort . | Derivation cohort . | Validation cohort . | P . |
|---|---|---|---|---|
| N (%) | 616 (100) | 308 (50) | 308 (50) | |
| HCT time period | August 1999-May 2015 | August 1999-April 2009 | May 2009-May 2015 | |
| Method, n (%) | .001 | |||
| MFC | 260 (58) | 110 (36) | 150 (49) | |
| qPCR | 356 (42) | 198 (64) | 158 (51) | |
| MRD categories, n (%) | <.001 | |||
| Negative | 331 (54) | 174 (56) | 157 (51) | |
| Low | 107 (17) | 32 (10) | 75 (24) | |
| High | 82 (13) | 49 (16) | 33 (11) | |
| Very high | 96 (16) | 53 (17) | 43 (14) | |
| Age at HCT, y | .279 | |||
| Median | 9.6 | 9.2 | 9.8 | |
| 1st quartile-3rd quartile | 6.0-14 | 6.0-14 | 6.0-14 | |
| Range | 0.5-21.9 | 0.5-21.9 | 0.7-21.4 | |
| Age groups, n (%) | .612 | |||
| <2 y | 26 (4) | 13 (4) | 13 (4) | |
| 2 to 10 y | 308 (50) | 160 (52) | 148 (48) | |
| ≥10 y | 282 (46) | 135 (44) | 147 (48) | |
| Sex, n (%) | .803 | |||
| Female | 234 (38) | 115 (37) | 119 (39) | |
| Male | 382 (62) | 193 (63) | 189 (61) | |
| Remission status, n (%) | .164 | |||
| CR1 | 236 (38) | 112 (36) | 124 (40) | |
| CR2 | 339 (55) | 170 (55) | 169 (55) | |
| >CR2 | 41 (7) | 26 (8) | 15 (5) | |
| Subgroup CR2, n (%) | ||||
| Time to relapse | .001 | |||
| Very early | 72 (21) | 34 (20) | 38 (22) | |
| Early | 114 (34) | 53 (31) | 61 (36) | |
| Late | 129 (38) | 62 (36) | 67 (40) | |
| No data | 24 (7) | 21 (12) | 3 (2) | |
| Site to relapse | .007 | |||
| Isolated BM | 240 (71) | 132 (78) | 108 (64) | |
| Isolatedextramedullary | 45 (13) | 13 (8) | 32 (19) | |
| BM combined | 51 (15) | 24 (14) | 27 (16) | |
| No data | 3 (1) | 1 (1) | 2 (1) | |
| Immunophenotype, n (%) | .266 | |||
| BCP | 494 (80) | 253 (82) | 241 (78) | |
| T lineage | 122 (20) | 55 (18) | 67 (22) | |
| BCR/ABL rearrangement, n (%) | .501 | |||
| Negative | 445 (72) | 224 (73) | 221 (72) | |
| Positive | 54 (9) | 30 (10) | 24 (8) | |
| No data | 117 (19) | 54 (18) | 63 (20) | |
| MLL rearrangement, n (%) | .011 | |||
| Negative | 374 (61) | 201 (65) | 173 (56) | |
| Positive | 22 (4) | 14 (5) | 8 (3) | |
| No data | 220 (36) | 93 (30) | 127 (41) | |
| Donor, n (%) | <.001 | |||
| MSD | 191 (31) | 98 (32) | 93 (30) | |
| MUD | 259 (42) | 142 (46) | 117 (38) | |
| MMD | 56 (9) | 36 (12) | 20 (6) | |
| Cord | 110 (18) | 32 (10) | 78 (25) | |
| Stem cell source, n (%) | <.001 | |||
| BM | 385 (62.5) | 200 (65) | 185 (60) | |
| PB | 104 (16.9) | 67 (22) | 37 (12) | |
| CB | 127 (20.6) | 41 (13) | 86 (28) | |
| Conditioning, n (%) | .044 | |||
| TBI-based | 521 (84.6) | 251 (81) | 270 (88) | |
| Non–TBI-based | 95 (15.4) | 57 (19) | 38 (12) |
P values were determined by using Fisher’s exact test, χ-quadrant test, or Wilcoxon-Mann-Whitney test; no data values were excluded. The variables time to relapse and site of relapse were available for all patients with remission = CR2 at HCT. The percentages of patients with both variables were calculated considering patients with CR2 as 100%.
BCP, B-cell precursor; BM, bone marrow; CB, cord blood; MMD, mismatched donor; MUD, matched unrelated donor; MSD, matched sibling donor; PB, peripheral blood.