Characteristics of study population
| Variable . | Adults, CR1/CR2, MAC/RIC . | Pediatrics, CR1/CR2, MAC . | All ages PIF/relapse/CR ≥3, MAC . |
|---|---|---|---|
| No. of patients | 2593 | 1619 | 1003 |
| No. of centers | 232 | 165 | 205 |
| Patient age, y | |||
| 1-9 | 0 | 941 (58) | 187 (19) |
| 10-18 | 0 | 678 (42) | 225 (22) |
| 19-29 | 928 (36) | 0 | 281 (28) |
| 30-39 | 568 (22) | 0 | 139 (14) |
| 40-49 | 558 (22) | 0 | 107 (11) |
| 50-59 | 402 (16) | 0 | 57 (6) |
| ≥60 | 137 (5) | 0 | 7 (<1) |
| Median (range) | 36 (18-75) | 9 (1-18) | 21 (1-67) |
| Sex | |||
| Male | 1537 (59) | 1018 (63) | 651 (65) |
| Female | 1056 (41) | 601 (37) | 352 (35) |
| KPS score | |||
| <90% | 733 (28) | 205 (13) | 293 (29) |
| ≥90% | 1774 (68) | 1345 (83) | 648 (65) |
| Missing | 86 (3) | 69 (4) | 62 (6) |
| HCT–comorbidity index | |||
| 0 | 518 (20) | 449 (28) | 157 (16) |
| 1 | 165 (6) | 46 (3) | 30 (3) |
| 2 | 129 (5) | 14 (<1) | 18 (2) |
| ≥3 | 283 (11) | 34 (2) | 45 (4) |
| NA | 1498 (58) | 1076 (66) | 753 (75) |
| Philadelphia positive | |||
| No | 1574 (61) | 1315 (81) | 813 (81) |
| Yes | 957 (37) | 249 (15) | 150 (15) |
| Missing or untested | 62 (2) | 55 (3) | 40 (4) |
| Immunophenotype | |||
| T cell | 381 (15) | 262 (16) | 128 (13) |
| B cell | 2119 (82) | 1248 (77) | 804 (80) |
| Missing | 93 (4) | 109 (7) | 71 (7) |
| WBC at diagnosis by age group and lineage, × 109/L | |||
| T, ≤100 | 214 (8) | 105 (6) | 70 (7) |
| T, >100 | 66 (3) | 112 (7) | 30 (3) |
| Adult B, ≤30 | 1200 (46) | 0 | 216 (22) |
| Adult B, >30 | 531 (20) | 0 | 124 (12) |
| Pediatric B, ≤30 | 0 | 646 (40) | 157 (16) |
| Pediatric B, 30-50 | 0 | 86 (5) | 31 (3) |
| Pediatric B, >50 | 0 | 276 (17) | 54 (5) |
| Missing (WBC or lineage) | 582 (22) | 394 (24) | 321 (32) |
| Extramedullary disease at diagnosis | |||
| No | 2171 (84) | 1301 (80) | 819 (82) |
| Yes | 328 (13) | 270 (17) | 146 (15) |
| Missing | 94 (4) | 48 (3) | 38 (4) |
| Previous CNS disease | |||
| No | 2330 (90) | 1360 (84) | 871 (87) |
| Yes | 169 (7) | 211 (13) | 94 (9) |
| Missing | 94 (4) | 48 (3) | 38 (4) |
| Disease status before HCT | |||
| Primary induction failure | 0 | 0 | 145 (14) |
| CR1 | 1815 (70) | 644 (40) | 0 |
| CR2 | 778 (30) | 975 (60) | 0 |
| CR ≥3 | 0 | 0 | 403 (40) |
| Relapse | 0 | 0 | 455 (45) |
| Cytogenetics scoring | |||
| Normal karyotype | 768 (30) | 400 (25) | 301 (30) |
| Philadelphia chromosome | 957 (37) | 249 (15) | 150 (15) |
| Other poor risk | 230 (9) | 281 (17) | 95 (9) |
| SR | 428 (17) | 460 (28) | 212 (21) |
| Not tested/missing | 210 (8) | 229 (14) | 245 (24) |
| MRD at time of HCT | |||
| No | 183 (7) | 109 (7) | 25 (2) |
| Yes | 326 (13) | 134 (8) | 56 (6) |
| Missing | 2084 (80) | 1376 (85) | 922 (92) |
| Time from diagnosis to HCT (CR1 cases) | |||
| <6 mo | 1120 (43) | 378 (23) | 0 |
| 6-12 mo | 602 (23) | 217 (13) | 0 |
| >12 mo | 93 (4) | 49 (3) | 0 |
| Not applicable (CR2+/PIF/relapse) | 778 (30) | 975 (60) | 1003 |
| Median (range) | 7 (<1-21) | 17 (<1-44) | |
| Time to achieve CR1, median (range), wk | 7 (<1-113) | 5 (<1-167) | 5 (<1-252) |
| Conditioning regimen | |||
| MAC with TBI | 1917 (74) | 1478 (91) | 874 (87) |
| MAC without TBI | 317 (12) | 141 (9) | 129 (13) |
| RIC | 359 (14) | 0 | 0 |
| Graft type | |||
| Bone marrow | 659 (25) | 687 (42) | 331 (33) |
| Peripheral blood | 1611 (62) | 239 (15) | 431 (43) |
| Single cord | 87 (3) | 591 (37) | 168 (17) |
| Double cords | 236 (9) | 102 (6) | 73 (7) |
| Donor type | |||
| HLA-identical sibling | 903 (35) | 367 (23) | 197 (20) |
| Well-matched unrelated or 8/8 | 934 (36) | 328 (20) | 334 (33) |
| Partially/mismatched unrelated or ≤7/8 | 433 (17) | 231 (14) | 231 (23) |
| Cord blood | 323 (12) | 693 (43) | 241 (24) |
| Donor–recipient sex match | |||
| Male–male | 951 (37) | 548 (34) | 382 (38) |
| Male–female | 562 (22) | 330 (20) | 191 (19) |
| Female–male | 559 (22) | 424 (26) | 248 (25) |
| Female–female | 473 (18) | 246 (15) | 147 (15) |
| Missing | 48 (2) | 71 (4) | 35 (3) |
| Donor–recipient CMV serology | |||
| +/+ | 763 (29) | 249 (15) | 227 (23) |
| +/− | 255 (10) | 115 (7) | 80 (8) |
| −/+ | 781 (30) | 538 (33) | 346 (34) |
| −/− | 676 (26) | 665 (41) | 302 (30) |
| Missing | 118 (5) | 52 (3) | 48 (5) |
| URD age, median (range), y | 33 (19-57) | 33 (18-57) | 34 (19-57) |
| GVHD prophylaxis | |||
| CNI + MTX | 1684 (65) | 882 (54) | 621 (62) |
| CNI + MMF | 549 (21) | 327 (20) | 207 (21) |
| Other | 357 (14) | 410 (25) | 172 (17) |
| Missing | 3 (<1) | 0 | 3 (<1) |
| In vivo TCD (ATG or alemtuzumab) | |||
| No | 2048 (79) | 991 (61) | 712 (71) |
| Yes | 544 (21) | 628 (39) | 290 (29) |
| Missing | 1 (<1) | 0 | 1 (<1) |
| Year of HCT | |||
| 2000-2003 | 597 (23) | 494 (31) | 376 (37) |
| 2004-2007 | 897 (35) | 580 (36) | 372 (37) |
| 2008-2011 | 619 (24) | 337 (21) | 175 (17) |
| 2012-2014 | 480 (19) | 208 (13) | 80 (8) |
| Median follow-up of survivors (range), mo | 70 (2-184) | 72 (2-191) | 86 (3-176) |
| Variable . | Adults, CR1/CR2, MAC/RIC . | Pediatrics, CR1/CR2, MAC . | All ages PIF/relapse/CR ≥3, MAC . |
|---|---|---|---|
| No. of patients | 2593 | 1619 | 1003 |
| No. of centers | 232 | 165 | 205 |
| Patient age, y | |||
| 1-9 | 0 | 941 (58) | 187 (19) |
| 10-18 | 0 | 678 (42) | 225 (22) |
| 19-29 | 928 (36) | 0 | 281 (28) |
| 30-39 | 568 (22) | 0 | 139 (14) |
| 40-49 | 558 (22) | 0 | 107 (11) |
| 50-59 | 402 (16) | 0 | 57 (6) |
| ≥60 | 137 (5) | 0 | 7 (<1) |
| Median (range) | 36 (18-75) | 9 (1-18) | 21 (1-67) |
| Sex | |||
| Male | 1537 (59) | 1018 (63) | 651 (65) |
| Female | 1056 (41) | 601 (37) | 352 (35) |
| KPS score | |||
| <90% | 733 (28) | 205 (13) | 293 (29) |
| ≥90% | 1774 (68) | 1345 (83) | 648 (65) |
| Missing | 86 (3) | 69 (4) | 62 (6) |
| HCT–comorbidity index | |||
| 0 | 518 (20) | 449 (28) | 157 (16) |
| 1 | 165 (6) | 46 (3) | 30 (3) |
| 2 | 129 (5) | 14 (<1) | 18 (2) |
| ≥3 | 283 (11) | 34 (2) | 45 (4) |
| NA | 1498 (58) | 1076 (66) | 753 (75) |
| Philadelphia positive | |||
| No | 1574 (61) | 1315 (81) | 813 (81) |
| Yes | 957 (37) | 249 (15) | 150 (15) |
| Missing or untested | 62 (2) | 55 (3) | 40 (4) |
| Immunophenotype | |||
| T cell | 381 (15) | 262 (16) | 128 (13) |
| B cell | 2119 (82) | 1248 (77) | 804 (80) |
| Missing | 93 (4) | 109 (7) | 71 (7) |
| WBC at diagnosis by age group and lineage, × 109/L | |||
| T, ≤100 | 214 (8) | 105 (6) | 70 (7) |
| T, >100 | 66 (3) | 112 (7) | 30 (3) |
| Adult B, ≤30 | 1200 (46) | 0 | 216 (22) |
| Adult B, >30 | 531 (20) | 0 | 124 (12) |
| Pediatric B, ≤30 | 0 | 646 (40) | 157 (16) |
| Pediatric B, 30-50 | 0 | 86 (5) | 31 (3) |
| Pediatric B, >50 | 0 | 276 (17) | 54 (5) |
| Missing (WBC or lineage) | 582 (22) | 394 (24) | 321 (32) |
| Extramedullary disease at diagnosis | |||
| No | 2171 (84) | 1301 (80) | 819 (82) |
| Yes | 328 (13) | 270 (17) | 146 (15) |
| Missing | 94 (4) | 48 (3) | 38 (4) |
| Previous CNS disease | |||
| No | 2330 (90) | 1360 (84) | 871 (87) |
| Yes | 169 (7) | 211 (13) | 94 (9) |
| Missing | 94 (4) | 48 (3) | 38 (4) |
| Disease status before HCT | |||
| Primary induction failure | 0 | 0 | 145 (14) |
| CR1 | 1815 (70) | 644 (40) | 0 |
| CR2 | 778 (30) | 975 (60) | 0 |
| CR ≥3 | 0 | 0 | 403 (40) |
| Relapse | 0 | 0 | 455 (45) |
| Cytogenetics scoring | |||
| Normal karyotype | 768 (30) | 400 (25) | 301 (30) |
| Philadelphia chromosome | 957 (37) | 249 (15) | 150 (15) |
| Other poor risk | 230 (9) | 281 (17) | 95 (9) |
| SR | 428 (17) | 460 (28) | 212 (21) |
| Not tested/missing | 210 (8) | 229 (14) | 245 (24) |
| MRD at time of HCT | |||
| No | 183 (7) | 109 (7) | 25 (2) |
| Yes | 326 (13) | 134 (8) | 56 (6) |
| Missing | 2084 (80) | 1376 (85) | 922 (92) |
| Time from diagnosis to HCT (CR1 cases) | |||
| <6 mo | 1120 (43) | 378 (23) | 0 |
| 6-12 mo | 602 (23) | 217 (13) | 0 |
| >12 mo | 93 (4) | 49 (3) | 0 |
| Not applicable (CR2+/PIF/relapse) | 778 (30) | 975 (60) | 1003 |
| Median (range) | 7 (<1-21) | 17 (<1-44) | |
| Time to achieve CR1, median (range), wk | 7 (<1-113) | 5 (<1-167) | 5 (<1-252) |
| Conditioning regimen | |||
| MAC with TBI | 1917 (74) | 1478 (91) | 874 (87) |
| MAC without TBI | 317 (12) | 141 (9) | 129 (13) |
| RIC | 359 (14) | 0 | 0 |
| Graft type | |||
| Bone marrow | 659 (25) | 687 (42) | 331 (33) |
| Peripheral blood | 1611 (62) | 239 (15) | 431 (43) |
| Single cord | 87 (3) | 591 (37) | 168 (17) |
| Double cords | 236 (9) | 102 (6) | 73 (7) |
| Donor type | |||
| HLA-identical sibling | 903 (35) | 367 (23) | 197 (20) |
| Well-matched unrelated or 8/8 | 934 (36) | 328 (20) | 334 (33) |
| Partially/mismatched unrelated or ≤7/8 | 433 (17) | 231 (14) | 231 (23) |
| Cord blood | 323 (12) | 693 (43) | 241 (24) |
| Donor–recipient sex match | |||
| Male–male | 951 (37) | 548 (34) | 382 (38) |
| Male–female | 562 (22) | 330 (20) | 191 (19) |
| Female–male | 559 (22) | 424 (26) | 248 (25) |
| Female–female | 473 (18) | 246 (15) | 147 (15) |
| Missing | 48 (2) | 71 (4) | 35 (3) |
| Donor–recipient CMV serology | |||
| +/+ | 763 (29) | 249 (15) | 227 (23) |
| +/− | 255 (10) | 115 (7) | 80 (8) |
| −/+ | 781 (30) | 538 (33) | 346 (34) |
| −/− | 676 (26) | 665 (41) | 302 (30) |
| Missing | 118 (5) | 52 (3) | 48 (5) |
| URD age, median (range), y | 33 (19-57) | 33 (18-57) | 34 (19-57) |
| GVHD prophylaxis | |||
| CNI + MTX | 1684 (65) | 882 (54) | 621 (62) |
| CNI + MMF | 549 (21) | 327 (20) | 207 (21) |
| Other | 357 (14) | 410 (25) | 172 (17) |
| Missing | 3 (<1) | 0 | 3 (<1) |
| In vivo TCD (ATG or alemtuzumab) | |||
| No | 2048 (79) | 991 (61) | 712 (71) |
| Yes | 544 (21) | 628 (39) | 290 (29) |
| Missing | 1 (<1) | 0 | 1 (<1) |
| Year of HCT | |||
| 2000-2003 | 597 (23) | 494 (31) | 376 (37) |
| 2004-2007 | 897 (35) | 580 (36) | 372 (37) |
| 2008-2011 | 619 (24) | 337 (21) | 175 (17) |
| 2012-2014 | 480 (19) | 208 (13) | 80 (8) |
| Median follow-up of survivors (range), mo | 70 (2-184) | 72 (2-191) | 86 (3-176) |
Data are n (%) unless otherwise indicated.
ATG, antithymocyte globulin; CNS, central nervous system; NA, not applicable (before the year 2007); PIF, primary induction failure; TCD, T-cell depletion; TKI, tyrosine kinase inhibitor; WBC, white blood cell.