Table 1.
Patient characteristics.
| Patient characteristics | CR1 | CR2 | |||||||
|---|---|---|---|---|---|---|---|---|---|
| Total | CCa | FTBIb | Total | CCc | FTBId | ||||
| Variable | Statistic/level | n = 1498 | n = 213 | n = 1285 | P value | n = 1556 | n = 211 | n = 1345 | P value |
| Follow-up (years) | Median [95% CI] | 6.3 [6.2–6.6] | 6.1 [6.0–6.3] | 6.8 [6.4–6.7.0] | <0.0001 | 6.2 [6.0–6.5] | 6.2 [6.2–6.5] | 6.2 [6.0–6.8] | 0.36 |
| Age (years) | Median (range) [IQR] | 11.2 (2.0–18.0) [7.1–15.4] | 8.8 (2.0–18.0) [4.3–14.6] | 11.5 (2.0–18.0) [7.7–15.5] | <0.0001 | 9.6 (2.0–18.0) [6.8–13.6] | 8.7 (2.0–17.9) [4.7–12.9] | 9.7 (2.1–18.0) [6.9–13.7] | 0.0003 |
| Patient sex | Male | 988 (66.1%) | 134 (63.8%) | 854 (66.5%) | 0.44 | 1034 (66.6%) | 146 (69.9%) | 888 (66.1%) | 0.28 |
| Female | 506 (33.9%) | 76 (36.2%) | 430 (33.5%) | 519 (33.4%) | 63 (30.1%) | 456 (33.9%) | |||
| Missing | 4 | 3 | 1 | – | 3 | 2 | 1 | – | |
| Immunophenotype | B | 908 (64.2%) | 146 (70.2%) | 762 (63.1%) | 0.13 | 1157 (80.6%) | 135 (70.0%) | 1022 (82.3%) | <0.0001 |
| T | 448 (31.6%) | 56 (26.9%) | 392 (32.5%) | 204 (14.2%) | 32 (16.6%) | 172 (13.8%) | |||
| Other | 59 (4.2%) | 6 (2.9%) | 53 (4.4%) | 74 (5.2%) | 26 (13.4%) | 48 (3.9%) | |||
| Missing | 83 | 5 | 78 | – | 121 | 18 | 103 | – | |
| Year of HSCT | Median (range) [IQR] | 2007 (2000–2012) [2004–2010] | 2010 (2000–2012) [2007–2011] | 2007 (2000–2012) [2004–2010] | <0.0001 | 2007 (2000–2012) [2004–2010] | 2009 (2000–2012) [2005–2011] | 2007 (2000–2012) [2004–2010] | <0.0001 |
| Diagnosis to HSCT (months) | Median (range) [IQR] | 6.7 (1.3–84.4) [5.5–8.3] | 7.5 (2.0–78.8) [6.2–9.5] | 6.6 (1.2–84.4) [5.5–8.2] | <0.0001 | 34.6 (1.3–159.7) [22.4–48.7] | 30.6 (5.1–145.4) [20.1–47.1] | 35.5 (1.3–159.7) [23.2–49.1] | 0.02 |
| Donor type | MRD | 756 (50.5%) | 109 (51.2%) | 647 (50.3%) | 0.82 | 672 (43.2%) | 102 (48.3%) | 570 (42.4%) | 0.1 |
| UD | 742 (49.5%) | 104 (48.8%) | 638 (49.7%) | 884 (56.8%) | 109 (51.7%) | 775 (57.6%) | |||
| Stem cell sources | BM | 1041 (69.5%) | 121 (56.8%) | 920 (71.6%) | <0.0001 | 1064 (68.4%) | 102 (48.3%) | 962 (71.5%) | <0.0001 |
| PBSC | 457 (30.5%) | 92 (43.2%) | 365 (28.4%) | 492 (31.6%) | 109 (51.7%) | 383 (28.5%) | |||
| Donor to patient sex mismatch | Female to male | 374 (25.2%) | 58 (27.4%) | 316 (24.9%) | 0.44 | 400 (25.8%) | 63 (30.0%) | 337 (25.2%) | 0.14 |
| Other | 1109 (74.8%) | 154 (72.6%) | 955 (75.1%) | 1149 (74.2%) | 147 (70.0%) | 1002 (74.8%) | |||
| Missing | 15 | 1 | 14 | – | 7 | 1 | 6 | – | |
| TBI dose (Gy) | Median (range) [IQR] | – | – | 12.0 (7.5–16.0) [12.0–12.0] | – | – | – | 12.0 (7.5–16.0) [12.0–12.0] | – |
| Missing | – | – | 263 | – | – | – | 294 | – | |
| Busulfan dose (mg/kgBW) | Median (range) [IQR] | – | 16.0 (9.6–20.0) [12.8–17.6] | – | – | – | 16.0 (9.6–20.0) [12.8–16.0] | – | – |
| Missing | – | 30 | – | – | – | 30 | – | – | |
| White blood count (cells/nl) at diagnosis | Median (range) [IQR] | 45.0 (0.2–900.0) [8.0–157.7] | 36.6 (0.3–862.0) [12.3–142.0] | 46.0 (0.2–900.0) [8.0–159.1] | 0.798 | 19.1 (0.1–672.0) [6.0–70.6] | 26.0 (0.6–600.0) [6.9–96.8] | 18.7 (0.1–672.0) [5.8–64.7] | 0.22 |
| Missing | 949 | 160 | 789 | – | 1100 | 160 | 940 | – | |
| CMV, donor/patient | −/− | 419 (34.8%) | 41 (22.5%) | 378 (36.9%) | 0.0003 | 426 (34.4%) | 48 (25.2%) | 378 (36.0%) | 0.006 |
| −/+ | 228 (18.9%) | 48 (26.4%) | 180 (17.6%) | 229 (18.5%) | 37 (19.5%) | 192 (18.3%) | |||
| +/− | 122 (10.1%) | 15 (8.2%) | 107 (10.5%) | 141 (11.4%) | 18 (9.5%) | 123 (11.7%) | |||
| +/+ | 436 (36.2%) | 78 (42.9%) | 358 (35.0%) | 443 (35.7%) | 87 (45.8%) | 356 (34.0%) | |||
| Missing | 293 | 31 | 262 | – | 317 | 21 | 296 | – | |
| Acute GvHD | No aGvHD | 587 (40.6%) | 112 (53.8%) | 475 (38.3%) | – | 550 (37.0%) | 99 (48.3%) | 451 (35.2%) | – |
| Grade I | 318 (22.0%) | 27 (13.0%) | 291 (23.5%) | 321 (21.6%) | 28 (13.7%) | 293 (22.9%) | |||
| Grade II | 366 (25.3%) | 39 (18.8%) | 327 (26.4%) | 418 (28.2%) | 52 (25.4%) | 366 (28.6%) | |||
| Grade III | 118 (8.1%) | 20 (9.6%) | 98 (7.9%) | 131 (8.8%) | 13 (6.3%) | 118 (9.2%) | |||
| Grade IV | 58 (4.0%) | 10 (4.8%) | 48 (3.9%) | 65 (4.4%) | 13 (6.3%) | 52 (4.1%) | |||
| Missing | 51 | 5 | 46 | – | 71 | 6 | 65 | – | |
| Chronic GvHD | No | 1041 (78.6%) | 159 (83.7%) | 882 (77.8%) | – | 1119 (80.9%) | 143 (77.3%) | 976 (81.5%) | – |
| Yes | 283 (21.4%) | 31 (16.3%) | 252 (22.2%) | 264 (19.1%) | 42 (22.7%) | 222 (18.5%) | |||
| Missing | 174 | 23 | 151 | – | 173 | 26 | 147 | – | |
| Extensive cGvHD | No | 1166 (89.3%) | 173 (91.0%) | 993 (89.0%) | – | 1232 (90.0%) | 161 (87.0%) | 1071 (90.5%) | – |
| Yes | 140 (10.7%) | 17 (9.0.%) | 123 (11.0%) | 136 (10.0%) | 24 (13.0%) | 112 (9.5%) | |||
| Missing | 192 | 23 | 169 | – | 188 | 26 | 162 | – | |
| Engraftment | No | 10 (0.7%) | 4 (1.9%) | 6 (0.5%) | – | 28 (1.8%) | 3 (1.5%) | 25 (1.9%) | – |
| Yes | 1470 (99.3%) | 208 (98.1%) | 1262 (99.5%) | 1505 (98.2%) | 204 (98.5%) | 1301 (98.1%) | |||
| Missing | 18 | 1 | 17 | – | 23 | 4 | 19 | – | |
Variables not shown: age (categorial), diagnosis to HSCT (categorial), donor (sex), and donor to patient sex.
AraC cytarabine, BM bone marrow, Bu busulfan, CC chemotherapy-based conditioning, CI confidence interval, CMV cytomegalovirus, CR1 first complete remission, CR2 second complete remission, Cy cyclophosphamide, Eto etoposide, Flu fludarabine, FTBI fractionated total body irradiation, GvHD graft versus host disease, HSCT hematopoietic stem cell transplantation, IQR interquartile range, Mel melphalan, MRD matched-related donor, PBSC peripheral blood stem cell, Thio thiotepa, UD unrelated donor.
aCC cohort (n = 213) includes: Bu/Cy (n = 68), Bu/Cy/Eto (n = 66), Bu/AraC/+/−Mel (n = 23), Bu/Cy/Mel (n = 20), Bu/Flu (n = 20), Bu/Cy/Thio (n = 14), Bu/Flu/Thio (n = 2).
bFTBI cohort (n = 1285) includes: FTBI/Cy (n = 494), FTBI/Eto (n = 419), FTBI/other (n = 245), FTBI/Cy/Eto (n = 78), FTBI/Mel (n = 44), FTBI/Cy/Flu (n = 5).
cCC cohort (n = 211) includes: Bu/Cy (n = 68), Bu/Cy/Eto (n = 52), Bu/AraC/+/−Mel (n = 35), Bu/Cy/Thio (n = 18), Bu/Cy/Mel (n = 17), Bu/Flu (n = 13), Bu/Flu/Thio (n = 8).
dFTBI cohort (n = 1345) includes: FTBI/Cy (n = 496), FTBI/Eto (n = 365), FTBI/other (n = 291), FTBI/Cy/Eto (n = 91), FTBI/Mel (n = 86), FTBI/Cy/Flu (n = 16).