TABLE 2.
Characteristics of Patients in R2 Arms
| Characteristic | SIM + Pulses (n = 391) | SIM, No Pulses (n = 392) | HDM + Pulses (n = 393) | MTX, No Pulses (n = 394) | All (N = 1,570) |
|---|---|---|---|---|---|
| Age,a years, median (IQR) | 5.0 (3.0-11.0) | 5.0 (3.0-11.0) | 5.0 (3.0-11.0) | 5.0 (3.0-11.0) | 5 (3.0-11.0) |
| Range | 1-25 | 1-25 | 1-24 | 1-24 | 1-25 |
| Age (group), No. (%) | |||||
| Under 10 | 280 (71.6) | 281 (71.7) | 268 (68.2) | 274 (69.5) | 1,103 (70.3) |
| 10-15 | 65 (16.6) | 59 (15.1) | 75 (19.1) | 73 (18.5) | 272 (17.3) |
| 16 and over | 46 (11.8) | 52 (13.3) | 50 (12.7) | 47 (11.9) | 195 (12.4) |
| Ethnicity, No. (%) | |||||
| Asian—Bangladeshi | 2 (0.6) | 2 (0.6) | 1 (0.3) | 2 (0.6) | 7 (0.5) |
| Asian—Pakistani | 10 (2.9) | 7 (2.1) | 7 (2.0) | 9 (2.6) | 33 (2.4) |
| Asian—Indian | 8 (2.4) | 5 (1.5) | 11 (3.2) | 6 (1.7) | 30 (2.2) |
| Black—African | 1 (0.3) | 1 (0.3) | 3 (0.9) | 3 (0.9) | 8 (0.6) |
| Chinese | 0 | 1 (0.3) | 2 (0.6) | 2 (0.6) | 5 (0.4) |
| Mixed—White and Black African | 8 (2.4) | 5 (1.5) | 5 (1.4) | 3 (0.9) | 21 (1.5) |
| Mixed—White and Black Caribbean | 2 (0.6) | 2 (0.6) | 1 (0.3) | 0 | 5 (0.4) |
| Other Asian background | 9 (2.6) | 9 (2.6) | 10 (2.9) | 4 (1.2) | 32 (2.3) |
| Other Black background | 1 (0.3) | 1 (0.3) | 1 (0.3) | 1 (0.3) | 4 (0.3) |
| Other ethnic | 8 (2.4) | 6 (1.8) | 3 (0.9) | 6 (1.7) | 23 (1.7) |
| Other mixed background | 5 (1.5) | 9 (2.6) | 5 (1.4) | 13 (3.8) | 32 (2.3) |
| White | 286 (84.1) | 292 (85.9) | 296 (85.8) | 296 (85.8) | 1,170 (85.4) |
| Missing | 51 | 52 | 48 | 49 | 200 |
| Sex, No. (%) | |||||
| Male | 226 (57.8) | 230 (58.7) | 231 (58.8) | 231 (58.6) | 918 (58.5) |
| Female | 165 (42.2) | 162 (41.3) | 162 (41.2) | 163 (41.4) | 652 (41.5) |
| Down syndrome, No. (%) | |||||
| No | 391 (100.0) | 392 (100.0) | 393 (100.0) | 394 (100.0) | 1,570 (100.0) |
| CNS status, No. (%) | |||||
| 1 | 342 (93.4) | 353 (93.9) | 348 (91.8) | 340 (92.4) | 1,383 (92.9) |
| 2 | 13 (3.6) | 14 (3.7) | 17 (4.5) | 18 (4.9) | 62 (4.2) |
| 3 | 11 (3.0) | 9 (2.4) | 14 (3.7) | 10 (2.7) | 44 (3.0) |
| Missing | 25 | 16 | 14 | 26 | 81 |
| Did patient have a traumatic lumbar puncture? No. (%) | |||||
| No | 239 (87.5) | 241 (87.6) | 233 (85.0) | 240 (87.6) | 953 (87.0) |
| Yes | 34 (12.5) | 34 (12.4) | 41 (15.0) | 34 (12.5) | 143 (13.0) |
| Traumatic (without blasts) | 29 (85.3) | 26 (81.2) | 34 (82.9) | 29 (85.3) | 118 (83.7) |
| Traumatic (with blasts) | 5 (14.7) | 6 (18.8) | 7 (17.1) | 5 (14.7) | 23 (16.3) |
| Missing | 0 | 2 | 0 | 0 | 2 |
| Missing | 118 | 117 | 119 | 120 | 474 |
| High-risk cytogenetics,b No. (%) | |||||
| No | 362 (96.3) | 360 (94.7) | 361 (93.5) | 371 (96.4) | 1,454 (95.2) |
| Yes | 14 (3.7) | 20 (5.3) | 25 (6.5) | 14 (3.6) | 73 (4.8) |
| iAMP21 | 5 (41.7) | 7 (35.0) | 6 (27.3) | 4 (40.0) | 22 (34.4) |
| t(17;19) | 0 | 0 | 2 (9.1) | 0 | 2 (3.1) |
| KMT2A rearrangement | 7 (58.3) | 8 (40.0) | 10 (45.5) | 5 (50.0) | 30 (46.9) |
| Near haploidy | 0 | 2 (10.0) | 2 (9.1) | 0 | 4 (6.2) |
| Low hypodiploidy | 0 | 3 (15.0) | 2 (9.1) | 1 (10.0) | 6 (9.4) |
| Missing | 2 | 0 | 3 | 4 | 9 |
| Missing | 15 | 12 | 7 | 9 | 43 |
| Disease/immunophenotype, No. (%) | |||||
| B-ALL | 329 (84.1) | 327 (83.4) | 326 (83.0) | 331 (84.0) | 1,313 (83.6) |
| T-ALL | 41 (10.5) | 44 (11.2) | 45 (11.5) | 41 (10.4) | 171 (10.9) |
| B-LBL | 3 (0.8) | 7 (1.8) | 7 (1.8) | 3 (0.8) | 20 (1.3) |
| T-LBL | 18 (4.6) | 14 (3.6) | 15 (3.8) | 19 (4.8) | 66 (4.2) |
| R1, No. (%) | |||||
| Standard dex | 140 (35.8) | 141 (36.0) | 142 (36.1) | 139 (35.3) | 562 (35.8) |
| Short dex | 143 (36.6) | 143 (36.5) | 144 (36.6) | 145 (36.8) | 575 (36.6) |
| No R1 | 108 (27.6) | 108 (27.6) | 107 (27.2) | 110 (27.9) | 433 (27.6) |
| Regimen after induction, No. (%) | |||||
| A | 131 (33.5) | 124 (31.6) | 116 (29.5) | 126 (32.0) | 497 (31.7) |
| B | 87 (22.3) | 84 (21.4) | 95 (24.2) | 93 (23.6) | 359 (22.9) |
| C | 173 (44.2) | 184 (46.9) | 182 (46.3) | 175 (44.4) | 714 (45.5) |
| ALL | SIM + Pulses (n = 370) | SIM, No Pulses (n = 371) | HDM + Pulses (n = 371) | MTX, No Pulses (n = 372) | All (N = 1,484) |
|---|---|---|---|---|---|
| WCC, median (IQR) | 11.4 (4.5-41.0) | 11.2 (4.3-34.0) | 12.2 (4.4-40.2) | 12.0 (4.8-44.4) | 11.7 (4.5-41.0) |
| Range | 0.6-440 | 0.1-817 | 0.5-756.9 | 0.3-578.4 | 0.1-817 |
| WCC group, No. (%) | |||||
| <50 | 290 (78.4) | 293 (79.2) | 299 (80.6) | 288 (77.4) | 1,170 (78.9) |
| ≥50 | 80 (21.6) | 77 (20.8) | 72 (19.4) | 84 (22.6) | 313 (21.1) |
| Missing | 0 | 1 | 0 | 0 | 1 |
| NCI risk group, No. (%) | |||||
| Standard | 217 (58.6) | 211 (57.0) | 210 (56.6) | 205 (55.1) | 843 (56.8) |
| High | 153 (41.4) | 159 (43.0) | 161 (43.4) | 167 (44.9) | 640 (43.2) |
| Missing | 0 | 1 | 0 | 0 | 1 |
| MRD (R2), No. (%) | |||||
| MRD low risk | 202 (54.6) | 188 (50.7) | 186 (50.1) | 199 (53.5) | 775 (52.2) |
| MRD intermediate risk | 148 (40.0) | 163 (43.9) | 162 (43.7) | 151 (40.6) | 624 (42.0) |
| MRD no result | 20 (5.4) | 20 (5.4) | 23 (6.2) | 22 (5.9) | 85 (5.7) |
NOTE. R2 was a factorial randomisation to HDM or SIM and to pulses or no pulses in maintenance. This second randomization was stratified as per R1 (disease type [ALL/LBL], sex, age, and WCC) with two additional factors: MRD (low or intermediate risk) and R1 steroid (standard or short).
Abbreviations: dex, dexamethasone; HDM, high-dose methotrexate; LBL, lymphoblastic lymphoma; MRD, minimal residual disease; MTX, methotrexate; NCI, National Cancer Institute; R1, randomization 1; R2, randomization 2; SIM, standard interim maintenance; WCC, white cell count.
a
Includes two patients who were age 25 years at random assignment, but age 24 years at diagnosis, as per protocol inclusion criteria.