Table 1.
Baseline demographics and disease characteristics of the HSCT population
| Characteristic | MOD | No MOD | All HSCT | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Overall (n = 512) | >16 years (n = 231) | ≤16 years (n = 281) | Overall (n = 488) | >16 years (n = 199) | ≤16 years (n = 289) | Overall (n = 1000) | >16 years (n = 430) | ≤16 years (n = 570) | |
| Sex, n (%) | |||||||||
| Male | 278 (54·3) | 119 (51·5) | 159 (56·6) | 290 (59·4) | 118 (59·3) | 172 (59·5) | 568 (56·8) | 237 (55·1) | 331 (58·1) |
| Female | 234 (45·7) | 112 (48·5) | 122 (43·4) | 198 (40·6) | 81 (40·7) | 117 (40·5) | 432 (43·2) | 193 (44·9) | 239 (41·9) |
| Race, n (%) | |||||||||
| White | 343 (67·0) | 175 (75·8) | 168 (59·8) | 284 (58·2) | 135 (67·8) | 149 (51·6) | 627 (62·7) | 310 (72·1) | 317 (55·6) |
| African American | 36 (7·0) | 13 (5·6) | 23 (8·2) | 44 (9·0) | 16 (8·0) | 28 (9·7) | 80 (8·0) | 29 (6·7) | 51 (8·9) |
| Asian | 20 (3·9) | 9 (3·9) | 11 (3·9) | 34 (7·0) | 13 (6·5) | 21 (7·3) | 54 (5·4) | 22 (5·1) | 32 (5·6) |
| Latino | 71 (13·9) | 23 (10·0) | 48 (17·1) | 92 (18·9) | 27 (13·6) | 65 (22·5) | 163 (16·3) | 50 (11·6) | 113 (19·8) |
| Age at time of HSCT, years, median (range) | 14·00 (0·10, 69·00) | 41·00 (17·00, 69·00) | 4·00 (0·10, 16·00) | 12·00 (0·10, 77·00) | 43·00 (17·00, 77·00) | 3·00 (0·10, 16·00) | 14·00 (0·10, 77·00) | 42·00 (17·00, 77·00) | 4·00 (0·10, 16·00) |
| Conditioning agents (>10%), n (%) | |||||||||
| Cyclophosphamide | 333 (65·0) | 154 (66·7) | 179 (63·7) | 241 (49·4) | 93 (46·7) | 148 (51·2) | 574 (57·4) | 247 (57·4) | 327 (57·4) |
| Busulfan | 262 (51·2) | 100 (43·3) | 162 (57·7) | 301 (61·7) | 101 (50·8) | 200 (69·2) | 563 (56·3) | 201 (46·7) | 362 (63·5) |
| Fludarabine | 165 (32·2) | 88 (38·1) | 77 (27·4) | 177 (36·3) | 97 (48·7) | 80 (27·7) | 342 (34·2) | 185 (43·0) | 157 (27·5) |
| Anti‐thymocyte globulin | 154 (30·1) | 35 (15·2) | 119 (42·3) | 152 (31·1) | 41 (20·6) | 111 (38·4) | 306 (30·6) | 76 (17·7) | 230 (40·4) |
| Total body irradiation | 172 (33·6) | 104 (45·0) | 68 (24·2) | 115 (23·6) | 66 (33·2) | 49 (17·0) | 287 (28·7) | 170 (39·5) | 117 (20·5) |
| Melphalan | 117 (22·9) | 30 (13·0) | 87 (31·0) | 142 (29·1) | 38 (19·1) | 104 (36·0) | 259 (25·9) | 68 (15·8) | 191 (33·5) |
| Thiotepa | 64 (12·5) | 26 (11·3) | 38 (13·5) | 70 (14·3) | 20 (10·1) | 50 (17·3) | 134 (13·4) | 46 (10·7) | 88 (15·4) |
| GVHD prophylaxis, n (%) | |||||||||
| Tacrolimus | 250 (48·8) | 162 (70·1) | 88 (31·3) | 233 (47·7) | 137 (68·8) | 96 (33·2) | 483 (48·3) | 299 (69·5) | 184 (32·3) |
| Methotrexate | 168 (32·8) | 101 (43·7) | 67 (23·8) | 164 (33·6) | 82 (41·2) | 82 (28·4) | 332 (33·2) | 183 (42·6) | 149 (26·1) |
| Ciclosporin | 159 (31·1) | 38 (16·5) | 121 (43·1) | 123 (25·2) | 26 (13·1) | 97 (33·6) | 282 (28·2) | 64 (14·9) | 218 (38·2) |
| Sirolimus | 59 (11·5) | 47 (20·3) | 12 (4·3) | 30 (6·1) | 26 (13·1) | 4 (1·4) | 89 (8·9) | 73 (17·0) | 16 (2·8) |
| None | 73 (14·3) | 14 (6·1) | 59 (21·0) | 108 (22·1) | 24 (12·1) | 84 (29·1) | 181 (18·1) | 38 (8·8) | 143 (25·1) |
| Othera | 182 (35·5) | 74 (32·0) | 108 (38·4) | 160 (32·8) | 66 (33·2) | 94 (32·5) | 342 (34·2) | 140 (32·6) | 202 (35·4) |
| Transplant type,b n (%) | |||||||||
| Allogeneic | 450 (87·9) | 220 (95·2) | 230 (81·9) | 393 (80·5) | 182 (91·5) | 211 (73·0) | 843 (84·3) | 402 (93·5) | 441 (77·4) |
| Autologous | 61 (11·9) | 11 (4·8) | 50 (17·8) | 94 (19·3) | 17 (8·5) | 77 (26·6) | 155 (15·5) | 28 (6·5) | 127 (22·3) |
| Primary disease (>10%), n (%) | |||||||||
| AML | 142 (27·7) | 88 (38·1) | 54 (19·2) | 119 (24·4) | 61 (30·7) | 58 (20·1) | 261 (26·1) | 149 (34·7) | 112 (19·6) |
| ALL | 111 (21·7) | 48 (20·8) | 63 (22·4) | 90 (18·4) | 49 (24·6) | 41 (14·2) | 201 (20·1) | 97 (22·6) | 104 (18·2) |
| Neuroblastoma | 43 (8·4) | 1 (0·4) | 42 (14·9) | 62 (12·7) | 0 (0) | 62 (21·5) | 105 (10·5) | 1 (0·2) | 104 (18·2) |
| Prior HSCTs, n (%) | |||||||||
| 0 | 428 (83·6) | 189 (81·8) | 239 (85·1) | 401 (82·2) | 162 (81·4) | 239 (82·7) | 829 (82·9) | 351 (81·6) | 478 (83·9) |
| ≥1 | 82 (16·0) | 41 (17·7) | 41 (14·6) | 83 (17·0) | 35 (17·6) | 48 (16·6) | 165 (16·5) | 76 (17·7) | 89 (15·6) |
| Unknown/missing | 2 (0·4) | 1 (0·4) | 1 (0·4) | 4 (0·8) | 2 (1·0) | 2 (0·7) | 6 (0·6) | 3 (0·7) | 3 (0·5) |
| VOD/SOS onset after Day 21, n (%) | 139 (27·1) | 88 (38·1) | 51 (18·1) | 125 (25·6) | 81 (40·7) | 44 (15·2) | 264 (26·4) | 169 (39·3) | 95 (16·7) |
| Time from diagnosis to start of defibrotide, n (%) | |||||||||
| Day of diagnosis | 156 (30·4) | 68 (29·4) | 88 (31·3) | 154 (31·6) | 52 (26·1) | 102 (35·3) | 310 (31·0) | 120 (27·9) | 190 (33·3) |
| Day 1 | 135 (26·4) | 63 (27·3) | 72 (25·6) | 151 (30·9) | 49 (24·6) | 102 (35·3) | 286 (28·6) | 112 (26·0) | 174 (30·5) |
| Day 2 | 68 (13·3) | 27 (11·7) | 41 (14·6) | 57 (11·7) | 26 (13·1) | 31 (10·7) | 125 (12·5) | 53 (12·3) | 72 (12·6) |
| Day 3 | 39 (7·6) | 16 (6·9) | 23 (8·2) | 38 (7·8) | 20 (10·1) | 18 (6·2) | 77 (7·7) | 36 (8·4) | 41 (7·2) |
| Day 4 | 33 (6·4) | 15 (6·5) | 18 (6·4) | 28 (5·7) | 12 (6·0) | 16 (5·5) | 61 (6·1) | 27 (6·3) | 34 (6·0) |
| Day 5 | 17 (3·3) | 10 (4·3) | 7 (2·5) | 19 (3·9) | 12 (6·0) | 7 (2·4) | 36 (3·6) | 22 (5·1) | 14 (2·5) |
| Day 6 | 9 (1·8) | 6 (2·6) | 3 (1·1) | 6 (1·2) | 3 (1·5) | 3 (1·0) | 15 (1·5) | 9 (2·1) | 6 (1·1) |
| Day 7 | 12 (2·3) | 5 (2·2) | 7 (2·5) | 7 (1·4) | 4 (2·0) | 3 (1·0) | 19 (1·9) | 9 (2·1) | 10 (1·8) |
| Days 8–14 | 29 (5·7) | 15 (6·5) | 14 (5·0) | 16 (3·3) | 11 (5·5) | 5 (1·7) | 45 (4·5) | 29 (6·7) | 19 (3·3) |
| Day ≥15 | 14 (2·7) | 6 (2·6) | 8 (2·8) | 12 (2·5) | 10 (5·0) | 2 (0·7) | 26 (2·6) | 16 (3·7) | 10 (1·8) |
ALL, acute lymphocytic leukaemia; AML, acute myelogenous leukaemia; GVHD, graft‐versus‐host disease; HSCT, haematopoietic stem cell transplant; MOD, multi‐organ dysfunction; VOD/SOS, veno‐occlusive disease/sinusoidal obstruction syndrome.
a
Includes medications that were not prespecified on the case report form.
b
Transplant type unknown for two patients.