Characteristics of the cohort
| . | Entire cohort . | Class I variant . | Class II variant . | Variant not classified . | P∗ value . |
|---|---|---|---|---|---|
| Number of patients | 577 (100%) | 209 (100%) | 316 (100%) | 52 (100%) | |
| Region | |||||
| Europe | 278 (48.2%) | 106 (50.7%) | 139 (44.0%) | 33 (63.5%) | .1545 |
| United States | 113 (19.6%) | 67 (32.1%) | 42 (13.3%) | 4 (7.7%) | |
| Rest of world | 186 (32.2%) | 36 (17.2%) | 135 (42.7%) | 15 (28.8%) | |
| Russia | 54 (9.4%) | 12 (5.7%) | 41 (13.0%) | 1 (1.9%) | |
| China | 103 (17.9%) | 22 (10.5%) | 79 (25.0%) | 2 (3.8%) | |
| Brazil | 15 (2.6%) | 0 (0.0%) | 6 (1.9) | 9 (17.3%) | |
| Other | 14 (2.4%) | 2 (1.0%) | 9 (2.8) | 3 (5.8%) | |
| Age at diagnosis, y | |||||
| ≤2 | 309 (57.0%) | 76 (36.4%) | 199 (63.0%) | 34 (65.4%) | <.0001 |
| >2-5 | 118 (20.5%) | 46 (22.0%) | 59 (18.7%) | 13 (25.0%) | |
| >5-11 | 57 (9.9%) | 23 (11.0%) | 31 (9.8%) | 3 (5.8%) | |
| >11-18 | 35 (6.1%) | 24 (11.5%) | 10 (3.2%) | 1 (1.9%) | |
| >18 | 20 (3.5%) | 14 (6.7%) | 6 (1.9%) | 0 (0.0%) | |
| Not reported | 38 (6.6%) | 26 (12.4%) | 11 (3.5%) | 1 (1.9%) | |
| Median (range) | 1.5 (0.0-68.0) | 2.4 (0.0-68.0) | 1.3 (0.0-33.0) | 1.2 (0.0-11.3) | |
| Age at last follow-up, y | |||||
| ≤2 | 41 (7.1%) | 12 (5.7%) | 27 (8.5%) | 2 (3.8%) | .1343 |
| >2-5 | 89 (15.4%) | 27 (12.9%) | 55 (17.4%) | 7 (13.5%) | |
| >5-11 | 121 (21.0%) | 42 (20.1%) | 69 (21.8%) | 10 (19.2%) | |
| >11-18 | 86 (14.9%) | 40 (19.1%) | 37 (11.7%) | 9 (17.3%) | |
| >18 | 82 (14.2%) | 44 (21.1%) | 28 (8.9%) | 10 (19.2%) | |
| Not reported/dead (n, %) | 158 (27.4%) | 44 (21.1%) | 100 (31.6%) | 14 (26.9%) | |
| Median (range) | 8.9 (0.3-71.1) | 11.0 (0.3-68.0) | 6.7 (0.5-71.1) | 11.0 (1.5-42.0) | |
| Survival status at last follow-up | |||||
| Alive | 464 (80.4%) | 190 (90.9%) | 235 (74.4%) | 39 (75.0%) | <.0001 |
| Deceased | 113 (19.6%) | 19 (9.1%) | 81 (25.6%) | 13 (25.0%) | |
| WAS score at last follow-up or before first procedure | |||||
| 1 | 54 (9.4%) | 47 (22.5%) | 7 (2.2%) | 0 (0.0%) | <.0001 |
| 2 | 144 (25.0%) | 84 (40.2%) | 53 (16.8%) | 7 (13.5%) | |
| 3 | 161 (27.9%) | 40 (19.1%) | 98 (31.0%) | 23 (44.2%) | |
| 4 | 109 (18.9%) | 10 (4.8%) | 86 (27.2%) | 13 (25.0%) | |
| 5 | 86 (14.9%) | 19 (9.1%) | 60 (19.0%) | 7 (13.5%) | |
| Not reported | 23 (4.0%) | 9 (4.3%) | 12 (3.8%) | 2 (3.8%) | |
| Procedures | |||||
| Splenectomy | 79 (13.7%) | 31 (14.8%) | 40 (12.7%) | 8 (15.4%) | .5456 |
| HSCT | 255 (44.2%) | 44 (21.1%) | 177 (56%) | 34 (65.4%) | |
| GT | 14 (2.4%) | 4 (1.9%) | 10 (3.2%) | 0 (0%) | |
| >1 procedure | 30 (5.2%) | 4 (1.9%) | 21 (6.6%) | 5 (9.6%) | |
| No procedure | 263 (45.6%) | 134 (64.1%) | 114 (36.1%) | 15 (28.8%) | |
| Age at first procedure, y | |||||
| ≤2 | 115 (19.9%) | 13 (6.2%) | 88 (27.8%) | 14 (26.9%) | .0001 |
| >2-5 | 103 (17.9%) | 28 (13.4%) | 63 (19.9%) | 12 (23.1%) | |
| >5-11 | 40 (6.9%) | 15 (7.2%) | 17 (5.4%) | 8 (15.4%) | |
| >11-18 | 20 (3.5%) | 6 (2.9%) | 12 (3.8%) | 2 (3.8%) | |
| >18 | 9 (1.6%) | 4 (1.9%) | 5 (1.6%) | 0 (0.0%) | |
| No procedure or not reported, n (%) | 290 (50.3%) | 143 (68.4%) | 131 (41.5%) | 16 (30.8%) | |
| Median (range) | 2.3 (0.4-33.7) | 3.5 (0.5-33.7) | 2.1 (0.4-29.0) | 2.2 (0.4-13.8) |
| . | Entire cohort . | Class I variant . | Class II variant . | Variant not classified . | P∗ value . |
|---|---|---|---|---|---|
| Number of patients | 577 (100%) | 209 (100%) | 316 (100%) | 52 (100%) | |
| Region | |||||
| Europe | 278 (48.2%) | 106 (50.7%) | 139 (44.0%) | 33 (63.5%) | .1545 |
| United States | 113 (19.6%) | 67 (32.1%) | 42 (13.3%) | 4 (7.7%) | |
| Rest of world | 186 (32.2%) | 36 (17.2%) | 135 (42.7%) | 15 (28.8%) | |
| Russia | 54 (9.4%) | 12 (5.7%) | 41 (13.0%) | 1 (1.9%) | |
| China | 103 (17.9%) | 22 (10.5%) | 79 (25.0%) | 2 (3.8%) | |
| Brazil | 15 (2.6%) | 0 (0.0%) | 6 (1.9) | 9 (17.3%) | |
| Other | 14 (2.4%) | 2 (1.0%) | 9 (2.8) | 3 (5.8%) | |
| Age at diagnosis, y | |||||
| ≤2 | 309 (57.0%) | 76 (36.4%) | 199 (63.0%) | 34 (65.4%) | <.0001 |
| >2-5 | 118 (20.5%) | 46 (22.0%) | 59 (18.7%) | 13 (25.0%) | |
| >5-11 | 57 (9.9%) | 23 (11.0%) | 31 (9.8%) | 3 (5.8%) | |
| >11-18 | 35 (6.1%) | 24 (11.5%) | 10 (3.2%) | 1 (1.9%) | |
| >18 | 20 (3.5%) | 14 (6.7%) | 6 (1.9%) | 0 (0.0%) | |
| Not reported | 38 (6.6%) | 26 (12.4%) | 11 (3.5%) | 1 (1.9%) | |
| Median (range) | 1.5 (0.0-68.0) | 2.4 (0.0-68.0) | 1.3 (0.0-33.0) | 1.2 (0.0-11.3) | |
| Age at last follow-up, y | |||||
| ≤2 | 41 (7.1%) | 12 (5.7%) | 27 (8.5%) | 2 (3.8%) | .1343 |
| >2-5 | 89 (15.4%) | 27 (12.9%) | 55 (17.4%) | 7 (13.5%) | |
| >5-11 | 121 (21.0%) | 42 (20.1%) | 69 (21.8%) | 10 (19.2%) | |
| >11-18 | 86 (14.9%) | 40 (19.1%) | 37 (11.7%) | 9 (17.3%) | |
| >18 | 82 (14.2%) | 44 (21.1%) | 28 (8.9%) | 10 (19.2%) | |
| Not reported/dead (n, %) | 158 (27.4%) | 44 (21.1%) | 100 (31.6%) | 14 (26.9%) | |
| Median (range) | 8.9 (0.3-71.1) | 11.0 (0.3-68.0) | 6.7 (0.5-71.1) | 11.0 (1.5-42.0) | |
| Survival status at last follow-up | |||||
| Alive | 464 (80.4%) | 190 (90.9%) | 235 (74.4%) | 39 (75.0%) | <.0001 |
| Deceased | 113 (19.6%) | 19 (9.1%) | 81 (25.6%) | 13 (25.0%) | |
| WAS score at last follow-up or before first procedure | |||||
| 1 | 54 (9.4%) | 47 (22.5%) | 7 (2.2%) | 0 (0.0%) | <.0001 |
| 2 | 144 (25.0%) | 84 (40.2%) | 53 (16.8%) | 7 (13.5%) | |
| 3 | 161 (27.9%) | 40 (19.1%) | 98 (31.0%) | 23 (44.2%) | |
| 4 | 109 (18.9%) | 10 (4.8%) | 86 (27.2%) | 13 (25.0%) | |
| 5 | 86 (14.9%) | 19 (9.1%) | 60 (19.0%) | 7 (13.5%) | |
| Not reported | 23 (4.0%) | 9 (4.3%) | 12 (3.8%) | 2 (3.8%) | |
| Procedures | |||||
| Splenectomy | 79 (13.7%) | 31 (14.8%) | 40 (12.7%) | 8 (15.4%) | .5456 |
| HSCT | 255 (44.2%) | 44 (21.1%) | 177 (56%) | 34 (65.4%) | |
| GT | 14 (2.4%) | 4 (1.9%) | 10 (3.2%) | 0 (0%) | |
| >1 procedure | 30 (5.2%) | 4 (1.9%) | 21 (6.6%) | 5 (9.6%) | |
| No procedure | 263 (45.6%) | 134 (64.1%) | 114 (36.1%) | 15 (28.8%) | |
| Age at first procedure, y | |||||
| ≤2 | 115 (19.9%) | 13 (6.2%) | 88 (27.8%) | 14 (26.9%) | .0001 |
| >2-5 | 103 (17.9%) | 28 (13.4%) | 63 (19.9%) | 12 (23.1%) | |
| >5-11 | 40 (6.9%) | 15 (7.2%) | 17 (5.4%) | 8 (15.4%) | |
| >11-18 | 20 (3.5%) | 6 (2.9%) | 12 (3.8%) | 2 (3.8%) | |
| >18 | 9 (1.6%) | 4 (1.9%) | 5 (1.6%) | 0 (0.0%) | |
| No procedure or not reported, n (%) | 290 (50.3%) | 143 (68.4%) | 131 (41.5%) | 16 (30.8%) | |
| Median (range) | 2.3 (0.4-33.7) | 3.5 (0.5-33.7) | 2.1 (0.4-29.0) | 2.2 (0.4-13.8) |
χ2 test for the null hypothesis that the proportions of subjects in category x are equal in class I variant and class II variant subjects. x is the respective category in the same row. Subjects with missing values are not included in the analysis. All P values are unadjusted.