Patient characteristics and treatment
| . | All, N (%) or mean (SD) . | Responders, n (%) or mean (SD) . | Non-responders∗, n (%) or mean (SD) . | P value† . |
|---|---|---|---|---|
| Total | 61 (100%) | 32 (52%) | 29 (48%) | |
| Female | 28 (46%) | 18 (56%) | 10 (34%) | .124 |
| Age at diagnosis, y | 5.7 (4.9) | 4.6 (4.6) | 6.8 (4.9) | .055 |
| Age at study, y | 24.0 (8.1) | 23.3 (7.9) | 24.7 (8.3) | .563 |
| Time since diagnosis, y | 18.2 (7.0) | 18.7 (6.9) | 17.9 (7.2) | .697 |
| Maximum extent SS | 44 (72%) | 18 (56%) | 26 (90%) | .004 |
| MFB | 5 | NA | ||
| Unifocal bone | 9 | NA | ||
| Skin | 2 | NA | ||
| Lymph node | 2 | NA | ||
| Maximum extent MS | 17 (28%) | 14 (44%) | 3 (10%) | .004 |
| RO+ | 5 (8%) | 4 (12%) | 1 (3%) | .357 |
| CNS+ | 7 (11%) | 5 (16%) | 2 (7%) | .429 |
| CNS-risk lesions | 16 (26%) | 13 (40%) | 3 (10%) | .009 |
| Systemic treatment‡ | 22 (36%) | 17 (53%) | 5 (17%) | .007 |
| VBL + pred | 16 | NA | ||
| MTX IV + pred | 1 | NA | ||
| + salvage | 3 | NA | ||
| + oral maintenance | 14 | NA | ||
| Local treatment | 5 (16%) | NA | ||
| Intralesional steroid injection | 3 | NA | ||
| Curettage | 2 | NA | ||
| Radiotherapy | 1 | NA | ||
| Wait and watch | 10 (31%) | NA |
| . | All, N (%) or mean (SD) . | Responders, n (%) or mean (SD) . | Non-responders∗, n (%) or mean (SD) . | P value† . |
|---|---|---|---|---|
| Total | 61 (100%) | 32 (52%) | 29 (48%) | |
| Female | 28 (46%) | 18 (56%) | 10 (34%) | .124 |
| Age at diagnosis, y | 5.7 (4.9) | 4.6 (4.6) | 6.8 (4.9) | .055 |
| Age at study, y | 24.0 (8.1) | 23.3 (7.9) | 24.7 (8.3) | .563 |
| Time since diagnosis, y | 18.2 (7.0) | 18.7 (6.9) | 17.9 (7.2) | .697 |
| Maximum extent SS | 44 (72%) | 18 (56%) | 26 (90%) | .004 |
| MFB | 5 | NA | ||
| Unifocal bone | 9 | NA | ||
| Skin | 2 | NA | ||
| Lymph node | 2 | NA | ||
| Maximum extent MS | 17 (28%) | 14 (44%) | 3 (10%) | .004 |
| RO+ | 5 (8%) | 4 (12%) | 1 (3%) | .357 |
| CNS+ | 7 (11%) | 5 (16%) | 2 (7%) | .429 |
| CNS-risk lesions | 16 (26%) | 13 (40%) | 3 (10%) | .009 |
| Systemic treatment‡ | 22 (36%) | 17 (53%) | 5 (17%) | .007 |
| VBL + pred | 16 | NA | ||
| MTX IV + pred | 1 | NA | ||
| + salvage | 3 | NA | ||
| + oral maintenance | 14 | NA | ||
| Local treatment | 5 (16%) | NA | ||
| Intralesional steroid injection | 3 | NA | ||
| Curettage | 2 | NA | ||
| Radiotherapy | 1 | NA | ||
| Wait and watch | 10 (31%) | NA |
CNS+, CNS involvement; MFB, multifocal bone; MS, multisystem; MTX, methotrexate; NA, not available; pred, prednisolone; RO+, risk organ involvement; SS, single system; VBL, vinblastine; y, years.
The ethical approval allowed for only limited data on nonresponders.
For comparison of responders and nonresponders, Fisher exact test was used for categorical variables and Mann-Whitney U test for numerical variables. P value in bold indicates statistically significant (P <.05).
For details, see supplemental Table 6.