Clinical characteristics of treated patients
| . | Vemurafenib* (n = 50) or dabrafenib (n = 1) . | Cobimetinib (n = 15) . |
|---|---|---|
| Sex | 15 females and 36 males | 3 females and 12 males |
| Age at diagnosis, median (range), y | 57 (17-72) | 56 (34-71) |
| BRAFV600E | 49 (96) | 10† (67) |
| BRAF WT | 2† (4) | 5 (33) |
| Mixed histiocytosis (ECD + LCH) | 15 (29) | 5 (33) |
| CNS | 26 (51) | 9 (60) |
| Cerebellar | 15 (29) | 7 (47) |
| Lung | 18 (35) | 6 (40) |
| Vascular | 39 (76) | 12 (80) |
| Heart | 38 (75) | 10 (67) |
| Xanthelasma | 19 (37) | 3 (20) |
| Diabetes insipidus | 23 (45) | 5 (33) |
| Retroperitoneal fibrosis | 33 (65) | 11 (73) |
| Bones | 44 (86) | 13 (87) |
| Previous treatments | ||
| Anakinra | 6 (12) | 2 (13) |
| Interferon-α | 36 (71) | 11 (73) |
| Deaths | 5 (10) | 0 |
| Targeted treatments‡ | ||
| Vemurafenib/dabrafenib, n | 51 | 12 |
| Cobimetinib, n | 12 | 15 |
| . | Vemurafenib* (n = 50) or dabrafenib (n = 1) . | Cobimetinib (n = 15) . |
|---|---|---|
| Sex | 15 females and 36 males | 3 females and 12 males |
| Age at diagnosis, median (range), y | 57 (17-72) | 56 (34-71) |
| BRAFV600E | 49 (96) | 10† (67) |
| BRAF WT | 2† (4) | 5 (33) |
| Mixed histiocytosis (ECD + LCH) | 15 (29) | 5 (33) |
| CNS | 26 (51) | 9 (60) |
| Cerebellar | 15 (29) | 7 (47) |
| Lung | 18 (35) | 6 (40) |
| Vascular | 39 (76) | 12 (80) |
| Heart | 38 (75) | 10 (67) |
| Xanthelasma | 19 (37) | 3 (20) |
| Diabetes insipidus | 23 (45) | 5 (33) |
| Retroperitoneal fibrosis | 33 (65) | 11 (73) |
| Bones | 44 (86) | 13 (87) |
| Previous treatments | ||
| Anakinra | 6 (12) | 2 (13) |
| Interferon-α | 36 (71) | 11 (73) |
| Deaths | 5 (10) | 0 |
| Targeted treatments‡ | ||
| Vemurafenib/dabrafenib, n | 51 | 12 |
| Cobimetinib, n | 12 | 15 |
Values are n (%) unless otherwise indicated.
LCH, Langerhans cell histiocytosis.
*Three patients received dabrafenib after vemurafenib due to side effects.
†Six patients received a BRAF inhibitor alone and then combination therapy, 3 patients received a BRAF inhibitor alone and then cobimetinib alone, 3 patients received combination therapy alone, and 3 patients received only cobimetinib.
‡Two patients had a MAP2K1 mutation.