Clinical characteristics according to lesional BRAFV600E status
| Characteristic . | BRAFV600E positive . | BRAFV600E negative . | P value . |
|---|---|---|---|
| Patients, no. | 74 | 82 | |
| Age at diagnosis, median (range), y | 42.9 (19-88) | 39.0 (18-73) | .19 |
| Sex | |||
| Male | 38 (51.4) | 48 (58.5) | .42 |
| Female | 36 (48.6) | 34 (41.5) | |
| Disease extent at diagnosis | |||
| MS | 27 (36.5) | 31 (37.8) | .87 |
| SS | 47 (63.5) | 51 (62.2) | |
| Detailed subtype∗ | |||
| MS, RO+† | 9 (12.2) | 6 (7.3) | .42 |
| MS, RO- | 18 (24.3) | 25 (30.5) | .47 |
| SS, bone | 29 (39.2) | 29 (35.4) | .74 |
| SS, UFB | 24 (32.4) | 24 (29.3) | .73 |
| SS, MFB | 5 (6.8) | 5 (6.1) | 1 |
| SS, skin | 6 (8.1) | 5 (6.1) | .76 |
| SS, lung | 10 (13.5) | 12 (14.6) | 1 |
| SS, other | 2 (2.7) | 5 (6.1) | .45 |
| Disease site(s) at diagnosis | |||
| Bone | 51 (68.9) | 51 (62.2) | .40 |
| Lung | 24 (32.4) | 32 (39.0) | .41 |
| Skin | 14 (18.9) | 11 (13.4) | .39 |
| Central nervous system‡ | 9 (12.2) | 9 (11.0) | 1 |
| Lymph node | 6 (8.1) | 12 (14.6) | .22 |
| RO† | 9 (12.2) | 7 (8.5) | .60 |
| Gastrointestinal tract | 2 (2.7) | 1 (1.2) | .60 |
| First-line therapy | |||
| Chemotherapy§ | 20 (27.0) | 22 (26.8) | 1 |
| Radiotherapy | 8 (10.8) | 7 (8.5) | .79 |
| Targeted therapy|| | 3 (4.1) | 1 (1.2) | .35 |
| None/other therapy | 43 (58.1) | 51 (62.2) | .63 |
| Unknown | 0 (0.0) | 1 (1.2) | 1 |
| Follow-up, median (range), y¶ | 4.4 (0-36) | 4.2 (0-31) | .40 |
| Characteristic . | BRAFV600E positive . | BRAFV600E negative . | P value . |
|---|---|---|---|
| Patients, no. | 74 | 82 | |
| Age at diagnosis, median (range), y | 42.9 (19-88) | 39.0 (18-73) | .19 |
| Sex | |||
| Male | 38 (51.4) | 48 (58.5) | .42 |
| Female | 36 (48.6) | 34 (41.5) | |
| Disease extent at diagnosis | |||
| MS | 27 (36.5) | 31 (37.8) | .87 |
| SS | 47 (63.5) | 51 (62.2) | |
| Detailed subtype∗ | |||
| MS, RO+† | 9 (12.2) | 6 (7.3) | .42 |
| MS, RO- | 18 (24.3) | 25 (30.5) | .47 |
| SS, bone | 29 (39.2) | 29 (35.4) | .74 |
| SS, UFB | 24 (32.4) | 24 (29.3) | .73 |
| SS, MFB | 5 (6.8) | 5 (6.1) | 1 |
| SS, skin | 6 (8.1) | 5 (6.1) | .76 |
| SS, lung | 10 (13.5) | 12 (14.6) | 1 |
| SS, other | 2 (2.7) | 5 (6.1) | .45 |
| Disease site(s) at diagnosis | |||
| Bone | 51 (68.9) | 51 (62.2) | .40 |
| Lung | 24 (32.4) | 32 (39.0) | .41 |
| Skin | 14 (18.9) | 11 (13.4) | .39 |
| Central nervous system‡ | 9 (12.2) | 9 (11.0) | 1 |
| Lymph node | 6 (8.1) | 12 (14.6) | .22 |
| RO† | 9 (12.2) | 7 (8.5) | .60 |
| Gastrointestinal tract | 2 (2.7) | 1 (1.2) | .60 |
| First-line therapy | |||
| Chemotherapy§ | 20 (27.0) | 22 (26.8) | 1 |
| Radiotherapy | 8 (10.8) | 7 (8.5) | .79 |
| Targeted therapy|| | 3 (4.1) | 1 (1.2) | .35 |
| None/other therapy | 43 (58.1) | 51 (62.2) | .63 |
| Unknown | 0 (0.0) | 1 (1.2) | 1 |
| Follow-up, median (range), y¶ | 4.4 (0-36) | 4.2 (0-31) | .40 |
Data are given as number (percentage) of each group, unless otherwise indicated. A graphical summary of these data is provided in supplemental Figure 1.
MFB, multifocal bone; RO, risk organ; UFB, unifocal bone.
Fisher exact tests comparing patients with vs without a disease extent subtype are shown.
The hematopoietic system, liver, and spleen were considered ROs, extrapolating from pediatric data.3,4
Given that the posterior pituitary and pituitary stalk are direct extensions of the hypothalamus, pituitary tumors are classified as central nervous system involvement.
With or without additional local therapy. Among BRAFV600E-positive patients, 1 of 20 also received radiotherapy. Among BRAFV600E-negative patients, 3 of 22 also received radiotherapy.
BRAF and/or MEK inhibitors.
Estimated with the reverse Kaplan-Meier method.