Table 3.
BRAF V600–mutant LCH efficacy summary based on investigator assessment
| Category | CDRB436A2102 (dabrafenib monotherapy) (n = 13) |
CTMT212X2101 (dabrafenib + trametinib) (n = 12) |
|---|---|---|
| Best overall response, n (%)∗ | ||
| CR | 6 (46.2) | 4 (33.3) |
| Regressive disease | 4 (30.8) | 3 (25.0) |
| SD | 3 (23.1) | 3 (25.0) |
| Progressive disease | 0 | 0 |
| Missing | 0 | 2 (16.7)† |
| ORR (95% CI), % | 76.9 (46.2-95.0) | 58.3 (27.7-84.8) |
| Median DOR (95% CI), mo | NR (11.1 to NR) | NR (NR to NR) |
| 12-month rate (95% CI), % | 90 (40-100) | 100 (NR to NR) |
| 24-month rate (95% CI), % | 90 (40-100) | 100 (NR to NR) |
∗
Responses were assessed per the Histiocyte Society criteria. Categories of nonactive disease include CR, which indicates resolution of all disease signs or symptoms (no evidence of disease), and regressive disease, which indicates regressions of disease signs and symptoms with no new lesions. Categories of persistent active disease include SD, which indicates the persistence of signs and symptoms but no new lesions, and progressive disease, which indicates the progression of signs or symptoms and/or the appearance of new lesions.
†
Two patients did not have any postbaseline assessments because of early discontinuation and were considered to be nonresponders.