Table 2 Best response and ORR in patient cohorts by investigator and independent radiology assessment

From: Dabrafenib plus trametinib in BRAFV600E-mutated rare cancers: the phase 2 ROAR trial

Cohorts

Investigator assessment

 

Independent radiology assessment

 

Best response

 

Best response

 
 

CR

PR

SD

PD

NE

RR

CR

PR

SD

PD

NE

RR

ATC (n = 36)

3 (8%)

17 (47%)

11 (31%)

4 (11%)

1 (3%)a

20 (56%)

(38.1%, 72.1%)

2 (6%)

17 (47%)

8 (22%)

8 (22%)

1 (3%)a

19 (53%)

(35.5%, 69.6%)

BTC (n = 43)

0

23 (53%)

16 (37%)

3 (7%)

1 (2%)b

23 (53%)

(37.7%, 68.8%)

1 (2%)

19 (44%)

15 (35%)

6 (14%)

2 (5%)c

20 (47%)

(31.2%, 62.3%)

ASI (n = 3)

—

2 (67%)

—

1 (33%)

—

67%

(9.4%, 99.2%)

—

2 (67%)

—

1 (33%)

—

67%

(9.4%, 99.2%)

LGG (n = 13)

1 (8%)

6 (46%)

3 (23%)

1 (8%)

0

7 (54%)

(25.1%, 80.8%)

1 (8%)

6 (46%)

2 (15%)

0

3 (23%)d

7 (54%)

(25.1%, 80.8%)

HGG (n = 45)

3 (7%)

12 (27%)

10 (22%)

20 (44%)

0

15 (33%)

(20.0%, 49.0%)

3 (7%)

11 (24%)

5 (11%)

21 (47%)

5 (11%)e

14 (31%)

(18.2%, 46.6%)

HCL (n = 55)

10 (18%)

26 (47%)f

13 (24%)

0

1 (2)

1 (2)g

49 (89%)

(77.8%, 95.9%)

NA

NA

NA

NA

NA

NA

MM (n = 10)

0h

2 (20%)i

1 (10%)

4 (40%)

0

5 (50%)

(18.7%, 81.3%)

NA

NA

NA

NA

NA

NA

  1. In the LGG cohort, minor response was reported in two (15%) patients by investigator review and in one (8%) patient by independent radiology review. Minor response is not included in the calculated response rate. In the HCL cohort, minor response was reported in four (7%) patients. In the MM cohort, no patient showed minimal response.
  2. ORRs were evaluated by RECIST 1.1 in the ATC and BTC cohorts, RANO criteria in the LGG and HGG cohorts and IMWG uniform response criteria for the MM cohort. The best response and RR criteria for HCL were adapted from NCCN guidelines, consensus resolution criteria and definitions in previous studies.
  3. RR is CR + PR in the ATC, BTC, LGG and HGG cohorts. RR in the HCL cohort was calculated as (CR−MRD) + (CR + MRD) + PR. RR in the MM cohort was calculated as sCR + CR + VGPR + PR. The CIs are exact two-sided 95% CI based on the Clopper–Pearson method.
  4. aNo post-baseline assessments.
  5. bReceived anti-cancer therapy before disease progression observed (at first post-baseline assessment).
  6. cNo measurable disease at baseline.
  7. dOne patient had no measurable disease at baseline, and another one had no post-baseline assessments.
  8. eOne patient had no measurable disease at baseline, and three patients had no post-baseline assessments. All progressed by investigator assessment before first radiological assessment; one patient had an SD assessment, which was before the minimum 6 weeks after first dose of study treatment.
  9. fValues show CR without and with MRD.
  10. gOne patient had no post-baseline assessments.
  11. hNo patient showed sCR in the MM cohort.
  12. iVGPR was seen in three (30%) patients in the MM cohort.
  13. MRD, minimal residual disease; NA, not available; NE, not evaluable; PD, progressive disease; RR, response rate; sCR, stringent complete response; SD, stable disease; VGPR, very good partial response.
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