Table 1 Patient and baseline characteristics (n = 137)

From: The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Characteristic

Arm 1 n = 77

Arm 2 n = 60

Total

n = 137

Age, years

 Median (range)

8 (2–21)

10 (1–24)

9 (1–24)

Gender, n (%)

 Male

40 (52)

33 (55)

73 (53)

 Female

37 (48)

27 (45)

64 (47)

Race, n (%)a

 White

41 (53)

38 (63)

79 (58)

 Asian

5 (6)

5 (8)

10 (7)

 Black

2 (3)

1 (2)

3 (2)

 Multiple

3 (4)

0

3 (2)

 Other

6 (8)

2 (3)

8 (6)

 Not specified

20 (26)

14 (23)

34 (25)

 Missing

0

0

0

Ethnicity, n (%)

 Hispanic or Latino

3 (4)

1 (2)

4 (3)

 Not Hispanic or Latino

51 (66)

47 (78)

98 (72)

 Not stated

21 (27)

12 (20)

33 (24)

 Missing

2 (3)

0

2 (1)

Primary tumor location, n (%)

 Optic pathway

39 (51)

29 (48)

68 (50)

 Deep midline structures

9 (12)

11 (18)

20 (15)

 Cerebral hemisphere

6 (8)

5 (8)

11 (8)

 Brain stem

6 (8)

2 (3)

8 (6)

 Cerebellum

5 (6)

2 (3)

7 (5)

 Otherb

12 (16)

11 (18)

23 (17)

Histology, n (%)

 Astrocytic

72 (94)

55 (92)

127 (93)

 Oligodendroglial

0

1 (2)

1 (1)

 Mixed glial-neuronal

4 (5)

4 (7)

8 (6)

 Other

1 (1)

0

1 (1)

BRAF alteration status, n (%)

 BRAF fusion

64 (83)

51 (85)

115 (84)

 KIAA1549::BRAF fusion

56 (73)

45 (75)

101 (74)

 Other

8c (10)

6 (10)

14 (10)

 BRAF V600E mutation

13 (17)

9 (15)

22 (16)

Baseline Lansky performance score, n/n (%)d

 50–70

3/71 (4)

9/51 (18)

12/122 (10)

 80–100

68/71 (96)

42/51 (82)

110/122 (90)

Baseline Karnofsky performance score, n/n (%)d

 50–70

2/6 (33)

0

2/15 (13)

 80–100

4/6 (67)

9/9 (100)

13/15 (87)

Prior lines of systemic therapy

 Median (range)

3 (1–9)

3 (1–10)

3 (1–10)

Number of prior lines, n (%)

 1

17 (22)

14 (23)

31 (23)

 2

21 (27)

13 (22)

34 (25)

 ≥3

39 (51)

33 (55)

72 (53)

Prior MAPK pathway targeted therapy, n (%)

 Prior MEK inhibitor

43 (56)

34 (57)

77 (56)

 Prior BRAF inhibitor

8 (10)

7 (12)

15 (11)

 Prior MEK and BRAF inhibitors

5 (7)

4 (7)

9 (7)

 Prior MEK and/or BRAF inhibitore

46 (60)

37 (62)

83 (61) 

Any prior surgery for treatment of primary disease, n (%)

 Pre-operative staging

  Localized disease

60 (78)

42 (70)

102 (74)

  Disseminated/metastatic disease

9 (12)

11 (18)

20 (15)

  Leptomeningeal spread

8 (10)

7 (12)

15 (11)

 Post-operative staging

  Gross total resection

1 (1)

0

1 (1)

  Subtotal resection

36 (47)

27 (45)

63 (46)

  Biopsy only, resection not attempted

40 (52)

33 (55)

73 (53)

Prior radiotherapy for primary disease, n (%)

6 (8)

4 (7)

10 (7)

  1. aThere were no Native Hawaiian or other Pacific Islander, American Indian or Alaska Native participants.
  2. bIncludes tumors that were extending into multiple regions of the brain, leptomeningeal disease and/or spinal disease.
  3. cIncludes six patients with BRAF duplication and two with BRAF rearrangement per fluorescence in situ hybridization or in situ hybridization.
  4. dDenominators for Lansky performance score and Karnofsky performance score summaries are the number of patients whose ages are <16 years and ≥16 years, respectively. Baseline is defined as the last available assessments before start of tovorafenib on cycle 1, day 1.
  5. eFive patients in arm 1 and four patients in arm 2 had previously received both a MEK inhibitor and also a BRAF inhibitor. These patients are recorded in both the ‘Prior MEK inhibitor’ and ‘Prior BRAF inhibitor’ groups.
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