Table 1.

Patient demographics, baseline characteristics, prior treatments, disposition, and dabrafenib exposurea

Part 1Part 2
CharacteristicDabrafenib 3.75 mg/kg (n = 3)Dabrafenib 4.5 mg/kg (n = 6)Dabrafenib 5.25 mg/kg (n = 6)Dabrafenib RP2D (n = 17)All patients treated with dabrafenib at RP2D (n = 24)All patients with LGG (N = 32)
Median age (range), years8 (4–13)8.5 (2–16)7.5 (3–11)11 (2–17)9.5 (2–17)8.5 (2–17)
 <2 years, n000000
 2–<6 years, n1225710
 6–<12 years, n1344812
 12–≤18 years, n1108910
Sex, n (%)
 Male2 (67)5 (83)3 (50)9 (53)13 (54)19 (59)
 Female1 (33)1 (17)3 (50)8 (47)11 (46)13 (41)
Race, n (%)
 White3 (100)5 (83)6 (100)13 (76)19 (79)27 (84)
 Black01 (17)02 (12)3 (13)3 (9)
 Asian0002 (12)2 (8)2 (6)
Performance status, n (%)b
 1002 (67)3 (50)3 (50)9 (53)12 (50)17 (53)
 80–901 (33)1 (17)2 (33)7 (41)10 (42)11 (34)
 <8002 (33)1 (17)1 (6)2 (8)4 (13)
Histology at initial diagnosis, n (%)
 Pilocytic astrocytoma1 (33)3 (50)1 (17)8 (47)10 (42)13 (41)
 Ganglioglioma01 (17)1 (17)5 (29)6 (25)7 (22)
 Pleomorphic xanthoastrocytoma001 (17)2 (12)3 (13)3 (9)
 Pilomyxoid astrocytoma1 (33)001 (6)1 (4)2 (6)
 Otherc1 (33)2 (33)3 (50)1 (6)4 (17)7 (22)
Histologic grade at initial diagnosis, n (%)d
 Grade I2 (67)4 (67)4 (67)12 (71)16 (67)22 (69)
 Grade II1 (33)2 (33)2 (33)4 (24)7 (29)9 (28)
Median time since initial diagnosis (range), months36 (32–39)15 (11–90)39 (18–83)26 (6–190)31 (6–190)32 (6–190)
Prior treatments, n (%)e
 Chemotherapy3 (100)5 (83)6 (100)14 (82)20 (83)28 (88)
 Radiotherapy1 (33)1 (17)1 (17)3 (18)5 (21)6 (19)
 Small-molecule therapy001 (17)1 (6)2 (8)2 (6)
 Immunotherapy0001 (6)1 (4)1 (3)
 Other0003 (18)3 (13)3 (9)
Median time from last recurrence to dabrafenib start (range), monthsfNANA0.8 (0.2–1.3)1.1 (0.1–81.5)1.1 (0.1–81.5)1.1 (0.1–81.5)
Median time from last progression to dabrafenib start (range), monthsg7.6 (0.5–14.7)0.8 (0.5–1.1)1.8 (0.2–26.2)1.6 (0.1–10.3)1.5 (0.1–26.2)1.1 (0.1–26.2)
Continuing treatment, n (%)2 (67)3 (50)2 (33)8 (47)10 (42)15 (47)
Discontinued treatment, n (%)1 (33)3 (50)4 (67)9 (53)14 (58)17 (53)
 Reasons for discontinuation
  Investigator discretion1 (33)1 (17)4 (67)5 (29)10 (42)11 (34)
  Disease progression02 (33)02 (12)2 (8)4 (13)
  Adverse event0002 (12)2 (8)2 (6)
Median duration of exposure to dabrafenib (range), weeks157 (62–159)120 (8–185)96 (25–152)105 (<1–149)104 (<1–152)108 (<1–185)
Patients with dose reductions and/or interruptions, n (%)1 (33)3 (50)1 (17)5 (29)6 (25)10 (31)
  • Abbreviation: NA, not applicable.

  • aAs of data cutoff (September 12, 2017).

  • bUsing Karnofsky (≥16 years of age; n = 28) or Lansky (<16 years of age; n = 4) performance status, as appropriate.

  • cDesmoplastic neuroepithelial neoplasm, cervicomedullary tumor, glioneuronal brain stem tumor, posterior fossa brain tumor, optic pathway glioma, gliomatosis cerebri, and other low-grade glioma.

  • dOne patient had missing data for disease grade at initial diagnosis but was confirmed to have LGG.

  • ePatients may have had multiple therapies and prior therapy type was undetermined in 2 patients; best response to last therapy received included 5 patients with a partial response, 13 patients with stable disease, and 9 patients with progressive disease (response to last therapy was undetermined in 5 patients).

  • fIn 11 patients with recurrence.

  • gIn 25 patients with disease progression.