| Variable . | NIVO3 n = 85 No. (%) . | NIVO3 + IPI1 n = 81 No. (%) . |
|---|---|---|
| Age | ||
| Median (range), years | 10.0 (1–21) | 11.0 (1–21) |
| Age, years | ||
| <2 | 1 (1.2) | 2 (2.5) |
| ≥2 and <12 | 46 (54.1) | 41 (50.6) |
| ≥12 and <18 | 30 (35.3) | 31 (38.3) |
| ≥18 | 8 (9.4) | 7 (8.6) |
| Sex | ||
| Male | 52 (61.2) | 44 (54.3) |
| Female | 33 (38.8) | 37 (45.7) |
| Disease diagnosis | ||
| Cohort 1: | ||
| DIPG | 18 (21.2) | 18 (22.2) |
| Diffuse midline gliomaa | 5 (5.9) | 4 (4.9) |
| Cohort 2: | ||
| HGGb | 16 (18.8) | 15 (18.5) |
| Cohort 3: | ||
| Medulloblastoma | 15 (17.6) | 15 (18.5) |
| Cohort 4: | ||
| Ependymoma | 12 (14.1) | 10 (12.3) |
| Cohort 5 (other diagnoses)c: | ||
| Atypical teratoid rhabdoid tumor | 4 (4.7) | 3 (3.7) |
| Pineoblastoma | 4 (4.7) | 0 |
| Choroid plexus carcinoma | 0 | 4 (4.9) |
| HGG | 2 (2.4) | 1 (1.2) |
| Anaplastic pleomorphic xanthoastrocytoma | 1 (1.2) | 1 (1.2) |
| Embryonal tumor with multilayered rosettes | 1 (1.2) | 1 (1.2) |
| Malignant germ cell tumor | 1 (1.2) | 1 (1.2) |
| Diffuse midline glioma | 1 (1.2) | 0 |
| Otherd | 5 (5.9) | 8 (9.9) |
| Disease stage | ||
| Localized | 54 (63.5) | 60 (74.1) |
| Metastatic | 31 (36.5) | 21 (25.9) |
| LPS/KPS | ||
| <80 | 17 (20.0) | 14 (17.3) |
| ≥80 | 68 (80.0) | 67 (82.7) |
| Time from initial diagnosis to first dosee | ||
| <6 months | 1 (1.2) | 2 (2.5) |
| 6 to <12 months | 7 (8.2) | 6 (7.4) |
| 12 to <18 months | 10 (11.8) | 11 (13.6) |
| 18 to <24 months | 5 (5.9) | 6 (7.4) |
| ≥24 months | 38 (44.7) | 34 (42.0) |
| Not reported | 1 (1.2) | 0 |
| Steroid usef | ||
| Yes | 17 (20.0) | 10 (12.3) |
| No | 68 (80.0) | 71 (87.7) |
| PD-L1 expression level | ||
| Quantifiable | 62 (72.9) | 57 (70.4) |
| <1% | 41 (66.1) | 42 (73.7) |
| ≥1% | 21 (33.9) | 15 (26.3) |
| <5% | 46 (74.2) | 46 (80.7) |
| ≥5% | 16 (25.8) | 11 (19.3) |
| Indeterminate/not evaluable | 1 (1.2) | 2 (2.5) |
| Not reportedg | 22 (25.9) | 22 (27.2) |
| DIPG (biopsy not required), n | 20 | 21 |
| Not reported, n | 2 | 1 |
| Prior RT | 80 (94.1) | 76 (83.8) |
| Prior systemic therapye | 56 (65.9) | 56 (69.1) |
| Prior surgery related to cancer | 66 (77.6) | 62 (76.5) |
| Variable . | NIVO3 n = 85 No. (%) . | NIVO3 + IPI1 n = 81 No. (%) . |
|---|---|---|
| Age | ||
| Median (range), years | 10.0 (1–21) | 11.0 (1–21) |
| Age, years | ||
| <2 | 1 (1.2) | 2 (2.5) |
| ≥2 and <12 | 46 (54.1) | 41 (50.6) |
| ≥12 and <18 | 30 (35.3) | 31 (38.3) |
| ≥18 | 8 (9.4) | 7 (8.6) |
| Sex | ||
| Male | 52 (61.2) | 44 (54.3) |
| Female | 33 (38.8) | 37 (45.7) |
| Disease diagnosis | ||
| Cohort 1: | ||
| DIPG | 18 (21.2) | 18 (22.2) |
| Diffuse midline gliomaa | 5 (5.9) | 4 (4.9) |
| Cohort 2: | ||
| HGGb | 16 (18.8) | 15 (18.5) |
| Cohort 3: | ||
| Medulloblastoma | 15 (17.6) | 15 (18.5) |
| Cohort 4: | ||
| Ependymoma | 12 (14.1) | 10 (12.3) |
| Cohort 5 (other diagnoses)c: | ||
| Atypical teratoid rhabdoid tumor | 4 (4.7) | 3 (3.7) |
| Pineoblastoma | 4 (4.7) | 0 |
| Choroid plexus carcinoma | 0 | 4 (4.9) |
| HGG | 2 (2.4) | 1 (1.2) |
| Anaplastic pleomorphic xanthoastrocytoma | 1 (1.2) | 1 (1.2) |
| Embryonal tumor with multilayered rosettes | 1 (1.2) | 1 (1.2) |
| Malignant germ cell tumor | 1 (1.2) | 1 (1.2) |
| Diffuse midline glioma | 1 (1.2) | 0 |
| Otherd | 5 (5.9) | 8 (9.9) |
| Disease stage | ||
| Localized | 54 (63.5) | 60 (74.1) |
| Metastatic | 31 (36.5) | 21 (25.9) |
| LPS/KPS | ||
| <80 | 17 (20.0) | 14 (17.3) |
| ≥80 | 68 (80.0) | 67 (82.7) |
| Time from initial diagnosis to first dosee | ||
| <6 months | 1 (1.2) | 2 (2.5) |
| 6 to <12 months | 7 (8.2) | 6 (7.4) |
| 12 to <18 months | 10 (11.8) | 11 (13.6) |
| 18 to <24 months | 5 (5.9) | 6 (7.4) |
| ≥24 months | 38 (44.7) | 34 (42.0) |
| Not reported | 1 (1.2) | 0 |
| Steroid usef | ||
| Yes | 17 (20.0) | 10 (12.3) |
| No | 68 (80.0) | 71 (87.7) |
| PD-L1 expression level | ||
| Quantifiable | 62 (72.9) | 57 (70.4) |
| <1% | 41 (66.1) | 42 (73.7) |
| ≥1% | 21 (33.9) | 15 (26.3) |
| <5% | 46 (74.2) | 46 (80.7) |
| ≥5% | 16 (25.8) | 11 (19.3) |
| Indeterminate/not evaluable | 1 (1.2) | 2 (2.5) |
| Not reportedg | 22 (25.9) | 22 (27.2) |
| DIPG (biopsy not required), n | 20 | 21 |
| Not reported, n | 2 | 1 |
| Prior RT | 80 (94.1) | 76 (83.8) |
| Prior systemic therapye | 56 (65.9) | 56 (69.1) |
| Prior surgery related to cancer | 66 (77.6) | 62 (76.5) |
Abbreviations: DIPG, diffuse intrinsic pontine glioma, HGG, high-grade glioma; IPI, ipilimumab; KPS, Karnofsky performance status; LPS, Lansky performance status; NIVO, nivolumab; NOS, not otherwise specified; PD-L1, programmed death ligand 1; PNET, primitive neuroectodermal tumor; RT, radiotherapy.
aCohort 1 eligibility included diffuse midline glioma with demonstrated H3K27M mutation.
bCohort 2 included patients with non-brain stem HGG.
cSeveral of these patients had complex diagnoses that could have placed them in more than one cohort; however, after discussion and agreement by the clinical team, the patients were ultimately placed into cohort 5 on a case-by-case basis.
dCohort 5 “other” diagnoses at study entry: NIVO3: isolated spine lesion (n = 1), CNS embryonal tumor, NOS group, WHO grade 4 (n = 1); disseminated oligodendroglial-like leptomeningeal tumor (n = 1); PNET (n = 1); high-grade neuroepithelial tumor (n = 1); NIVO3 + IPI1: PNET (n = 2); CNS embryonal tumor NOS (n = 1); ependymoblastoma (n = 1); cribriform neuroepithelial tumor (n = 1); atypical meningioma WHO grade 2 (n = 1); supratentorial PNET (n = 1); anaplastic astrocytoma grade 3 (n = 1).
eExcluding DIPG and diffuse midline glioma.
fBaseline corticosteroid use is based on corticosteroid use within 5 days prior to first dose date. Patients receiving corticosteroids for tumor-associated intracranial mass effect at the time of screening were required to discontinue or taper use to ≤0.05 mg/kg of dexamethasone daily (or equivalent) at study entry.
gDIPG cohort included in total output. Tissue collection was not required for DIPG cohort.
| Variable . | NIVO3 n = 85 No. (%) . | NIVO3 + IPI1 n = 81 No. (%) . |
|---|---|---|
| Age | ||
| Median (range), years | 10.0 (1–21) | 11.0 (1–21) |
| Age, years | ||
| <2 | 1 (1.2) | 2 (2.5) |
| ≥2 and <12 | 46 (54.1) | 41 (50.6) |
| ≥12 and <18 | 30 (35.3) | 31 (38.3) |
| ≥18 | 8 (9.4) | 7 (8.6) |
| Sex | ||
| Male | 52 (61.2) | 44 (54.3) |
| Female | 33 (38.8) | 37 (45.7) |
| Disease diagnosis | ||
| Cohort 1: | ||
| DIPG | 18 (21.2) | 18 (22.2) |
| Diffuse midline gliomaa | 5 (5.9) | 4 (4.9) |
| Cohort 2: | ||
| HGGb | 16 (18.8) | 15 (18.5) |
| Cohort 3: | ||
| Medulloblastoma | 15 (17.6) | 15 (18.5) |
| Cohort 4: | ||
| Ependymoma | 12 (14.1) | 10 (12.3) |
| Cohort 5 (other diagnoses)c: | ||
| Atypical teratoid rhabdoid tumor | 4 (4.7) | 3 (3.7) |
| Pineoblastoma | 4 (4.7) | 0 |
| Choroid plexus carcinoma | 0 | 4 (4.9) |
| HGG | 2 (2.4) | 1 (1.2) |
| Anaplastic pleomorphic xanthoastrocytoma | 1 (1.2) | 1 (1.2) |
| Embryonal tumor with multilayered rosettes | 1 (1.2) | 1 (1.2) |
| Malignant germ cell tumor | 1 (1.2) | 1 (1.2) |
| Diffuse midline glioma | 1 (1.2) | 0 |
| Otherd | 5 (5.9) | 8 (9.9) |
| Disease stage | ||
| Localized | 54 (63.5) | 60 (74.1) |
| Metastatic | 31 (36.5) | 21 (25.9) |
| LPS/KPS | ||
| <80 | 17 (20.0) | 14 (17.3) |
| ≥80 | 68 (80.0) | 67 (82.7) |
| Time from initial diagnosis to first dosee | ||
| <6 months | 1 (1.2) | 2 (2.5) |
| 6 to <12 months | 7 (8.2) | 6 (7.4) |
| 12 to <18 months | 10 (11.8) | 11 (13.6) |
| 18 to <24 months | 5 (5.9) | 6 (7.4) |
| ≥24 months | 38 (44.7) | 34 (42.0) |
| Not reported | 1 (1.2) | 0 |
| Steroid usef | ||
| Yes | 17 (20.0) | 10 (12.3) |
| No | 68 (80.0) | 71 (87.7) |
| PD-L1 expression level | ||
| Quantifiable | 62 (72.9) | 57 (70.4) |
| <1% | 41 (66.1) | 42 (73.7) |
| ≥1% | 21 (33.9) | 15 (26.3) |
| <5% | 46 (74.2) | 46 (80.7) |
| ≥5% | 16 (25.8) | 11 (19.3) |
| Indeterminate/not evaluable | 1 (1.2) | 2 (2.5) |
| Not reportedg | 22 (25.9) | 22 (27.2) |
| DIPG (biopsy not required), n | 20 | 21 |
| Not reported, n | 2 | 1 |
| Prior RT | 80 (94.1) | 76 (83.8) |
| Prior systemic therapye | 56 (65.9) | 56 (69.1) |
| Prior surgery related to cancer | 66 (77.6) | 62 (76.5) |
| Variable . | NIVO3 n = 85 No. (%) . | NIVO3 + IPI1 n = 81 No. (%) . |
|---|---|---|
| Age | ||
| Median (range), years | 10.0 (1–21) | 11.0 (1–21) |
| Age, years | ||
| <2 | 1 (1.2) | 2 (2.5) |
| ≥2 and <12 | 46 (54.1) | 41 (50.6) |
| ≥12 and <18 | 30 (35.3) | 31 (38.3) |
| ≥18 | 8 (9.4) | 7 (8.6) |
| Sex | ||
| Male | 52 (61.2) | 44 (54.3) |
| Female | 33 (38.8) | 37 (45.7) |
| Disease diagnosis | ||
| Cohort 1: | ||
| DIPG | 18 (21.2) | 18 (22.2) |
| Diffuse midline gliomaa | 5 (5.9) | 4 (4.9) |
| Cohort 2: | ||
| HGGb | 16 (18.8) | 15 (18.5) |
| Cohort 3: | ||
| Medulloblastoma | 15 (17.6) | 15 (18.5) |
| Cohort 4: | ||
| Ependymoma | 12 (14.1) | 10 (12.3) |
| Cohort 5 (other diagnoses)c: | ||
| Atypical teratoid rhabdoid tumor | 4 (4.7) | 3 (3.7) |
| Pineoblastoma | 4 (4.7) | 0 |
| Choroid plexus carcinoma | 0 | 4 (4.9) |
| HGG | 2 (2.4) | 1 (1.2) |
| Anaplastic pleomorphic xanthoastrocytoma | 1 (1.2) | 1 (1.2) |
| Embryonal tumor with multilayered rosettes | 1 (1.2) | 1 (1.2) |
| Malignant germ cell tumor | 1 (1.2) | 1 (1.2) |
| Diffuse midline glioma | 1 (1.2) | 0 |
| Otherd | 5 (5.9) | 8 (9.9) |
| Disease stage | ||
| Localized | 54 (63.5) | 60 (74.1) |
| Metastatic | 31 (36.5) | 21 (25.9) |
| LPS/KPS | ||
| <80 | 17 (20.0) | 14 (17.3) |
| ≥80 | 68 (80.0) | 67 (82.7) |
| Time from initial diagnosis to first dosee | ||
| <6 months | 1 (1.2) | 2 (2.5) |
| 6 to <12 months | 7 (8.2) | 6 (7.4) |
| 12 to <18 months | 10 (11.8) | 11 (13.6) |
| 18 to <24 months | 5 (5.9) | 6 (7.4) |
| ≥24 months | 38 (44.7) | 34 (42.0) |
| Not reported | 1 (1.2) | 0 |
| Steroid usef | ||
| Yes | 17 (20.0) | 10 (12.3) |
| No | 68 (80.0) | 71 (87.7) |
| PD-L1 expression level | ||
| Quantifiable | 62 (72.9) | 57 (70.4) |
| <1% | 41 (66.1) | 42 (73.7) |
| ≥1% | 21 (33.9) | 15 (26.3) |
| <5% | 46 (74.2) | 46 (80.7) |
| ≥5% | 16 (25.8) | 11 (19.3) |
| Indeterminate/not evaluable | 1 (1.2) | 2 (2.5) |
| Not reportedg | 22 (25.9) | 22 (27.2) |
| DIPG (biopsy not required), n | 20 | 21 |
| Not reported, n | 2 | 1 |
| Prior RT | 80 (94.1) | 76 (83.8) |
| Prior systemic therapye | 56 (65.9) | 56 (69.1) |
| Prior surgery related to cancer | 66 (77.6) | 62 (76.5) |
Abbreviations: DIPG, diffuse intrinsic pontine glioma, HGG, high-grade glioma; IPI, ipilimumab; KPS, Karnofsky performance status; LPS, Lansky performance status; NIVO, nivolumab; NOS, not otherwise specified; PD-L1, programmed death ligand 1; PNET, primitive neuroectodermal tumor; RT, radiotherapy.
aCohort 1 eligibility included diffuse midline glioma with demonstrated H3K27M mutation.
bCohort 2 included patients with non-brain stem HGG.
cSeveral of these patients had complex diagnoses that could have placed them in more than one cohort; however, after discussion and agreement by the clinical team, the patients were ultimately placed into cohort 5 on a case-by-case basis.
dCohort 5 “other” diagnoses at study entry: NIVO3: isolated spine lesion (n = 1), CNS embryonal tumor, NOS group, WHO grade 4 (n = 1); disseminated oligodendroglial-like leptomeningeal tumor (n = 1); PNET (n = 1); high-grade neuroepithelial tumor (n = 1); NIVO3 + IPI1: PNET (n = 2); CNS embryonal tumor NOS (n = 1); ependymoblastoma (n = 1); cribriform neuroepithelial tumor (n = 1); atypical meningioma WHO grade 2 (n = 1); supratentorial PNET (n = 1); anaplastic astrocytoma grade 3 (n = 1).
eExcluding DIPG and diffuse midline glioma.
fBaseline corticosteroid use is based on corticosteroid use within 5 days prior to first dose date. Patients receiving corticosteroids for tumor-associated intracranial mass effect at the time of screening were required to discontinue or taper use to ≤0.05 mg/kg of dexamethasone daily (or equivalent) at study entry.
gDIPG cohort included in total output. Tissue collection was not required for DIPG cohort.
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