Table 1 Clinical Characteristics of the 28 patients in this cohort
Characteristics | N | % |
|---|---|---|
Age (years) | ||
Median: 7.3 years (range 2.3–24) | ||
< 3 | 2 | 7 |
3–10 | 16 | 57 |
10 + | 10 | 36 |
Gender | ||
Female | 15 | 54 |
Male | 13 | 46 |
Overall survival (months) | ||
Median: 11.5 months (range 0–82) | ||
< 12 | 14 | 50 |
12–24 | 11 | 39 |
> 24 | 3 | 11 |
Symptom duration (months) | ||
< 6 | 19 | 68 |
6–12 | 2 | 7 |
12–24 | 5 | 18 |
Not available | 2 | 7 |
Radiotherapy | 26 | 93 |
Photon irradiation | 24 | 86 |
Proton irradiation | 2 | 7 |
Re-irradiation | 5 | 18 |
Systemc therapy | 25 | 89 |
Radiosensitizer | 11 | 44 |
Adjuvant therapy | 22 | 79 |
Specific systemic therapy agent(s) received | ||
Bevacizumab | 8 | 30 |
Vorinostat (HDAC inhibitor) | 7 | 26 |
CDK4/6 inhibitor (ribcoclib [n = 6], palbociclib [n = 1]) | 7 | 26 |
Everolimus (mTOR inhibitor) | 4 | 15 |
Irinotecan | 4 | 15 |
Temozolomide | 3 | 11 |
PARP inhibitor | 3 | 11 |
Tyrosine kinase inhibitor (cabozantinib [n = 1], pazopanib [n = 1], dasatinib [n = 1]) | 3 | 11 |
WEE-1 inhibitor | 2 | 7 |
Tivantinib (cMET inhibitor) | 2 | 7 |
ONC201 | 1 | 4 |
Trametinib (MEK inhibitor) | 1 | 4 |
Imetelstat (Telomerase inhibitor) | 1 | 4 |
Distant metastases at diagnosisa | 1 | 4 |
Distant metastases at autopsy | 9 | 32 |
Cerebral cortex | 7 | 25 |
Cerebellar peduncles and/or cerebellum | 4 | 14 |
Thalamus and/or subthalamus | 3 | 11 |
Basal ganglia | 3 | 11 |
Cervical spinal cord | 1 | 4 |
Histone mutation status | ||
H3.3 | 20 | 71 |
H3.1 | 7 | 25 |
WT | 1 | 4 |
Extent of pathological spread (autopsy) | ||
Extension beyond brainstem | 22 | 79 |
Limited to brainstem | 5 | 18 |
Insuffient information | 1 | 4 |
Location of pathologic spread (autopsy) | ||
Cerebellar peduncles and/or cerebellum | 21 | 75 |
Thalamus and/or subthalamus | 15 | 54 |
Cervical cord | 13 | 46 |
Basal ganglia | 8 | 29 |
Cortex | 2 | 7 |