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Investigator-assessed Best Confirmed Response

a

by Tumor Type

Tumor type

High-grade

glioma

n = 8

Low-grade glioma

n = 15

LCH

n = 2

Other solid

tumor

n = 2

Total

N =

27

Best response, n

Complete response

(CR)

3

(2AA, 1 APXA)

2

5

Partial response (PR)

2

(GBM, AGG)

5

(2 PA, PMA, GG, LGG)

7

Stable disease (SD)

7

(3 PA, GG, PXA,

2 LGG)

1

(PTC)

5

Progressive disease

3

(GBM, AG, AA)

1

(PA)

1

(NB)

5

Not evaluable

2

(GG

b,

GG

c

)

5

Response rate, n (%)

CR+PR

5

(63)

5

(33)

2

(100)

0

12

(44)

a

Extracranial solid tumors assessed by RECIST (Response Evaluation Criteria In Solid Tumors); brain tumors assessed by

RANO (Response Assessment in Neuro-Oncology);

b

missing data in eCRF: PI reports SD weeks 8-16;

c

missing data in

eCRF: PI reports PR weeks 16-24.

AA

, anaplastic astrocytoma;

AG

, anaplastic glioma;

AGG

, anaplastic ganglioglioma;

APXA

, anaplastic pleomorphic xanthoastrocytoma ;

GBM

,

gliobastoma multiforme;

GG

, ganglioglioma;

LGG

, low grade glioma;

NB

, neuroblastoma;

PA

, pilocytic astorcytoma;

PMA

, pilomyxoid

astrocytoma;

PTC

, papillary thyroid carcinoma;

PXA

, pleomorphic xanthoastrocytoma.

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