Table 3.

Prediction of patient treatment response by molecular, high‐throughput drug screening (HTS) or patient‐derived xenograft (PDX).

Patient ID	Diagnosis	Treatment received	Molecular target	Tier	Clinical outcome	Molecular predicted patient drug response	HTS predicted patient drug response a	PDX predicted patient drug response a
Molecular‐guided treatment
RA‐049	ALCL	Ceritinib	NPM1 ‐ ALK	1M	CR	Yes	−	Yes
RA‐057	IFS	Larotrectinib	SPECC1L ‐ NTRK3	1M	PR	Yes	−	−
RA‐031	HGG	Larotrectinib	ETV6 ‐ NTRK3	2M	PR	Yes	−	−
RA‐024	HGG	Nivolumab	Hypermutation	1M	SD	Yes	−	−
RA‐048	DMG	Sirolimus	PIK3CA mut	4M	PD	No	Yes	−
RA‐002	HGG	Sirolimus	TSC1 mut/LOH	2M	PD	No	No	No
RA‐039	NBL	Ceritinib	ALK amplification	3M	PD	No	−	No
WE‐012	EWS	Olaparib + irinotecan	STAG2 and TP53 mut	3M	PD	No	−	−
RA‐030	HGG	Nivolumab	Hypermutation	1M	PD	No	−	−
RA‐007	DMG	Sirolimus	mTOR mutation	4M	PD	No	−	−
RA‐033	DMG	Dasatinib	PDGFRA amp	4M	PD	No	−	−
RA‐037	DMG	Sirolimus	PIK3CA mut	4M	PD	No	−	−
RA‐044	DMG	Sirolimus + dasatinib	PTEN mut/LOH PDGFRA/KIT amp	4M	PD	No	−	−
WE‐011	DMG	Sirolimus	PIK3CA mut	4M	PD	No	−	−
Other treatment
RA‐002	HGG	TMZ	NA	NA	PD	NA	Yes	Yes
RA‐027	NBL	Cyclo/Topo IRN/TMZ	NA	NA	PD PD	NA	Yes Yes	No No
RA‐039	NBL	Cyclo/Topo	NA	NA	Mixed	NA	–	Yes
RA‐045	T‐ALL	Carfilzomib + VXL	NA	NA	CR	NA	–	Yes

ALCL, anaplastic large cell lymphoma; amp, amplification; CR, complete response; Cyclo/Topo, cyclophosphamide/topotecan; DMG, diffuse midline glioma H3 K27M mutant; EWS, Ewing’s sarcoma; HGG, high grade glioma; IFS, infantile fibrosarcoma; IRN, irinotecan; LOH, loss of heterozygosity; MCR, maintained complete response; mut, mutation; NA, not applicable; NBL, neuroblastoma; PD, progressive disease; PR, partial response; SD, stable disease; T‐ALL, T‐cell acute lymphoblastic lymphoma; TMZ, temozolomide; VXL, vincristine/ dexamethasone/L‐asparaginase.

^a “–” denotes high‐throughput drug screening (HTS) or PDX not performed.