. 2021 Dec 20;14(4):e14608. doi: 10.15252/emmm.202114608

Table 2.

Correlation between drug sensitivity predicted by molecular testing and patient‐derived xenograft (PDX) responses.

Patient ID	Diagnosis	Molecular target	PDX treatment	PDX response ^a	PDX correlated with molecular
RA‐001	EWS	TP53, STAG2 mut	IRN + TMZ + talazoparib	MCR	Yes
RA‐001	EWS	TP53, STAG2 mut	Talazoparib	PD	No
WE‐012	EWS	TP53, STAG2 mut	IRN + TMZ + talazoparib	CR	Yes
WE‐012	EWS	TP53, STAG2 mut	Talazoparib	PD	No
RA‐039	NBL	ALK amplification	Ceritinib	CR	Yes
RA‐039	NBL	ALK amplification	Cyclo/Topo/ceritinib	MCR	Yes
RA‐049	ALCL	NPM1 ‐ ALK	Ceritinib	CR	Yes
			Alectinib	MCR	Yes
			Brentuximab + ceritinib	MCR	Yes
RA‐002	HGG	TSC1 mut (LOH)	Temsirolimus	R	Yes
RA‐045	T‐ALL	CDKN2A/B loss	Palbociclib	SD	No
RA‐054	RMS	CDK4 amplification High FGFR4 RNA	Palbociclib	PD	No
			Palbociclib + temsirolimus	PD	No
			Ponatinib	PD	No
RA‐029	RMS	High FGFR4 RNA	Ponatinib	PD	No
RA‐017	OST	CCNE1 amplification	Dinaciclib	PD	No
RA‐017	OST	CCNE1 amplification	Dinaciclib + cisplatin	PD	No
RA‐013	NBL	High BCL RNA	Venetoclax	PD	No
RA‐027	NBL	NF1 mutation with LOH	Trametinib	PD	No
RA‐027	NBL	NF1 mutation with LOH	Trametinib + isotretinoin	PD	No
RA‐028	HGG	PDGFRA mutation CDKN2A/B loss	Palbociclib	PD	No
RA‐028	HGG	PDGFRA mutation CDKN2A/B loss	Temsirolimus + palbociclib	PD	No

ALCL, anaplastic large cell lymphoma; CR, complete response; Cyclo/Topo, cyclophosphamide/topotecan; EWS, Ewing’s sarcoma; HGG, high grade glioma; IRN, irinotecan; LOH, loss of heterozygosity; MCR, maintained complete response; NBL, neuroblastoma; OST, osteosarcoma; PD, progressive disease; R, response; RMS, rhabdomyosarcoma; SD, stable disease; T‐ALL, T‐cell acute lymphoblastic leukemia; TMZ, temozolomide.

^{^a}

Objective response in a non‐CNS (central nervous system) tumor is classified by Point‐to‐Point Tunneling Protocol (PPTP) criteria (Houghton et al, 2007). In the CNS tumor, the response is defined as significantly prolonged event‐free survival (EFS) where median EFS of the treatment group is at least twice longer than that of control and with significant difference in EFS between treated and control (P ≤ 0.05).