Table 3.

Studies correlating PDX treatment results with clinical data

Tumor type (reference)	Definition activity	Standard agent	n	RR (%)	Clinical correlates
CRC	TR > 50%	Cetuximab	47	10	N/A
CRC KRAS WT (28)		Cetuximab	38	17
CRC (29)	T/C < 10%	5-Fluorouracil	52	13	N/A
		Oxaliplatin	52	0
		Irinotecan	49	38
		Cetuximab	52	26
HBC (34)	Complete response	AC	17	76	Response to treatment in the PDX model was concordant with clinical data in 5/7 patients.
	TGI > 50% or T/C GD > 2-fold	Docetaxel	17	47
		Trastuzumab	2	50
		GnRH antagonist	1	100
HBC (16)	RR > 30%	Docetaxel	7	14	92% correlation between clinical responses and responses in PDX
		Doxorubicin	4	0
		Trastuzumab–lapatinib	1	100
NSCLC (20)	Statistically significant decrement in tumor area in treated vs. control tumors	Cisplatin–vinorelbine	32	28	PDX models from 6/7 patients with early recurrent disease were resistant to the clinically used regimen.
		Cisplatin–docetaxel	19	42
		Cisplatin–gemcitabine	16	44
NSCLC (21)	T/C < 5%	Etoposide	25	4	N/A
		Carboplatin	25	12
		Gemcitabine	25	12
		Paclitaxel	25	16
		Vinorelbine	11	0
		Cetuximab	25	12
		Erlotinib	25	1
SCCHN (25)	T/C < 20%	Cetuximab	11	9%	N/A
RCC (15)	Statistically significant differences in TGI	Sunitinib	8	Active	N/A
		Sirolimus		Active
		Erlotinib		Inactive
PDAC (26)	T/C < 20%	Gemcitabine	14	36	N/A
		Erlotinib		0
		Temsirolimus		0
PDAC (33)	TGI > 85%	Gemcitabine	23	17%	Response to gemcitabine in the PDX model predicted longer time to progression in patients.

NOTE: This table provides a summary of studies in which PDX models from different cancer types have been treated with agents used in the clinical care of these patients.

Abbreviations: AC, adriamycin–cyclophosphamide; CRC, colorectal cancer; GD, growth delay; GnRH, gonadotrophin-releasing hormone; HBC, human breast cancer; N/A, not available; RR, response rate; TGI, tumor growth inhibition; TR, tumor regression; T/C, treated divided by control; WT, wild-type.